Perfluorohexyloctane (NOV03) for the Treatment of Signs and Symptoms of Dry Eye Disease (DED)

• Sponsor: Novaliq GmbH (Industry)
• Overall Status: Completed
• CT.gov ID: NCT03333057
• Collaborator: (none)
• Enrollment: 336
• Locations: 11
• Arms: 4
• Actual Duration (Months): 6.7
• Patients Per Site: 30.5
• Patients Per Site Per Month: 4.5

Study Details

Study Description

Brief Summary

This study will evaluate the efficacy, safety and tolerability of perfluorohexyloctane (NOV03) at two different dosing regimens compared to saline solution in subjects with Dry Eye Disease (DED).

Condition or Disease	Intervention/Treatment	Phase
Dry Eye Disease (DED)	Drug: NOV03; Drug: Placebo	Phase 2

Study Design

• Study Type: Interventional
• Actual Enrollment: 336 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Double (Participant, Investigator)
• Primary Purpose: Treatment
• Official Title: A Phase 2, Multi-Center, Randomized, Double-Masked, Saline-Controlled Study to Evaluate the Effect of Perfluorohexyloctane (NOV03) at Two Different Dosing Regimens on Signs and Symptoms of Dry Eye Disease (DED)
• Actual Study Start Date: Jan 3, 2018
• Actual Primary Completion Date: May 18, 2018
• Actual Study Completion Date: Jul 27, 2018

Arms and Interventions

Arm	Intervention/Treatment
Experimental: NOV03 4 times daily (QID) Perfluorohexyloctance solution 4 times daily (QID)	Drug: NOV03; Perfluorohexyloctane
Experimental: NOV03 2 times daily (BID) Perfluorohexyloctance solution 2 times daily (BID)	Drug: NOV03; Perfluorohexyloctane
Placebo Comparator: Placebo 4 times daily (QID) Saline solution (0.9% sodium chloride solution) 4 times daily (QID)	Drug: Placebo; Saline solution (0.9% sodium chloride solution)
Placebo Comparator: Placebo 2 times daily (BID) Saline solution (0.9% sodium chloride solution) 2 times daily (BID)	Drug: Placebo; Saline solution (0.9% sodium chloride solution)

Outcome Measures

Primary Outcome Measures

1. Corneal Fluorescein Staining (CFS) Total (National Eye Institute (NEI) Grading) [baseline and 2 months]

   The primary efficacy variable was comparison of absolute and change from baseline between treatments for total Corneal Fluorescein Staining (tCFS) according to the National Eye Institute grading (NEI Scale) at week 8 The NEI Scale is a standardized grading system. 0-3 is used for each of the five areas on each cornea. Grade 0 will be specified when no staining is present. The maximum total score for each eye is 15. Higher values describe greater staining and corneal damage.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Signed ICF (Informed Consent Form)

• Subject-reported history of Drye Eye Disease (DED) in both eyes

• Ability and willingness to follow instructions, including participation in all study assessments and visits

Exclusion Criteria:

• Women who are pregnant, nursing or planning pregnancy

• Unwillingness to submit a blood pregnancy test at screening and the last visit (or early termination visit) if of childbearing potential, or unwillingness to use acceptable means of birth control

• Clinically significant slit-lamp findings or abnormal lid anatomy at screening

• Ocular/peri-ocular malignancy

• History of herpetic keratitis

• Active ocular allergies or ocular allergies that are expected to be active during the study

• Ongoing ocular or systemic infection

• Wear contact lenses within 1 month prior to screening or anticipated use of contact lenses during the study

• Intra-ocular surgery or ocular laser surgery within the previous 6 months, or have planned ocular and/or lid surgeries over the study period

• Presence of uncontrolled systemic diseases

• Presence of known allergy and/or sensitivity to the study drug or saline components

• Use of any topical steroids treatments, topical cyclosporine, lifitegrast, serum tears or topical anti-glaucoma medication within 2 months prior to screening

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Investigational site	Artesia	California	United States	90701
2	Investigational Site	Newport Beach	California	United States	92660
3	Investigational Site	Newport Beach	California	United States	92663
4	Investigational Site	Santa Ana	California	United States	92705
5	Investigational Site	Torrance	California	United States	90505
6	Investigational site	Jacksonville	Florida	United States	32256
7	Investigational Site	Lake Villa	Illinois	United States	60046
8	Investigational Site	Kansas City	Missouri	United States	64111
9	Investigational Site	Pennington	New Jersey	United States	08534
10	Investigational Site	Nashville	Tennessee	United States	37205
11	Investigational Site	Nashville	Tennessee	United States	37215

Sponsors and Collaborators

• Novaliq GmbH

Investigators

• Study Director: Sonja Kroesser, PhD, Novaliq GmbH

Study Documents (Full-Text)

• Study Protocol and Statistical Analysis Plan - Dec 7, 2017

More Information

Additional Information:

• Publication of Study Results in Cornea

Publications

None provided.

