Piiloset Trehalose Emulsion Eye Drop Study in Moderate or Severe Dry Eye
Sponsor
Kuopio University Hospital (Other)
Overall Status
Completed
CT.gov ID
NCT03569202
Collaborator
Finnsusp Ltd. (Industry), 4Pharma Ltd. (Industry), Business Finland (Other)
64
1
2
5.6
11.4
Study Details
Study Description
Brief Summary
The study evaluates the safety, ocular tolerability and efficacy of emulsion eye drops with sacha inchi seed oil, trehalose and hyaluronic acid in the treatment of moderate or severe dry eye in adult patients. The investigative device is studied in comparison with control eye drops containing hyaluronic acid for up to 30 days.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
N/A |
Detailed Description
According to the current view on dry eye disease, tear film instability, tear film hyperosmolarity, and ocular surface inflammation and damage are identified as etiological factors. The first-line medicinal management options include ocular lubricants such as eye drops and sprays. Emulsion eye drops are a relatively recent entity among topical therapies with an intention to account for deficiencies in the outermost lipid layer of the tear film.
Piiloset Trehalose Emulsion Eye Drops was developed to target and restore the three main layers (mucin, aqueous, and lipid) of the tear film and to counteract the key etiological factors leading to dry eye. Hyaluronic acid and the oil component are intended to restore tear film instability, the hypo-osmolar composition and trehalose as an osmoprotectant are intended to protect the ocular epithelium against hyperosmolarity, and ocular surface inflammation and cellular damage are prevented by the cytoprotective and/or anti-oxidative action of hyaluronic acid, trehalose, and sacha inchi oil components. Additional water binding in the tear film is provided by hyaluronic acid, trehalose, and glycerol. Lubricating the ocular surface by high-molecular-weight hyaluronic acid will extend the precorneal retention time. Adjustments made to certain physical and chemical parameters in the formulation are anticipated to improve tear film spreading and adhesion. The optically clear o/w emulsion formulation is free of preservatives, materials of animal origin, phosphates, or alcohol. The combined action of the individual components is expected to produce clinically relevant mitigation of the signs and symptoms of dry eye.
The study comprises three Parts with scheduled visits on Day 1 (all Parts) and on end-of-study date (Parts 2 and 3) to the study centre. Each study subject will participate in no more than one Part of the study.
Study Design
Study Type:
Interventional
Actual Enrollment
:
64 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Intervention Model Description:
Part 1. Piiloset Trehalose Emulsion Eye Drop is administered on the worst eye four times during office hours of one day at the study centre.
Part 2. Piiloset Trehalose Emulsion Eye Drop is self-administered on a randomized eye and the control eye drops on the contralateral eye three times a day for 10 days in a double-blinded fashion.
Part 3. Either Piiloset Trehalose Emulsion Eye Drop or the control eye drops are self-administered on both eyes three times a day for 30 days in a randomized and double-blinded fashion.Part 1. Piiloset Trehalose Emulsion Eye Drop is administered on the worst eye four times during office hours of one day at the study centre.
Part 2. Piiloset Trehalose Emulsion Eye Drop is self-administered on a randomized eye and the control eye drops on the contralateral eye three times a day for 10 days in a double-blinded fashion.
Part 3. Either Piiloset Trehalose Emulsion Eye Drop or the control eye drops are self-administered on both eyes three times a day for 30 days in a randomized and double-blinded fashion.
Masking:
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose:
Treatment
Official Title:
A Randomized, Active-controlled, Parallel, Double-blind Study on the Safety, Ocular Tolerability and Efficacy of Piiloset Trehalose Emulsion Eye Drop in Adult Patients With Moderate or Severe Dry Eye
Actual Study Start Date
:
May 21, 2018
Actual Primary Completion Date
:
Nov 8, 2018
Actual Study Completion Date
:
Nov 8, 2018
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Emulsion Eye Drops Daily treatment with Piiloset Trehalose Emulsion Eye Drops |
Device: Piiloset Trehalose Emulsion Eye Drops
Topical application of preservative-free multidose Piiloset Trehalose Emulsion Eye Drops 3 times daily
|
| Active Comparator: Control Eye Drops Daily treatment with Hyaluronic Acid Eye Drops (a CE-marked medical device) |
Device: Control Eye Drops
Topical application of preservative-free multidose Hyaluronic Acid Eye Drops 3 times daily
|
Outcome Measures
Primary Outcome Measures
- Change From Baseline OSDI [From baseline to Day 30 (Part 3)]
Ocular Surface Disease Index (OSDI) is a questionnaire consisting of 12 questions subdivided into 3 subscales for measuring the frequency of dry eye symptoms, vision-related quality of life and environmental triggers during the previous week. Answers to each question are on a scale of 0 (None of the time) to 4 (All of the time). Both total OSDI and its subscale scores are on a scale of 0 to 100, calculated as follows: score = (sum of scores for questions answered/number of questions answered) x 25. Higher scores represent greater disability.
- Change From Baseline Tear Osmolarity [From baseline to Day 30 (Part 3)]
Instrumental assay of tear fluid osmolarity (mOsm/L)
- Change From Baseline TBUT [From baseline to Day 30 (Part 3)]
Tear film break-up time (TBUT) (s)
Secondary Outcome Measures
- Change From Baseline Blink Rate [From baseline to Day 30 (Part 3)]
Measurement of spontaneous eyelid blinks per minute
- Change From Baseline Ocular Protection Index (OPI) [From baseline to Day 30 (Part 3)]
OPI is the ratio of TBUT/IBI (IBI, interblink interval calculated from blink rate). An OPI value >1 indicates that TBUT (s) exceeds IBI (s) and that the ocular surface is mostly tear-film protected, because tear film break-ups do not take place within spontaneous blink cycles.
- Change From Baseline Corneal Staining [From baseline to Day 30 (Part 3)]
Corneal staining score is assessed as the average number of punctate dots on an ordinal scale of 0, 1, 2, 3, 4, and 5 (Oxford scale) when compared to a pictorial standard. The number of dots increases on a log scale. Higher score represents more intense staining.
- Change From Baseline Conjunctival (Temporal) Staining [From baseline to Day 30 (Part 3)]
Conjunctival staining score is assessed as the average number of punctate dots on an ordinal scale of 0, 1, 2, 3, 4, and 5 (Oxford scale) when compared to a pictorial standard. The number of dots increases on a log scale. Higher score represents more intense staining.
- Change From Baseline Conjunctival (Nasal) Staining [From baseline to Day 30 (Part 3)]
Conjunctival staining score is assessed as the average number of punctate dots on an ordinal scale of 0, 1, 2, 3, 4, and 5 (Oxford scale) when compared to a pictorial standard. The number of dots increases on a log scale. Higher score represents more intense staining.
