Trial Outcomes & Findings for SOLUTION: Study of Oral Liprotamase Unit-Matched Therapy Of Non-Porcine Origin in Patients With Cystic Fibrosis (NCT NCT02279498)
NCT ID: NCT02279498
Last Updated: 2018-08-14
Results Overview
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
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
128 participants
Primary outcome timeframe
Baseline, 7 weeks
Results posted on
2018-08-14
Participant Flow
Participant milestones
| Measure |
Liprotamase
Individually-optimized dose to be administered orally
Liprotamase: oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement
|
Porcine (Pig) PERT
Individually-optimized dose to be administered orally
porcine (pig) PERT: oral, enterically-coated, pancreatic replacement enzymes prepared from a porcine source
|
|---|---|---|
|
Overall Study
STARTED
|
65
|
63
|
|
Overall Study
COMPLETED
|
47
|
59
|
|
Overall Study
NOT COMPLETED
|
18
|
4
|
Reasons for withdrawal
| Measure |
Liprotamase
Individually-optimized dose to be administered orally
Liprotamase: oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement
|
Porcine (Pig) PERT
Individually-optimized dose to be administered orally
porcine (pig) PERT: oral, enterically-coated, pancreatic replacement enzymes prepared from a porcine source
|
|---|---|---|
|
Overall Study
Adverse Event
|
3
|
0
|
|
Overall Study
Lack of Efficacy
|
4
|
0
|
|
Overall Study
Lost to Follow-up
|
0
|
1
|
|
Overall Study
Withdrawal by Subject
|
9
|
2
|
|
Overall Study
Undefined
|
2
|
1
|
Baseline Characteristics
SOLUTION: Study of Oral Liprotamase Unit-Matched Therapy Of Non-Porcine Origin in Patients With Cystic Fibrosis
Baseline characteristics by cohort
| Measure |
Liprotamase
n=65 Participants
Individually-optimized dose to be administered orally
Liprotamase: oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement
|
Porcine (Pig) PERT
n=63 Participants
Individually-optimized dose to be administered orally
porcine (pig) PERT: oral, enterically-coated, pancreatic replacement enzymes prepared from a porcine source
|
Total
n=128 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
22.5 years
STANDARD_DEVIATION 8.54 • n=5 Participants
|
21.0 years
STANDARD_DEVIATION 8.95 • n=7 Participants
|
21.8 years
STANDARD_DEVIATION 8.74 • n=5 Participants
|
|
Sex: Female, Male
Female
|
30 Participants
n=5 Participants
|
31 Participants
n=7 Participants
|
61 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
35 Participants
n=5 Participants
|
32 Participants
n=7 Participants
|
67 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
63 Participants
n=5 Participants
|
61 Participants
n=7 Participants
|
124 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Coefficient of fat absorption (CFA)
|
88.7 fat absorbed as percent of fat ingested
n=5 Participants
|
89.4 fat absorbed as percent of fat ingested
n=7 Participants
|
89.1 fat absorbed as percent of fat ingested
n=5 Participants
|
|
Coefficient of nitrogen absorption (CNA)
|
96.9 % of nitrogen ingested
n=5 Participants
|
97.3 % of nitrogen ingested
n=7 Participants
|
97.1 % of nitrogen ingested
n=5 Participants
|
|
pre-randomization enzyme dose
|
6299 lipase units/kg/day
n=5 Participants
|
6264 lipase units/kg/day
n=7 Participants
|
6282 lipase units/kg/day
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline, 7 weeksPopulation: 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.
