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SOLUTION: Study of Oral Liprotamase Unit-Matched Therapy Of Non-Porcine Origin in Patients With Cystic Fibrosis

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ClinicalTrials.gov Identifier: NCT02279498
Recruitment Status : Completed
First Posted : October 31, 2014
Results First Posted : August 14, 2018
Last Update Posted : August 14, 2018
Sponsor:
Information provided by (Responsible Party):
Anthera Pharmaceuticals

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Exocrine Pancreatic Insufficiency
Cystic Fibrosis
Interventions Drug: Liprotamase
Drug: porcine (pig) PERT
Enrollment 128
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Liprotamase Porcine (Pig) PERT
Hide Arm/Group Description

Individually-optimized dose to be administered orally

Liprotamase: oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement

Individually-optimized dose to be administered orally

porcine (pig) PERT: oral, enterically-coated, pancreatic replacement enzymes prepared from a porcine source

Period Title: Overall Study
Started 65 63
Completed 47 59
Not Completed 18 4
Reason Not Completed
Adverse Event             3             0
Lack of Efficacy             4             0
Lost to Follow-up             0             1
Withdrawal by Subject             9             2
Undefined             2             1
Arm/Group Title Liprotamase Porcine (Pig) PERT Total
Hide Arm/Group Description

Individually-optimized dose to be administered orally

Liprotamase: oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement

Individually-optimized dose to be administered orally

porcine (pig) PERT: oral, enterically-coated, pancreatic replacement enzymes prepared from a porcine source

Total of all reporting groups
Overall Number of Baseline Participants 65 63 128
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 65 participants 63 participants 128 participants
22.5  (8.54) 21.0  (8.95) 21.8  (8.74)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 65 participants 63 participants 128 participants
Female
30
  46.2%
31
  49.2%
61
  47.7%
Male
35
  53.8%
32
  50.8%
67
  52.3%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 65 participants 63 participants 128 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
1
   1.5%
0
   0.0%
1
   0.8%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
White
63
  96.9%
61
  96.8%
124
  96.9%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
1
   1.5%
2
   3.2%
3
   2.3%
Coefficient of fat absorption (CFA)  
Mean (Full Range)
Unit of measure:  Fat absorbed as percent of fat ingested
Number Analyzed 65 participants 63 participants 128 participants
88.7
(80 to 98)
89.4
(79 to 97)
89.1
(79 to 98)
Coefficient of nitrogen absorption (CNA)  
Mean (Full Range)
Unit of measure:  % of nitrogen ingested
Number Analyzed 65 participants 63 participants 128 participants
96.9
(92 to 99)
97.3
(94 to 99)
97.1
(92 to 99)
pre-randomization enzyme dose  
Mean (Full Range)
Unit of measure:  Lipase units/kg/day
Number Analyzed 65 participants 63 participants 128 participants
6299
(1190 to 9981)
6264
(1692 to 9962)
6282
(1190 to 9981)
1.Primary Outcome
Title Treatment Difference in Coefficient of Fat Absorption (CFA) Change From Baseline
Hide Description The primary endpoint evaluates the difference between treatment arms in change from baseline in coefficient of fat absorption (CFA). As such, descriptive statistics for individual treatment arms are not provided in this measure, but are reported in the secondary endpoints
Time Frame Baseline, 7 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis population evaluates all subjects who received at least one dose of study drug. Missing Visit 7 CFA values were multiply imputed using baseline CFA, baseline BMI, sex, age, acid suppression usage, and region.
Arm/Group Title Treatment Difference
Hide Arm/Group Description:
analysed as LS mean difference in change in CFA from baseline for Liprotamase - Porcine enzymes
Overall Number of Participants Analyzed 128
Least Squares Mean (95% Confidence Interval)
Unit of Measure: percent change from baseline
-11.852
(-16.0 to -7.7)
2.Secondary Outcome
Title Coefficient of Fat Absorption (CFA)
Hide Description Change from baseline in coefficient of fat absorption
Time Frame Baseline, 7 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis population evaluates observed case data without multiple imputation for missing values
Arm/Group Title Liprotamase Porcine (Pig) PERT
Hide Arm/Group Description:

Individually-optimized dose to be administered orally

Liprotamase: oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement

Individually-optimized dose to be administered orally

porcine (pig) PERT: oral, enterically-coated, pancreatic replacement enzymes prepared from a porcine source

Overall Number of Participants Analyzed 53 62
Mean (Standard Deviation)
Unit of Measure: fat absorbed as % of fat ingested
76.5  (16.13) 89.5  (5.84)
3.Secondary Outcome
Title Coefficient of Nitrogen Absorption (CNA)
Hide Description Change from baseline in coefficient of nitrogen absorption
Time Frame Baseline, 7 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis population evaluates observed case data without multiple imputation for missing values
Arm/Group Title Liprotamase Porcine (Pig) PERT
Hide Arm/Group Description:

Individually-optimized dose to be administered orally

Liprotamase: oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement

Individually-optimized dose to be administered orally

porcine (pig) PERT: oral, enterically-coated, pancreatic replacement enzymes prepared from a porcine source

Overall Number of Participants Analyzed 53 62
Mean (Standard Deviation)
Unit of Measure: percent of nitrogen ingested
95.8  (2.46) 97.5  (1.21)
Time Frame 6 months
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Liprotamase Porcine (Pig) PERT
Hide Arm/Group Description

Individually-optimized dose to be administered orally

Liprotamase: oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement

Individually-optimized dose to be administered orally

porcine (pig) PERT: oral, enterically-coated, pancreatic replacement enzymes prepared from a porcine source

All-Cause Mortality
Liprotamase Porcine (Pig) PERT
Affected / at Risk (%) Affected / at Risk (%)
Total   0/65 (0.00%)      0/63 (0.00%)    
Show Serious Adverse Events Hide Serious Adverse Events
Liprotamase Porcine (Pig) PERT
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   7/65 (10.77%)      6/63 (9.52%)    
Gastrointestinal disorders     
Constipation  1  1/65 (1.54%)  1 0/63 (0.00%)  0
Distal intestinal obstruction syndrome  1  1/65 (1.54%)  1 0/63 (0.00%)  0
General disorders     
Systemic inflammatory response syndrome  1  1/65 (1.54%)  1 0/63 (0.00%)  0
Infections and infestations     
Infective pulmonary exacerbation of cystic fibrosis  1  3/65 (4.62%)  3 5/63 (7.94%)  5
Injury, poisoning and procedural complications     
Tibia fracture  1  0/65 (0.00%)  0 1/63 (1.59%)  1
Investigations     
Clostridium test positive  1  1/65 (1.54%)  1 0/63 (0.00%)  0
Renal and urinary disorders     
Hydronephrosis  1  1/65 (1.54%)  1 0/63 (0.00%)  0
1
Term from vocabulary, MedDRA (19.0)
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 2%
Liprotamase Porcine (Pig) PERT
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   41/65 (63.08%)      38/63 (60.32%)    
Gastrointestinal disorders     
Constipation  1  2/65 (3.08%)  2 1/63 (1.59%)  1
Gastrooesophageal reflux disease  1  2/65 (3.08%)  2 1/63 (1.59%)  1
Nausea  1  2/65 (3.08%)  2 0/63 (0.00%)  0
General disorders     
Pyrexia  1  1/65 (1.54%)  1 3/63 (4.76%)  3
Systemic inflammatory response syndrome  1  1/65 (1.54%)  1 0/63 (0.00%)  0
Chest discomfort  1  0/65 (0.00%)  0 1/63 (1.59%)  1
Infections and infestations     
Infective pulmonary exacerbation  1  13/65 (20.00%)  13 17/63 (26.98%)  17
Nasopharyngitis  1  3/65 (4.62%)  3 1/63 (1.59%)  1
Bronchitis  1  2/65 (3.08%)  2 2/63 (3.17%)  2
Respiratory tract infection  1  4/65 (6.15%)  4 5/63 (7.94%)  5
Viral infection  1  2/65 (3.08%)  2 0/63 (0.00%)  0
Investigations     
Alanine aminotransferase increased  1  2/65 (3.08%)  2 0/63 (0.00%)  0
Metabolism and nutrition disorders     
Abnormal loss of weight  1  4/65 (6.15%)  4 1/63 (1.59%)  1
Nervous system disorders     
Headache  1  4/65 (6.15%)  4 3/63 (4.76%)  3
Respiratory, thoracic and mediastinal disorders     
Cough  1  2/65 (3.08%)  2 6/63 (9.52%)  6
Dyspnoea  1  2/65 (3.08%)  2 0/63 (0.00%)  0
Haemoptysis  1  2/65 (3.08%)  2 2/63 (3.17%)  2
Nasal congestion  1  2/65 (3.08%)  2 3/63 (4.76%)  3
Productive cough  1  2/65 (3.08%)  2 1/63 (1.59%)  1
1
Term from vocabulary, MedDRA (19.0)
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
 
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Monica Gangal
Organization: Anthera Pharmaceuticals
Phone: 510-856-5600
EMail: info@anthera.com
Layout table for additonal information
Responsible Party: Anthera Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02279498     History of Changes
Other Study ID Numbers: AN-EPI3331
First Submitted: October 28, 2014
First Posted: October 31, 2014
Results First Submitted: April 10, 2018
Results First Posted: August 14, 2018
Last Update Posted: August 14, 2018