Other Outcome Measures
- Change From Baseline Visual Acuity [From baseline to Day 30 (Part 3)]
Best corrected visual acuity (ETDRS charts 1 & 2, 2000 series)
- Change From Baseline Conjunctival Redness [From baseline to Day 30 (Part 3)]
Conjunctival redness score is assessed on an ordinal scale of 0 to 4 (IER grading scale): 0 = not existing, 1 = very slight, 2 = slight, 3 = moderate, 4 = severe. Higher score represents more intense redness.
- Change From Baseline Lid Redness [From baseline to Day 30 (Part 3)]
Eyelid redness score is assessed on an ordinal scale of 0 to 4 (IER grading scale): 0 = not existing, 1 = very slight, 2 = slight, 3 = moderate, 4 = severe. Higher score represents more intense redness.
- Change From Baseline Intraocular Pressure [From baseline to Day 30 (Part 3)]
Intraocular pressure measured using Goldmann applanation tonometry (mmHg)
Eligibility Criteria
Criteria
Ages Eligible for Study:
18 Years
to 80 Years
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
Inclusion Criteria:
-
Ability and willingness to give informed written consent prior to any screening procedure after explanation of the nature and possible consequences of the study.
-
Age between 18 and 80 years.
-
At least two the following conditions (A and B):
- Symptomatic dry eye with OSDI score ≥20. AND B1. Tear film break-up time (TBUT) <10 seconds. OR B2. Positive ocular (corneal and conjunctival) staining pattern
-
Body weight at least 45 kg.
-
Under stable topical and/or systemic therapy for at least 4 weeks before the study procedures and apparent ability and willingness to abstain from other therapies until completion of the study period.
-
Ability and willingness to self-administer eye drops.
-
Ability and willingness to understand and fill in the OSDI questionnaire.
-
Ability and willingness to comply with the study protocol and other study-related procedures.
Exclusion Criteria:
-
History of ocular surgery, trauma, or refractive laser vision correction procedure less than 3 months earlier.
-
Evidence of acute or chronic infection in the cornea or conjunctiva.
-
Diagnosis of Sjögren's syndrome.
-
Unwillingness or apparent disability to discontinue contact lens use during study period and at least one week before the first dosing day.
-
Current ocular allergy symptoms.
-
Known allergy to any constituent of the trehalose emulsion eye drops or control eye drops.
-
Currently pregnant, nursing or planning to become pregnant before completion of the study period.
-
Any other condition that may, in the Investigator's opinion, jeopardize the safety or availability of the subject or adherence to the study protocol or may interfere with the interpretation of the results and would thus make the subject inappropriate for entry in the study.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Department of Ophthalmology, Kuopio University Hospital | Kuopio | Finland | FI-70200 |
Sponsors and Collaborators
- Kuopio University Hospital
- Finnsusp Ltd.
- 4Pharma Ltd.
- Business Finland
Investigators
- Principal Investigator: Kai Kaarniranta, Professor, Kuopio University Hospital
Study Documents (Full-Text)
More Information
Additional Information:
Publications
None provided.Responsible Party:
Kai Kaarniranta,
MD, PhD, Professor,
Kuopio University Hospital
ClinicalTrials.gov Identifier:
NCT03569202
Other Study ID Numbers:
- 5503762
First Posted:
Jun 26, 2018
Last Update Posted:
Dec 13, 2019
Last Verified:
Nov 1, 2019
Individual Participant Data (IPD) Sharing Statement:
No
Plan to Share IPD:
No
Studies a U.S. FDA-regulated Drug Product:
No
Studies a U.S. FDA-regulated Device Product:
No
Keywords provided by Kai Kaarniranta,
MD, PhD, Professor,
Kuopio University Hospital
Additional relevant MeSH terms:
Study Results
Participant Flow
| Recruitment Details | The study comprised three sequential parts. Parts 1 to 3 involved different participants each, thus no participant flow between parts (or periods) took place. |
|---|---|
| Pre-assignment Detail |
| Arm/Group Title | Emulsion Eye Drops | Control Eye Drops |
|---|---|---|
| Arm/Group Description | Daily treatment with Piiloset Trehalose Emulsion Eye Drops Piiloset Trehalose Emulsion Eye Drops: Topical application of preservative-free multidose Piiloset Trehalose Emulsion Eye Drops Part 1: on one eye 4 times during one day Part 2: on one randomized eye 3 times a day for 10 days Part 3: on both eyes 3 times a day for 30 days | Daily treatment with Hyaluronic Acid Eye Drops (a CE-marked medical device) Control Eye Drops: Topical application of preservative-free multidose Hyaluronic Acid Eye Drops Part 1: not used Part 2: on randomized contralateral eye 3 times a day for 10 days Part 3: on both eyes 3 times a day for 30 days |
| Period Title: Part 1 | ||
| STARTED | 3 | 0 |
| COMPLETED | 3 | 0 |
| NOT COMPLETED | 0 | 0 |
| Period Title: Part 1 | ||
| STARTED | 9 | 9 |
| COMPLETED | 9 | 9 |
| NOT COMPLETED | 0 | 0 |
| Period Title: Part 1 | ||
| STARTED | 26 | 26 |
| COMPLETED | 24 | 25 |
| NOT COMPLETED | 2 | 1 |
Baseline Characteristics
| Arm/Group Title | Emulsion Eye Drops | Control Eye Drops | Total |
|---|---|---|---|
| Arm/Group Description | Daily treatment with Piiloset Trehalose Emulsion Eye Drops Results are reported for Part 3 only: Topical application of preservative-free multidose Piiloset Trehalose Emulsion Eye Drops on both eyes 3 times a day for 30 days | Daily treatment with Hyaluronic Acid Eye Drops (a CE-marked medical device) Results are reported for Part 3 only: Topical application of preservative-free multidose Hyaluronic Acid Eye Drops on both eyes 3 times a day for 30 days | Total of all reporting groups |
| Overall Participants | 26 | 26 | 52 |
| Age (years) [Mean (Standard Deviation) ] | |||
| Mean (Standard Deviation) [years] |
55.1
(13.8)
|
51.5
(11.4)
|
53.3
(12.6)
|
| Sex: Female, Male (Count of Participants) | |||
| Female |
18
69.2%
|
18
69.2%
|
36
69.2%
|
| Male |
8
30.8%
|
8
30.8%
|
16
30.8%
|
| Race and Ethnicity Not Collected (Count of Participants) | |||
| Count of Participants [Participants] |
0
0%
|
||
| Region of Enrollment (participants) [Number] | |||
| Finland |
26
100%
|
26
100%
|
52
100%
|
| Body Weight (kg) [Mean (Standard Deviation) ] | |||
| Mean (Standard Deviation) [kg] |
75.1
(11.7)
|
76.9
(15.8)
|
76.0
(13.8)
|
| Ocular Surface Disease Index (OSDI) (units on a scale) [Mean (Standard Deviation) ] | |||
| Mean (Standard Deviation) [units on a scale] |
