EASY: Extended Access to Sollpura Over Years

• Sponsor: Anthera Pharmaceuticals (Industry)
• Overall Status: Completed
• CT.gov ID: NCT02823964
• Collaborator: (none)
• Enrollment: 25
• Locations: 15
• Arm: 1
• Actual Duration (Months): 17.9
• Patients Per Site: 1.7
• Patients Per Site Per Month: 0.1

Study Details

Study Description

Brief Summary

Liprotamase consists of 3 soluble, non-porcine digestive enzymes, lipase, protease, and amylase, combined in a fixed ratio. Liprotamase is stable in the stomach and can be formulated without enteric coating for administration either as a capsule or as a dosing solution dissolved in water or juice.

Condition or Disease	Intervention/Treatment	Phase
Exocrine Pancreatic Insufficiency; Cystic Fibrosis	Drug: Liprotamase	Phase 4

Detailed Description

The purpose of the present study is to evaluate the safety of long-term use of liprotamase in the management of cystic fibrosis-related exocrine pancreatic insufficiency

Study Design

• Study Type: Interventional
• Actual Enrollment: 25 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: An Open-Label Study Evaluating the Efficacy and Safety of Liprotamase in Subjects With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis
• Actual Study Start Date: Sep 1, 2016
• Actual Primary Completion Date: Mar 1, 2018
• Actual Study Completion Date: Mar 1, 2018

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Liprotamase Oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement	Drug: Liprotamase; Oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement; Other Names: Sollpura

Outcome Measures

Primary Outcome Measures

1. Safety, as Measured by Number of Participants With Adverse Events Including Clinical or Laboratory Abnormalities [12 months]

   Descriptive analysis

Eligibility Criteria

Criteria

• Ages Eligible for Study: 7 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Male or female subjects with cystic fibrosis-related exocrine pancreatic insufficiency who received liprotamase and completed Study AN-EPI3331 (SOLUTION: Study of Oral Liprotamase Unit-Matched Therapy Of Non-Porcine Origin in Patients With Cystic Fibrosis)

Exclusion Criteria:

• Any medical, psychological, or social condition that may put the subject at increased risk by participating in this study.

• Females who are nursing, pregnant, intending to become pregnant, or intending to nurse during the time of the study.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Investigator Site 114	Aurora	Colorado	United States	80045
2	Investigator Site 134	Jackson	Mississippi	United States	39216
3	Investigator Site 121	Burlington	Vermont	United States	05401
4	Investigator Site 501	Brno		Czechia	625 00
5	Investigator Site 303	Debrecen	Hajdu-Bihar	Hungary	4031
6	Investigator Site 302	Torokbalint	Pest County	Hungary	2045
7	Investigator Site 304	Mosdos	Somogy County	Hungary	7257
8	Investigator Site 301	Ajka	Veszprem County	Hungary	8400
9	Investigator Site 601	Jerusalem		Israel	9124001
10	Investigator Site 203	Karpacz		Poland	58-540
11	Investigator Site 206	Lodz		Poland	90-329
12	Investigator Site 205	Lublin		Poland	20-362
13	Investigator Site 202	Rabka-Zdroj		Poland	34-700
14	Investigator Site 209	Rzeszow		Poland	35-612
15	Investigator Site 401	Madrid		Spain	28046

Sponsors and Collaborators

• Anthera Pharmaceuticals

Investigators

None specified.

Study Documents (Full-Text)

• Study Protocol and Statistical Analysis Plan - Oct 31, 2017

More Information

Publications

None provided.

• Responsible Party: Anthera Pharmaceuticals
• ClinicalTrials.gov Identifier: NCT02823964
• Other Study ID Numbers: AN-EPI3334
• First Posted: Jul 6, 2016
• Last Update Posted: May 17, 2018
• Last Verified: Apr 1, 2018

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Keywords provided by Anthera Pharmaceuticals: exocrine pancreatic insufficiency; cystic fibrosis; pancreatic enzyme replacement therapy
• Additional relevant MeSH terms: Cystic Fibrosis; Exocrine Pancreatic Insufficiency; Fibrosis

Study Results

Participant Flow

Arm/Group Title	Liprotamase
Arm/Group Description	Oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement Liprotamase: Oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement
Period Title: Overall Study
STARTED	25
COMPLETED	20
NOT COMPLETED	5

Baseline Characteristics

Arm/Group Title	Liprotamase
Arm/Group Description	Oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement Liprotamase: Oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement
Overall Participants	25
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	22.94 (6.84)
Sex: Female, Male (Count of Participants)
Female	11 44%
Male	14 56%
Race (NIH/OMB) (Count of Participants)
American Indian or Alaska Native	0 0%
Asian	0 0%
Native Hawaiian or Other Pacific Islander	0 0%
Black or African American	0 0%
White	25 100%
More than one race	0 0%
Unknown or Not Reported	0 0%

Outcome Measures

1. Primary Outcome

Title	Safety, as Measured by Number of Participants With Adverse Events Including Clinical or Laboratory Abnormalities
Description	Descriptive analysis
Time Frame	12 months

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Liprotamase
Arm/Group Description	Oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement Liprotamase: Oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement
Measure Participants	25
Safety population	25 100%
total number of subjects with adverse events	9 36%

Adverse Events

Time Frame	1 year
Arm/Group Title	Liprotamase
Arm/Group Description	Oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement Liprotamase: Oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement
All Cause Mortality
	Liprotamase
	Affected / at Risk (%)	# Events
Total	0/25 (0%)
Serious Adverse Events
	Liprotamase
	Affected / at Risk (%)	# Events
Total	3/25 (12%)
Infections and infestations
Infective pulmonary exacerbation of cystic fibrosis	2/25 (8%)	2
Injury, poisoning and procedural complications
Scalded Skin	1/25 (4%)	1
Other (Not Including Serious) Adverse Events
	Liprotamase
	Affected / at Risk (%)	# Events
Total	9/25 (36%)
Gastrointestinal disorders
Gastrointestinal disorders	2/25 (8%)	2
General disorders
Abdominal pain	1/25 (4%)	1
Hepatobiliary disorders
Cholelithiasis	1/25 (4%)	1
Immune system disorders
Minocycline allergy	1/25 (4%)	1
Infections and infestations
Infective pulmonary exacerbation of CF	4/25 (16%)	4
Viral infection	2/25 (8%)	2
Injury, poisoning and procedural complications
Scalded skin	1/25 (4%)	1
Investigations
Investigations	3/25 (12%)	3
Musculoskeletal and connective tissue disorders
Musculoskeletal and connective tissue disorders	2/25 (8%)	2
Renal and urinary disorders
Kidney stone	2/25 (8%)	2
Respiratory, thoracic and mediastinal disorders
Hemoptoe	1/25 (4%)	1

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

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