• Responsible Party: Novaliq GmbH
• ClinicalTrials.gov Identifier: NCT03333057
• Other Study ID Numbers: NVU-002 (SEECASE)
• First Posted: Nov 6, 2017
• Last Update Posted: Jan 4, 2022
• Last Verified: Oct 1, 2019

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Additional relevant MeSH terms: Dry Eye Syndromes; Keratoconjunctivitis Sicca; Eye Diseases

Study Results

Participant Flow

Arm/Group Title	NOV03 2 Times Daily (BID)	NOV03 4 Times Daily (QID)	Placebo BID/QID
Arm/Group Description	Perfluorohexyloctance solution 2 times daily (BID) NOV03: Perfluorohexyloctane	Perfluorohexyloctance solution 4 times daily NOV03: Perfluorohexyloctane	Saline solution (0.9% sodium chloride solution) 2/4 times daily (BID/QID) Placebo: Saline solution (0.9% sodium chloride solution)
Period Title: Overall Study
STARTED	111	114	111
COMPLETED	105	110	108
NOT COMPLETED	6	4	3

Baseline Characteristics

Arm/Group Title	NOV03 2 Times Daily (BID)	NOV03 4 Times Daily (QID)	Placebo BID/QID	Total
Arm/Group Description	Perfluorohexyloctance solution 2 times daily (BID) NOV03: Perfluorohexyloctane	Perfluorohexyloctance solution 4 times daily NOV03: Perfluorohexyloctane	Saline solution (0.9% sodium chloride solution) 2/4 times daily (BID/QID) Placebo: Saline solution (0.9% sodium chloride solution)	Total of all reporting groups
Overall Participants	111	114	111	336
Age (years) [Mean (Full Range) ]
Mean (Full Range) [years]	54.0	53.0	53.8	53.6
Sex: Female, Male (Count of Participants)
Female	84 75.7%	79 69.3%	80 72.1%	243 72.3%
Male	27 24.3%	35 30.7%	31 27.9%	93 27.7%
Ethnicity (NIH/OMB) (Count of Participants)
Hispanic or Latino	26 23.4%	30 26.3%	21 18.9%	77 22.9%
Not Hispanic or Latino	85 76.6%	84 73.7%	90 81.1%	259 77.1%
Unknown or Not Reported	0 0%	0 0%	0 0%	0 0%
Race (NIH/OMB) (Count of Participants)
American Indian or Alaska Native	0 0%	1 0.9%	1 0.9%	2 0.6%
Asian	20 18%	18 15.8%	21 18.9%	59 17.6%
Native Hawaiian or Other Pacific Islander	0 0%	2 1.8%	0 0%	2 0.6%
Black or African American	13 11.7%	13 11.4%	8 7.2%	34 10.1%
White	78 70.3%	80 70.2%	81 73%	239 71.1%
More than one race	0 0%	0 0%	0 0%	0 0%
Unknown or Not Reported	0 0%	0 0%	0 0%	0 0%
Region of Enrollment (participants) [Number]
United States	111 100%	114 100%	111 100%	336 100%

Outcome Measures

1. Primary Outcome

Title	Corneal Fluorescein Staining (CFS) Total (National Eye Institute (NEI) Grading)
Description	The primary efficacy variable was comparison of absolute and change from baseline between treatments for total Corneal Fluorescein Staining (tCFS) according to the National Eye Institute grading (NEI Scale) at week 8 The NEI Scale is a standardized grading system. 0-3 is used for each of the five areas on each cornea. Grade 0 will be specified when no staining is present. The maximum total score for each eye is 15. Higher values describe greater staining and corneal damage.
Time Frame	baseline and 2 months

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	NOV03 2 Times Daily (BID)	NOV03 4 Times Daily (QID)	Placebo BID/QID
Arm/Group Description	Perfluorohexyloctance solution 2 times daily (BID) NOV03: Perfluorohexyloctane	Perfluorohexyloctance solution 4 times daily NOV03:Perfluorohexyloctane	Saline solution (0.9% sodium chloride solution) 2/4 times daily (BID/QID) Placebo: Saline solution (0.9% sodium chloride solution)
Measure Participants	111	114	111
Mean (95% Confidence Interval) [score on a scale]	-1.78	-2.11	-0.93

Adverse Events

Time Frame	whole study duration (8 weeks per patient)
Arm/Group Title	NOV03 2 Times Daily (BID)		NOV03 4 Times Daily (QID)		Placebo BID/QID
Arm/Group Description	Perfluorohexyloctance solution 2 times daily (BID) NOV03: Perfluorohexyloctane		Perfluorohexyloctance solution 4 times daily NOV03: Perfluorohexyloctane		Saline solution (0.9% sodium chloride solution) 2/4 times daily (BID/QID) Placebo: Saline solution (0.9% sodium chloride solution)
All Cause Mortality
	NOV03 2 Times Daily (BID)		NOV03 4 Times Daily (QID)		Placebo BID/QID
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/111 (0%)		0/114 (0%)		0/111 (0%)
Serious Adverse Events
	NOV03 2 Times Daily (BID)		NOV03 4 Times Daily (QID)		Placebo BID/QID
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	1/111 (0.9%)		3/114 (2.6%)		0/111 (0%)
Gastrointestinal disorders
Diverticulum	0/111 (0%)	0	1/114 (0.9%)	1	0/111 (0%)	0
General disorders
Chest pain	1/111 (0.9%)	1	0/114 (0%)	0	0/111 (0%)	0
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Endometrial adenocarcinoma	0/111 (0%)	0	1/114 (0.9%)	1	0/111 (0%)	0
Pregnancy, puerperium and perinatal conditions
Abortion spontaneous	0/111 (0%)	0	1/114 (0.9%)	1	0/111 (0%)	0
Other (Not Including Serious) Adverse Events
	NOV03 2 Times Daily (BID)		NOV03 4 Times Daily (QID)		Placebo BID/QID
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	1/111 (0.9%)		4/114 (3.5%)		3/111 (2.7%)
Eye disorders
Vision blurred	0/111 (0%)	0	2/114 (1.8%)	2	1/111 (0.9%)	1
General disorders
Instillation site pain	1/111 (0.9%)	2	2/114 (1.8%)	2	0/111 (0%)	0
Instillation site irritation	0/111 (0%)	0	0/114 (0%)	0	2/111 (1.8%)	2

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.