48.1
(10.8)
|
45.6
(11.3)
|
46.9
(11.0)
|
| Tear Break-Up Time (TBUT) (s) [Mean (Standard Deviation) ] | |||
| Mean (Standard Deviation) [s] |
3.35
(2.62)
|
3.46
(2.04)
|
3.40
(2.33)
|
| Tear osmolarity (mOsm/L) [Mean (Standard Deviation) ] | |||
| Mean (Standard Deviation) [mOsm/L] |
304.0
(11.8)
|
307.3
(12.4)
|
305.6
(12.1)
|
| Blink rate (Blinks/min) [Mean (Standard Deviation) ] | |||
| Mean (Standard Deviation) [Blinks/min] |
18.17
(11.49)
|
22.27
(12.93)
|
20.22
(12.28)
|
| Ocular protection index (OPI) (ratio) [Mean (Standard Deviation) ] | |||
| Mean (Standard Deviation) [ratio] |
0.98
(0.937)
|
1.23
(1.188)
|
1.11
(1.067)
|
| Corneal staining (score on a scale) [Mean (Standard Deviation) ] | |||
| Mean (Standard Deviation) [score on a scale] |
1.37
(0.794)
|
1.21
(0.603)
|
1.29
(0.703)
|
| Conjunctival staining (temporal) (score on a scale) [Mean (Standard Deviation) ] | |||
| Mean (Standard Deviation) [score on a scale] |
1.06
(0.432)
|
1.06
(0.497)
|
1.06
(0.461)
|
| Conjunctival staining (nasal) (score on a scale) [Mean (Standard Deviation) ] | |||
| Mean (Standard Deviation) [score on a scale] |
1.23
(0.430)
|
1.23
(0.406)
|
1.23
(0.414)
|
| Conjunctival redness (score on a scale) [Mean (Standard Deviation) ] | |||
| Mean (Standard Deviation) [score on a scale] |
1.98
(0.685)
|
1.94
(0.829)
|
1.96
(0.753)
|
| Lid redness (score on a scale) [Mean (Standard Deviation) ] | |||
| Mean (Standard Deviation) [score on a scale] |
1.29
(0.751)
|
1.29
(0.724)
|
1.29
(0.730)
|
| Intraocular pressure (mmHg (Goldmann tonometer)) [Mean (Standard Deviation) ] | |||
| Mean (Standard Deviation) [mmHg (Goldmann tonometer)] |
14.92
(2.134)
|
15.69
(2.558)
|
15.31
(2.364)
|
| Ocular surgery, trauma, refractive laser vision correction (Count of Participants) | |||
| Count of Participants [Participants] |
0
0%
|
0
0%
|
0
0%
|
| Eye infection (Count of Participants) | |||
| Count of Participants [Participants] |
0
0%
|
0
0%
|
0
0%
|
| Allergy (Count of Participants) | |||
| Count of Participants [Participants] |
0
0%
|
0
0%
|
0
0%
|
| Current or planned pregnancy and nursing (Count of Participants) | |||
| Count of Participants [Participants] |
0
0%
|
0
0%
|
0
0%
|
Outcome Measures
| Title | Change From Baseline OSDI |
|---|---|
| Description | Ocular Surface Disease Index (OSDI) is a questionnaire consisting of 12 questions subdivided into 3 subscales for measuring the frequency of dry eye symptoms, vision-related quality of life and environmental triggers during the previous week. Answers to each question are on a scale of 0 (None of the time) to 4 (All of the time). Both total OSDI and its subscale scores are on a scale of 0 to 100, calculated as follows: score = (sum of scores for questions answered/number of questions answered) x 25. Higher scores represent greater disability. |
| Time Frame | From baseline to Day 30 (Part 3) |
Outcome Measure Data
| Analysis Population Description |
|---|
| Intent-to-treat (ITT) dataset |
| Arm/Group Title | Emulsion Eye Drops | Control Eye Drops |
|---|---|---|
| Arm/Group Description | Daily treatment with Piiloset Trehalose Emulsion Eye Drops Results are reported for Part 3 only: Topical application of preservative-free multidose Piiloset Trehalose Emulsion Eye Drops on both eyes 3 times a day for 30 days | Daily treatment with Hyaluronic Acid Eye Drops (a CE-marked medical device) Results are reported for Part 3 only: Topical application of preservative-free multidose Hyaluronic Acid Eye Drops on both eyes 3 times a day for 30 days |
| Measure Participants | 26 | 26 |
| Mean (Standard Deviation) [units on a scale] |
-24.64
(16.186)
|
-26.53
(14.620)
|
Statistical Analysis 1
| Statistical Analysis Overview | Comparison Group Selection | Emulsion Eye Drops, Control Eye Drops |
|---|---|---|
| Comments | Between-group comparison, change from baseline | |
| Type of Statistical Test | Superiority | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.662 |
| Comments | A two-sided p-value less than 0.05 was considered statistically significant. | |
| Method | ANOVA | |
| Comments | ||
| Method of Estimation | Estimation Parameter | Mean Difference (Final Values) |
| Estimated Value | 1.8837 | |
| Confidence Interval |
(2-Sided) 95% -6.7081 to 10.4756 |
|
| Parameter Dispersion |
Type: Value: |
|
| Estimation Comments | ||
Statistical Analysis 2
| Statistical Analysis Overview | Comparison Group Selection | Emulsion Eye Drops |
|---|---|---|
| Comments | Within-group change from baseline | |
| Type of Statistical Test | Other | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | <0.0001 |
| Comments | A two-sided p-value less than 0.05 was considered statistically significant. | |
| Method | ANOVA | |
| Comments | ||
| Method of Estimation | Estimation Parameter | Mean Difference (Final Values) |
| Estimated Value | -24.6445 | |
| Confidence Interval |
(2-Sided) 95% -30.7199 to -18.5692 |
|
| Parameter Dispersion |
Type: Value: |
|
| Estimation Comments | ||
Statistical Analysis 3
| Statistical Analysis Overview | Comparison Group Selection | Control Eye Drops |
|---|---|---|
| Comments | Within-group change from baseline | |
| Type of Statistical Test | Other | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | <0.0001 |
| Comments | A two-sided p-value less than 0.05 was considered statistically significant. | |
| Method | ANOVA | |
| Comments | ||
| Method of Estimation | Estimation Parameter | Mean Difference (Final Values) |
| Estimated Value | -26.5283 | |
| Confidence Interval |
(2-Sided) 95% -32.6036 to -20.4529 |
|
| Parameter Dispersion |
Type: Value: |
|
| Estimation Comments | ||
| Title | Change From Baseline Tear Osmolarity |
|---|---|
| Description | Instrumental assay of tear fluid osmolarity (mOsm/L) |
| Time Frame | From baseline to Day 30 (Part 3) |
Outcome Measure Data
| Analysis Population Description |
|---|
| Intent-to-treat (ITT) dataset |
| Arm/Group Title | Emulsion Eye Drops | Control Eye Drops |
|---|---|---|
| Arm/Group Description | Daily treatment with Piiloset Trehalose Emulsion Eye Drops Results are reported for Part 3 only: Topical application of preservative-free multidose Piiloset Trehalose Emulsion Eye Drops on both eyes 3 times a day for 30 days | Daily treatment with Hyaluronic Acid Eye Drops (a CE-marked medical device) Results are reported for Part 3 only: Topical application of preservative-free multidose Hyaluronic Acid Eye Drops on both eyes 3 times a day for 30 days |
| Measure Participants | 26 | 26 |
| Mean (Standard Deviation) [mOsm/L] |
-4.75
(16.224)
|
-1.77
(21.531)
|
Statistical Analysis 1
| Statistical Analysis Overview | Comparison Group Selection | Emulsion Eye Drops, Control Eye Drops |
|---|---|---|
| Comments | Between-group comparison, change from baseline | |
| Type of Statistical Test | Other | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.575 |
| Comments | A two-sided p-value less than 0.05 was considered statistically significant. | |
| Method | ANOVA | |
| Comments | ||
| Method of Estimation | Estimation Parameter | Mean Difference (Final Values) |
| Estimated Value | -2.9808 | |
| Confidence Interval |
(2-Sided) 95% -13.6005 to 7.6390 |
|
| Parameter Dispersion |
Type: Value: |
|
| Estimation Comments | ||
Statistical Analysis 2
| Statistical Analysis Overview | Comparison Group Selection | Emulsion Eye Drops |
|---|---|---|
| Comments | Within-group change from baseline | |
| Type of Statistical Test | Other | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.2098 |
| Comments | A two-sided p-value less than 0.05 was considered statistically significant. | |
| Method | ANOVA | |
| Comments | ||
| Method of Estimation | Estimation Parameter | Mean Difference (Final Values) |
| Estimated Value | -4.7500 | |
| Confidence Interval |
(2-Sided) 95% -12.2593 to 2.7593 |
|
| Parameter Dispersion |
Type: Value: |
|
| Estimation Comments | ||
Statistical Analysis 3
| Statistical Analysis Overview | Comparison Group Selection | Control Eye Drops |
|---|---|---|
| Comments | Within-group change from baseline | |
| Type of Statistical Test | Other | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.6381 |
| Comments | A two-sided p-value less than 0.05 was considered statistically significant. | |
| Method | ANOVA | |
| Comments | ||
| Method of Estimation | Estimation Parameter | Mean Difference (Final Values) |
| Estimated Value | -1.7692 | |
| Confidence Interval |
(2-Sided) 95% -9.2785 to 5.7401 |
|
| Parameter Dispersion |
Type: Value: |
|
| Estimation Comments | ||
| Title | Change From Baseline TBUT |
|---|---|
| Description | Tear film break-up time (TBUT) (s) |
| Time Frame | From baseline to Day 30 (Part 3) |
Outcome Measure Data
| Analysis Population Description |
|---|
| Intent-to-treat (ITT) dataset |
| Arm/Group Title | Emulsion Eye Drops | Control Eye Drops |
|---|---|---|
| Arm/Group Description | Daily treatment with Piiloset Trehalose Emulsion Eye Drops Results are reported for Part 3 only: Topical application of preservative-free multidose Piiloset Trehalose Emulsion Eye Drops on both eyes 3 times a day for 30 days | Daily treatment with Hyaluronic Acid Eye Drops (a CE-marked medical device) Results are reported for Part 3 only: Topical application of preservative-free multidose Hyaluronic Acid Eye Drops on both eyes 3 times a day for 30 days |
| Measure Participants | 26 | 26 |
| Mean (Standard Deviation) [s] |
1.71
(3.424)
|
0.87
(1.809)
|
Statistical Analysis 1
| Statistical Analysis Overview | Comparison Group Selection | Emulsion Eye Drops, Control Eye Drops |
|---|---|---|
| Comments | Between-group comparison, change from baseline | |
| Type of Statistical Test | Other | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.271 |
| Comments | A two-sided p-value less than 0.05 was considered statistically significant. | |
| Method | ANOVA | |
| Comments | ||
| Method of Estimation | Estimation Parameter | Mean Difference (Final Values) |
| Estimated Value | 0.8462 | |
| Confidence Interval |
(2-Sided) 95% -0.6792 to 2.3715 |
|
| Parameter Dispersion |
Type: Value: |
|
| Estimation Comments | ||
Statistical Analysis 2
| Statistical Analysis Overview | Comparison Group Selection | Emulsion Eye Drops |
|---|---|---|
| Comments | Within-group change from baseline | |
| Type of Statistical Test | Other | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.0025 |
| Comments | A two-sided p-value less than 0.05 was considered statistically significant. | |
| Method | ANOVA | |
| Comments | ||
| Method of Estimation | Estimation Parameter | Mean Difference (Final Values) |
| Estimated Value | 1.7115 | |
| Confidence Interval |
(2-Sided) 95% 0.6330 to 2.7901 |
|
| Parameter Dispersion |
Type: Value: |
|
| Estimation Comments | ||
Statistical Analysis 3
| Statistical Analysis Overview | Comparison Group Selection | Control Eye Drops |
|---|---|---|
| Comments | Within-group change from baseline | |
| Type of Statistical Test | Other | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.1134 |
| Comments | A two-sided p-value less than 0.05 was considered statistically significant. | |
| Method | ANOVA | |
| Comments | ||
| Method of Estimation | Estimation Parameter | Mean Difference (Final Values) |
| Estimated Value | 0.8654 | |
| Confidence Interval |
(2-Sided) 95% -0.2132 to 1.9440 |
|
| Parameter Dispersion |
Type: Value: |
|
| Estimation Comments | ||
| Title | Change From Baseline Blink Rate |
|---|---|
| Description | Measurement of spontaneous eyelid blinks per minute |
| Time Frame | From baseline to Day 30 (Part 3) |
Outcome Measure Data
| Analysis Population Description |
|---|
| Intent-to-treat (ITT) dataset |
| Arm/Group Title | Emulsion Eye Drops | Control Eye Drops |
|---|---|---|
| Arm/Group Description | Daily treatment with Piiloset Trehalose Emulsion Eye Drops Results are reported for Part 3 only: Topical application of preservative-free multidose Piiloset Trehalose Emulsion Eye Drops on both eyes 3 times a day for 30 days | Daily treatment with Hyaluronic Acid Eye Drops (a CE-marked medical device) Results are reported for Part 3 only: Topical application of preservative-free multidose Hyaluronic Acid Eye Drops on both eyes 3 times a day for 30 days |
| Measure Participants | 26 | 26 |
| Mean (Standard Deviation) [Blinks/min] |
0.02
(8.721)
|
-2.15
(10.161)
|
Statistical Analysis 1
| Statistical Analysis Overview | Comparison Group Selection | Emulsion Eye Drops, Control Eye Drops |
|---|---|---|
| Comments | Between-group comparison, change from baseline | |
| Type of Statistical Test | Other | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.412 |
| Comments | A two-sided p-value less than 0.05 was considered statistically significant. | |
| Method | ANOVA | |
| Comments | ||
| Method of Estimation | Estimation Parameter | Mean Difference (Final Values) |
| Estimated Value | 2.1731 | |
| Confidence Interval |
(2-Sided) 95% -3.1016 to 7.4477 |
|
| Parameter Dispersion |
Type: Value: |
|
| Estimation Comments | ||
Statistical Analysis 2
| Statistical Analysis Overview | Comparison Group Selection | Emulsion Eye Drops |
|---|---|---|
| Comments | Within-group change from baseline | |
| Type of Statistical Test | Other | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.9918 |
| Comments | A two-sided p-value less than 0.05 was considered statistically significant. | |
| Method | ANOVA | |
| Comments | ||
| Method of Estimation | Estimation Parameter | Mean Difference (Final Values) |
| Estimated Value | 0.01923 | |
| Confidence Interval |
(2-Sided) 95% -3.7105 to 3.7490 |
|
| Parameter Dispersion |
Type: Value: |
|
| Estimation Comments | ||
Statistical Analysis 3
| Statistical Analysis Overview | Comparison Group Selection | Control Eye Drops |
|---|---|---|
| Comments | Within-group change from baseline | |
| Type of Statistical Test | Other | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.2516 |
| Comments | A two-sided p-value less than 0.05 was considered statistically significant. | |
| Method | ANOVA | |
| Comments | ||
| Method of Estimation | Estimation Parameter | Mean Difference (Final Values) |
| Estimated Value | -2.1538 | |
| Confidence Interval |
(2-Sided) 95% -5.8836 to 1.5759 |
|
| Parameter Dispersion |
Type: Value: |
|
| Estimation Comments | ||
| Title | Change From Baseline Ocular Protection Index (OPI) |
|---|---|
| Description | OPI is the ratio of TBUT/IBI (IBI, interblink interval calculated from blink rate). An OPI value >1 indicates that TBUT (s) exceeds IBI (s) and that the ocular surface is mostly tear-film protected, because tear film break-ups do not take place within spontaneous blink cycles. |
| Time Frame | From baseline to Day 30 (Part 3) |
Outcome Measure Data
| Analysis Population Description |
|---|
| Intent-to-treat (ITT) dataset |
| Arm/Group Title | Emulsion Eye Drops | Control Eye Drops |
|---|---|---|
| Arm/Group Description | Daily treatment with Piiloset Trehalose Emulsion Eye Drops Results are reported for Part 3 only: Topical application of preservative-free multidose Piiloset Trehalose Emulsion Eye Drops on both eyes 3 times a day for 30 days | Daily treatment with Hyaluronic Acid Eye Drops (a CE-marked medical device) Results are reported for Part 3 only: Topical application of preservative-free multidose Hyaluronic Acid Eye Drops on both eyes 3 times a day for 30 days |
| Measure Participants | 26 | 26 |
| Mean (Standard Deviation) [ratio] |
0.56
(1.078)
|
0.05
(0.680)
|
Statistical Analysis 1
| Statistical Analysis Overview | Comparison Group Selection | Emulsion Eye Drops, Control Eye Drops |
|---|---|---|
| Comments | Between-group comparison, change from baseline | |
| Type of Statistical Test | Other | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.047 |
| Comments | A two-sided p-value less than 0.05 was considered statistically significant. | |
| Method | ANOVA | |
| Comments | ||
| Method of Estimation | Estimation Parameter | Mean Difference (Final Values) |
| Estimated Value | 0.5093 | |
| Confidence Interval |
(2-Sided) 95% 0.007267 to 1.0113 |
|
| Parameter Dispersion |
Type: Value: |
|
| Estimation Comments | ||
Statistical Analysis 2
| Statistical Analysis Overview | Comparison Group Selection | Emulsion Eye Drops |
|---|---|---|
| Comments | Within-group change from baseline | |
| Type of Statistical Test | Other | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.0026 |
| Comments | A two-sided p-value less than 0.05 was considered statistically significant. | |
| Method | ANOVA | |
| Comments | ||
| Method of Estimation | Estimation Parameter | Mean Difference (Final Values) |
| Estimated Value | 0.5596 | |
| Confidence Interval |
(2-Sided) 95% 0.2046 to 0.9146 |
|
| Parameter Dispersion |
Type: Value: |
|
| Estimation Comments | ||
Statistical Analysis 3
| Statistical Analysis Overview | Comparison Group Selection | Control Eye Drops |
|---|---|---|
| Comments | Within-group change from baseline | |
| Type of Statistical Test | Other | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.7770 |
| Comments | A two-sided p-value less than 0.05 was considered statistically significant. | |
| Method | ANOVA | |
| Comments | ||
| Method of Estimation | Estimation Parameter | Mean Difference (Final Values) |
| Estimated Value | 0.05032 | |
| Confidence Interval |
(2-Sided) 95% -0.3047 to 0.4053 |
|
| Parameter Dispersion |
Type: Value: |
|
| Estimation Comments | ||
| Title | Change From Baseline Corneal Staining |
|---|---|
| Description | Corneal staining score is assessed as the average number of punctate dots on an ordinal scale of 0, 1, 2, 3, 4, and 5 (Oxford scale) when compared to a pictorial standard. The number of dots increases on a log scale. Higher score represents more intense staining. |
| Time Frame | From baseline to Day 30 (Part 3) |
Outcome Measure Data
| Analysis Population Description |
|---|
| Per-protocol (PP) dataset |
| Arm/Group Title | Emulsion Eye Drops | Control Eye Drops |
|---|---|---|
| Arm/Group Description | Daily treatment with Piiloset Trehalose Emulsion Eye Drops Results are reported for Part 3 only: Topical application of preservative-free multidose Piiloset Trehalose Emulsion Eye Drops on both eyes 3 times a day for 30 days | Daily treatment with Hyaluronic Acid Eye Drops (a CE-marked medical device) Results are reported for Part 3 only: Topical application of preservative-free multidose Hyaluronic Acid Eye Drops on both eyes 3 times a day for 30 days |
| Measure Participants | 24 | 25 |
| Mean (Standard Deviation) [score on a scale] |
-0.38
(0.695)
|
-0.22
(0.522)
|
Statistical Analysis 1
| Statistical Analysis Overview | Comparison Group Selection | Emulsion Eye Drops, Control Eye Drops |
|---|---|---|
| Comments | Between-group comparison, change from baseline | |
| Type of Statistical Test | Other | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.447 |
| Comments | A two-sided p-value less than 0.05 was considered statistically significant. | |
| Method | Wilcoxon rank sum test | |
| Comments | ||
| Method of Estimation | Estimation Parameter | Wilcoxon Z statistic |
| Estimated Value | -0.7604 | |
| Confidence Interval |
(2-Sided) % to |
|
| Parameter Dispersion |
Type: Value: |
|
| Estimation Comments | ||
Statistical Analysis 2
| Statistical Analysis Overview | Comparison Group Selection | Emulsion Eye Drops |
|---|---|---|
| Comments | Within-group change from baseline | |
| Type of Statistical Test | Other | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.014 |
| Comments | A two-sided p-value less than 0.05 was considered statistically significant. | |
| Method | Wilcoxon rank sum test | |
| Comments | ||
Statistical Analysis 3
| Statistical Analysis Overview | Comparison Group Selection | Control Eye Drops |
|---|---|---|
| Comments | Within-group change from baseline | |
| Type of Statistical Test | Other | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.070 |
| Comments | A two-sided p-value less than 0.05 was considered statistically significant. | |
| Method | Wilcoxon rank sum test | |
| Comments | ||
| Title | Change From Baseline Conjunctival (Temporal) Staining |
|---|---|
| Description | Conjunctival staining score is assessed as the average number of punctate dots on an ordinal scale of 0, 1, 2, 3, 4, and 5 (Oxford scale) when compared to a pictorial standard. The number of dots increases on a log scale. Higher score represents more intense staining. |
| Time Frame | From baseline to Day 30 (Part 3) |
Outcome Measure Data
| Analysis Population Description |
|---|
| Per-protocol (PP) dataset |
| Arm/Group Title | Emulsion Eye Drops | Control Eye Drops |
|---|---|---|
| Arm/Group Description | Daily treatment with Piiloset Trehalose Emulsion Eye Drops Results are reported for Part 3 only: Topical application of preservative-free multidose Piiloset Trehalose Emulsion Eye Drops on both eyes 3 times a day for 30 days | Daily treatment with Hyaluronic Acid Eye Drops (a CE-marked medical device) Results are reported for Part 3 only: Topical application of preservative-free multidose Hyaluronic Acid Eye Drops on both eyes 3 times a day for 30 days |
| Measure Participants | 24 | 25 |
| Mean (Standard Deviation) [score on a scale] |
-0.19
(0.507)
|
-0.02
(0.810)
|
Statistical Analysis 1
| Statistical Analysis Overview | Comparison Group Selection | Emulsion Eye Drops, Control Eye Drops |
|---|---|---|
| Comments | Between-group comparison, change from baseline | |
| Type of Statistical Test | Other | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.522 |
| Comments | A two-sided p-value less than 0.05 was considered statistically significant. | |
| Method | Wilcoxon rank sum test | |
| Comments | ||
| Method of Estimation | Estimation Parameter | Wilcoxon Z statistic |
| Estimated Value | -0.6396 | |
| Confidence Interval |
(2-Sided) % to |
|
| Parameter Dispersion |
Type: Value: |
|
| Estimation Comments | ||
Statistical Analysis 2
| Statistical Analysis Overview | Comparison Group Selection | Emulsion Eye Drops |
|---|---|---|
| Comments | Within-group change from baseline | |
| Type of Statistical Test | Other | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.119 |
| Comments | A two-sided p-value less than 0.05 was considered statistically significant. | |
| Method | Wilcoxon rank sum test | |
| Comments | ||
Statistical Analysis 3
| Statistical Analysis Overview | Comparison Group Selection | Control Eye Drops |
|---|---|---|
| Comments | Within-group change from baseline | |
| Type of Statistical Test | Other | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.900 |
| Comments | A two-sided p-value less than 0.05 was considered statistically significant. | |
| Method | Wilcoxon rank sum test | |
| Comments | ||
| Title | Change From Baseline Conjunctival (Nasal) Staining |
|---|---|
| Description | Conjunctival staining score is assessed as the average number of punctate dots on an ordinal scale of 0, 1, 2, 3, 4, and 5 (Oxford scale) when compared to a pictorial standard. The number of dots increases on a log scale. Higher score represents more intense staining. |
| Time Frame | From baseline to Day 30 (Part 3) |
Outcome Measure Data
| Analysis Population Description |
|---|
| Per-protocol (PP) dataset |
| Arm/Group Title | Emulsion Eye Drops | Control Eye Drops |
|---|---|---|
| Arm/Group Description | Daily treatment with Piiloset Trehalose Emulsion Eye Drops Results are reported for Part 3 only: Topical application of preservative-free multidose Piiloset Trehalose Emulsion Eye Drops on both eyes 3 times a day for 30 days | Daily treatment with Hyaluronic Acid Eye Drops (a CE-marked medical device) Results are reported for Part 3 only: Topical application of preservative-free multidose Hyaluronic Acid Eye Drops on both eyes 3 times a day for 30 days |
| Measure Participants | 24 | 25 |
| Mean (Standard Deviation) [score on a scale] |
-0.27
(0.608)
|
-0.12
(0.666)
|
Statistical Analysis 1
| Statistical Analysis Overview | Comparison Group Selection | Emulsion Eye Drops, Control Eye Drops |
|---|---|---|
| Comments | Between-group comparison, change from baseline | |
| Type of Statistical Test | Other | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.590 |
| Comments | A two-sided p-value less than 0.05 was considered statistically significant. | |
| Method | Wilcoxon rank sum test | |
| Comments | ||
| Method of Estimation | Estimation Parameter | Wilcoxon Z statistic |
| Estimated Value | -0.5387 | |
| Confidence Interval |
(2-Sided) % to |
|
| Parameter Dispersion |
Type: Value: |
|
| Estimation Comments | ||
Statistical Analysis 2
| Statistical Analysis Overview | Comparison Group Selection | Emulsion Eye Drops |
|---|---|---|
| Comments | Within-group change from baseline | |
| Type of Statistical Test | Other | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.043 |
| Comments | A two-sided p-value less than 0.05 was considered statistically significant. | |
| Method | Wilcoxon rank sum test | |
| Comments | ||
Statistical Analysis 3
| Statistical Analysis Overview | Comparison Group Selection | Control Eye Drops |
|---|---|---|
| Comments | Within-group change from baseline | |
| Type of Statistical Test | Other | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.442 |
| Comments | A two-sided p-value less than 0.05 was considered statistically significant. | |
| Method | Wilcoxon rank sum test | |
| Comments | ||
| Title | Change From Baseline Visual Acuity |
|---|---|
| Description | Best corrected visual acuity (ETDRS charts 1 & 2, 2000 series) |
| Time Frame | From baseline to Day 30 (Part 3) |
Outcome Measure Data
| Analysis Population Description |
|---|
| Safety dataset |
| Arm/Group Title | Emulsion Eye Drops | Control Eye Drops |
|---|---|---|
| Arm/Group Description | Daily treatment with Piiloset Trehalose Emulsion Eye Drops Results are reported for Part 3 only: Topical application of preservative-free multidose Piiloset Trehalose Emulsion Eye Drops on both eyes 3 times a day for 30 days | Daily treatment with Hyaluronic Acid Eye Drops (a CE-marked medical device) Results are reported for Part 3 only: Topical application of preservative-free multidose Hyaluronic Acid Eye Drops on both eyes 3 times a day for 30 days |
| Measure Participants | 26 | 26 |
| Mean (Standard Deviation) [logMAR chart log units] |
-0.03
(0.071)
|
-0.04
(0.080)
|
| Title | Change From Baseline Conjunctival Redness |
|---|---|
| Description | Conjunctival redness score is assessed on an ordinal scale of 0 to 4 (IER grading scale): 0 = not existing, 1 = very slight, 2 = slight, 3 = moderate, 4 = severe. Higher score represents more intense redness. |
| Time Frame | From baseline to Day 30 (Part 3) |
Outcome Measure Data
| Analysis Population Description |
|---|
| Safety dataset |
| Arm/Group Title | Emulsion Eye Drops | Control Eye Drops |
|---|---|---|
| Arm/Group Description | Daily treatment with Piiloset Trehalose Emulsion Eye Drops Results are reported for Part 3 only: Topical application of preservative-free multidose Piiloset Trehalose Emulsion Eye Drops on both eyes 3 times a day for 30 days | Daily treatment with Hyaluronic Acid Eye Drops (a CE-marked medical device) Results are reported for Part 3 only: Topical application of preservative-free multidose Hyaluronic Acid Eye Drops on both eyes 3 times a day for 30 days |
| Measure Participants | 26 | 26 |
| Mean (Standard Deviation) [score on a scale] |
-0.46
(0.647)
|
-0.27
(0.652)
|
Statistical Analysis 1
| Statistical Analysis Overview | Comparison Group Selection | Emulsion Eye Drops, Control Eye Drops |
|---|---|---|
| Comments | Between-group comparison, change from baseline | |
| Type of Statistical Test | Other | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.357 |
| Comments | A two-sided p-value less than 0.05 was considered statistically significant. | |
| Method | Wilcoxon rank sum test | |
| Comments | ||
| Method of Estimation | Estimation Parameter | Wilcoxon Z statistic |
| Estimated Value | 0.9208 | |
| Confidence Interval |
(2-Sided) % to |
|
| Parameter Dispersion |
Type: Value: |
|
| Estimation Comments | ||
Statistical Analysis 2
| Statistical Analysis Overview | Comparison Group Selection | Emulsion Eye Drops |
|---|---|---|
| Comments | Within-group change from baseline | |
| Type of Statistical Test | Other | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.001 |
| Comments | A two-sided p-value less than 0.05 was considered statistically significant. | |
| Method | Wilcoxon rank sum test | |
| Comments | ||
Statistical Analysis 3
| Statistical Analysis Overview | Comparison Group Selection | Control Eye Drops |
|---|---|---|
| Comments | Within-group change from baseline | |
| Type of Statistical Test | Other | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.065 |
| Comments | A two-sided p-value less than 0.05 was considered statistically significant. | |
| Method | Wilcoxon rank sum test | |
| Comments | ||
| Title | Change From Baseline Lid Redness |
|---|---|
| Description | Eyelid redness score is assessed on an ordinal scale of 0 to 4 (IER grading scale): 0 = not existing, 1 = very slight, 2 = slight, 3 = moderate, 4 = severe. Higher score represents more intense redness. |
| Time Frame | From baseline to Day 30 (Part 3) |
Outcome Measure Data
| Analysis Population Description |
|---|
| Safety dataset |
| Arm/Group Title | Emulsion Eye Drops | Control Eye Drops |
|---|---|---|
| Arm/Group Description | Daily treatment with Piiloset Trehalose Emulsion Eye Drops Results are reported for Part 3 only: Topical application of preservative-free multidose Piiloset Trehalose Emulsion Eye Drops on both eyes 3 times a day for 30 days | Daily treatment with Hyaluronic Acid Eye Drops (a CE-marked medical device) Results are reported for Part 3 only: Topical application of preservative-free multidose Hyaluronic Acid Eye Drops on both eyes 3 times a day for 30 days |
| Measure Participants | 26 | 26 |
| Mean (Standard Deviation) [score on a scale] |
-0.37
(0.672)
|
-0.19
(0.549)
|
Statistical Analysis 1
| Statistical Analysis Overview | Comparison Group Selection | Emulsion Eye Drops, Control Eye Drops |
|---|---|---|
| Comments | Between-group comparison, change from baseline | |
| Type of Statistical Test | Other | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.296 |
| Comments | A two-sided p-value less than 0.05 was considered statistically significant. | |
| Method | Wilcoxon rank sum test | |
| Comments | ||
| Method of Estimation | Estimation Parameter | Wilcoxon Z statistic |
| Estimated Value | 1.0440 | |
| Confidence Interval |
(2-Sided) % to |
|
| Parameter Dispersion |
Type: Value: |
|
| Estimation Comments | ||
Statistical Analysis 2
| Statistical Analysis Overview | Comparison Group Selection | Emulsion Eye Drops |
|---|---|---|
| Comments | Within-group change from baseline | |
| Type of Statistical Test | Other | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.012 |
| Comments | A two-sided p-value less than 0.05 was considered statistically significant. | |
| Method | Wilcoxon rank sum test | |
| Comments | ||
Statistical Analysis 3
| Statistical Analysis Overview | Comparison Group Selection | Control Eye Drops |
|---|---|---|
| Comments | Within-group change from baseline | |
| Type of Statistical Test | Other | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | >0.05 |
| Comments | A two-sided p-value less than 0.05 was considered statistically significant. | |
| Method | Wilcoxon rank sum test | |
| Comments | ||
| Title | Change From Baseline Intraocular Pressure |
|---|---|
| Description | Intraocular pressure measured using Goldmann applanation tonometry (mmHg) |
| Time Frame | From baseline to Day 30 (Part 3) |
Outcome Measure Data
| Analysis Population Description |
|---|
| Safety dataset |
| Arm/Group Title | Emulsion Eye Drops | Control Eye Drops |
|---|---|---|
| Arm/Group Description | Daily treatment with Piiloset Trehalose Emulsion Eye Drops Results are reported for Part 3 only: Topical application of preservative-free multidose Piiloset Trehalose Emulsion Eye Drops on both eyes 3 times a day for 30 days | Daily treatment with Hyaluronic Acid Eye Drops (a CE-marked medical device) Results are reported for Part 3 only: Topical application of preservative-free multidose Hyaluronic Acid Eye Drops on both eyes 3 times a day for 30 days |
| Measure Participants | 26 | 26 |
| Mean (Standard Deviation) [mmHg] |
-0.12
(1.705)
|
-0.42
(1.653)
|
| Title | Change From Baseline Tear Osmolarity in Hyperosmolar Participants |
|---|---|
| Description | Instrumental assay of tear fluid osmolarity (mOsm/L) in a hyperosmolar subgroup of participants (mean of eyes ≥308 mOsm/L) |
| Time Frame | From baseline to Day 30 (Part 3) |
Outcome Measure Data
| Analysis Population Description |
|---|
| ITT population with hyperosmolar tears (mean of eyes ≥308 mOsm/L) |
| Arm/Group Title | Emulsion Eye Drops | Control Eye Drops |
|---|---|---|
| Arm/Group Description | Daily treatment with Piiloset Trehalose Emulsion Eye Drops Results are reported for Part 3 only: Topical application of preservative-free multidose Piiloset Trehalose Emulsion Eye Drops on both eyes 3 times a day for 30 days | Daily treatment with Hyaluronic Acid Eye Drops (a CE-marked medical device) Results are reported for Part 3 only: Topical application of preservative-free multidose Hyaluronic Acid Eye Drops on both eyes 3 times a day for 30 days |
| Measure Participants | 8 | 14 |
| Mean (Standard Deviation) [mOsm/L] |
-17.06
(10.425)
|
-15.07
(16.596)
|
Statistical Analysis 1
| Statistical Analysis Overview | Comparison Group Selection | Emulsion Eye Drops, Control Eye Drops |
|---|---|---|
| Comments | Between-group comparison, change from baseline | |
| Type of Statistical Test | Other | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.764 |
| Comments | A two-sided p-value less than 0.05 was considered statistically significant. | |
| Method | ANOVA | |
| Comments | ||
| Method of Estimation | Estimation Parameter | Mean Difference (Final Values) |
| Estimated Value | -1.9911 | |
| Confidence Interval |
(2-Sided) 95% -15.6117 to 11.6295 |
|
| Parameter Dispersion |
Type: Value: |
|
| Estimation Comments | ||
Statistical Analysis 2
| Statistical Analysis Overview | Comparison Group Selection | Emulsion Eye Drops |
|---|---|---|
| Comments | Within-group change from baseline | |
| Type of Statistical Test | Other | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.0038 |
| Comments | A two-sided p-value less than 0.05 was considered statistically significant. | |
| Method | ANOVA | |
| Comments | ||
| Method of Estimation | Estimation Parameter | Mean Difference (Final Values) |
| Estimated Value | -17.0625 | |
| Confidence Interval |
(2-Sided) 95% -27.9280 to -6.1970 |
|
| Parameter Dispersion |
Type: Value: |
|
| Estimation Comments | ||
Statistical Analysis 3
| Statistical Analysis Overview | Comparison Group Selection | Control Eye Drops |
|---|---|---|
| Comments | Within-group change from baseline | |
| Type of Statistical Test | Other | |
| Comments | ||
| Statistical Test of Hypothesis | p-Value | 0.0011 |
| Comments | A two-sided p-value less than 0.05 was considered statistically significant. | |
| Method | ANOVA | |
| Comments | ||
| Method of Estimation | Estimation Parameter | Mean Difference (Final Values) |
| Estimated Value | -15.0714 | |
| Confidence Interval |
(2-Sided) 95% -23.2850 to -6.8579 |
|
| Parameter Dispersion |
Type: Value: |
|
| Estimation Comments | ||
Adverse Events
| Time Frame | Part 1: 1 day; Part 2: 10 days; Part 3: 30 days | |||
|---|---|---|---|---|
| Adverse Event Reporting Description | ADE (adverse device effect); AE (adverse event); SADE (serious adverse device effect); SAE (serious adverse event); USADE (unanticipated serious adverse device effect) Total Number of Participants at Risk (safety population); Emulsion Eye Drops: 3 + 9 + 26 = 38 participants; Control Eye Drops: 0 + 9 + 26 = 35 participants. Adverse events were collected throughout the study during visits to study site, in study diaries, and from direct contacts to study personnel between visits. | |||
| Arm/Group Title | Emulsion Eye Drops | Control Eye Drops | ||
| Arm/Group Description | Daily treatment with Piiloset Trehalose Emulsion Eye Drops Adverse events are reported for the safety population in all parts of the study, including all randomized subjects who received Piiloset Trehalose Emulsion Eye Drops at least once and from whom at least one safety measurement was obtained after randomization. | Daily treatment with Hyaluronic Acid Eye Drops (a CE-marked medical device) Adverse events are reported for the safety population in all parts of the study, including all randomized subjects who received Hyaluronic Acid Eye Drops at least once and from whom at least one safety measurement was obtained after randomization. | ||
All Cause Mortality |
||||
| Emulsion Eye Drops | Control Eye Drops | |||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 0/38 (0%) | 0/35 (0%) | ||
Serious Adverse Events |
||||
| Emulsion Eye Drops | Control Eye Drops | |||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 0/38 (0%) | 0/35 (0%) | ||
Other (Not Including Serious) Adverse Events |
||||
| Emulsion Eye Drops | Control Eye Drops | |||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 1/38 (2.6%) | 0/35 (0%) | ||
| Skin and subcutaneous tissue disorders | ||||
| AE (adverse event) | 1/38 (2.6%) | 1 | 0/35 (0%) | 0 |
Limitations/Caveats
[Not Specified]
More Information
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
The disclosure restrictions on the PI are that the sponsor can review results communications 30 days prior to planned date of public release. If public release of information in communications may jeopardize protecting sponsor's immaterial rights, the sponsor can additionally embargo the communications for a period that is less than or equal to 90 days from the time submitted to the sponsor for review. The sponsor cannot require changes to the communication and cannot extend the embargo.
Results Point of Contact
| Name/Title | Dr. Jarmo Laihia, Manager of Clinical & Formulation Development |
|---|---|
| Organization | Finnsusp Ltd. |
| Phone | 358 50 377 1212 |
| jarmo.laihia@piiloset.fi |
Responsible Party:
Kai Kaarniranta,
MD, PhD, Professor,
Kuopio University Hospital
ClinicalTrials.gov Identifier:
NCT03569202
Other Study ID Numbers:
- 5503762
First Posted:
Jun 26, 2018
Last Update Posted:
Dec 13, 2019
Last Verified:
Nov 1, 2019