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
Outcome measures
| Measure |
Treatment Difference
n=128 Participants
analysed as LS mean difference in change in CFA from baseline for Liprotamase - Porcine enzymes
|
Porcine (Pig) PERT
Individually-optimized dose to be administered orally
porcine (pig) PERT: oral, enterically-coated, pancreatic replacement enzymes prepared from a porcine source
|
|---|---|---|
|
Treatment Difference in Coefficient of Fat Absorption (CFA) Change From Baseline
|
-11.852 percent change from baseline
Interval -16.0 to -7.7
|
—
|
SECONDARY outcome
Timeframe: Baseline, 7 weeksPopulation: Analysis population evaluates observed case data without multiple imputation for missing values
Change from baseline in coefficient of fat absorption
Outcome measures
| Measure |
Treatment Difference
n=53 Participants
analysed as LS mean difference in change in CFA from baseline for Liprotamase - Porcine enzymes
|
Porcine (Pig) PERT
n=62 Participants
Individually-optimized dose to be administered orally
porcine (pig) PERT: oral, enterically-coated, pancreatic replacement enzymes prepared from a porcine source
|
|---|---|---|
|
Coefficient of Fat Absorption (CFA)
|
76.5 fat absorbed as % of fat ingested
Standard Deviation 16.13
|
89.5 fat absorbed as % of fat ingested
Standard Deviation 5.84
|
SECONDARY outcome
Timeframe: Baseline, 7 weeksPopulation: Analysis population evaluates observed case data without multiple imputation for missing values
Change from baseline in coefficient of nitrogen absorption
Outcome measures
| Measure |
Treatment Difference
n=53 Participants
analysed as LS mean difference in change in CFA from baseline for Liprotamase - Porcine enzymes
|
Porcine (Pig) PERT
n=62 Participants
Individually-optimized dose to be administered orally
porcine (pig) PERT: oral, enterically-coated, pancreatic replacement enzymes prepared from a porcine source
|
|---|---|---|
|
Coefficient of Nitrogen Absorption (CNA)
|
95.8 percent of nitrogen ingested
Standard Deviation 2.46
|
97.5 percent of nitrogen ingested
Standard Deviation 1.21
|
Adverse Events
Liprotamase
Serious events: 7 serious events
Other events: 41 other events
Deaths: 0 deaths
Porcine (Pig) PERT
Serious events: 6 serious events
Other events: 38 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Liprotamase
n=65 participants at risk
Individually-optimized dose to be administered orally
Liprotamase: oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement
|
Porcine (Pig) PERT
n=63 participants at risk
Individually-optimized dose to be administered orally
porcine (pig) PERT: oral, enterically-coated, pancreatic replacement enzymes prepared from a porcine source
|
|---|---|---|
|
Infections and infestations
Infective pulmonary exacerbation of cystic fibrosis
|
4.6%
3/65 • Number of events 3 • 6 months
|
7.9%
5/63 • Number of events 5 • 6 months
|
|
Gastrointestinal disorders
Constipation
|
1.5%
1/65 • Number of events 1 • 6 months
|
0.00%
0/63 • 6 months
|
|
Gastrointestinal disorders
Distal intestinal obstruction syndrome
|
1.5%
1/65 • Number of events 1 • 6 months
|
0.00%
0/63 • 6 months
|
|
General disorders
Systemic inflammatory response syndrome
|
1.5%
1/65 • Number of events 1 • 6 months
|
0.00%
0/63 • 6 months
|
|
Investigations
Clostridium test positive
|
1.5%
1/65 • Number of events 1 • 6 months
|
0.00%
0/63 • 6 months
|
|
Renal and urinary disorders
Hydronephrosis
|
1.5%
1/65 • Number of events 1 • 6 months
|
0.00%
0/63 • 6 months
|
|
Injury, poisoning and procedural complications
Tibia fracture
|
0.00%
0/65 • 6 months
|
1.6%
1/63 • Number of events 1 • 6 months
|
Other adverse events
| Measure |
Liprotamase
n=65 participants at risk
Individually-optimized dose to be administered orally
Liprotamase: oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement
|
Porcine (Pig) PERT
n=63 participants at risk
Individually-optimized dose to be administered orally
porcine (pig) PERT: oral, enterically-coated, pancreatic replacement enzymes prepared from a porcine source
|
|---|---|---|
|
Infections and infestations
Infective pulmonary exacerbation
|
20.0%
13/65 • Number of events 13 • 6 months
|
27.0%
17/63 • Number of events 17 • 6 months
|
|
Investigations
Alanine aminotransferase increased
|
3.1%
2/65 • Number of events 2 • 6 months
|
0.00%
0/63 • 6 months
|
|
General disorders
Pyrexia
|
1.5%
1/65 • Number of events 1 • 6 months
|
4.8%
3/63 • Number of events 3 • 6 months
|
|
General disorders
Systemic inflammatory response syndrome
|
1.5%
1/65 • Number of events 1 • 6 months
|
0.00%
0/63 • 6 months
|
|
General disorders
Chest discomfort
|
0.00%
0/65 • 6 months
|
1.6%
1/63 • Number of events 1 • 6 months
|
|
Infections and infestations
Nasopharyngitis
|
4.6%
3/65 • Number of events 3 • 6 months
|
1.6%
1/63 • Number of events 1 • 6 months
|
|
Infections and infestations
Bronchitis
|
3.1%
2/65 • Number of events 2 • 6 months
|
3.2%
2/63 • Number of events 2 • 6 months
|
|
Infections and infestations
Respiratory tract infection
|
6.2%
4/65 • Number of events 4 • 6 months
|
7.9%
5/63 • Number of events 5 • 6 months
|
|
Infections and infestations
Viral infection
|
3.1%
2/65 • Number of events 2 • 6 months
|
0.00%
0/63 • 6 months
|
|
Nervous system disorders
Headache
|
6.2%
4/65 • Number of events 4 • 6 months
|
4.8%
3/63 • Number of events 3 • 6 months
|
|
Gastrointestinal disorders
Constipation
|
3.1%
2/65 • Number of events 2 • 6 months
|
1.6%
1/63 • Number of events 1 • 6 months
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
3.1%
2/65 • Number of events 2 • 6 months
|
1.6%
1/63 • Number of events 1 • 6 months
|
|
Gastrointestinal disorders
Nausea
|
3.1%
2/65 • Number of events 2 • 6 months
|
0.00%
0/63 • 6 months
|
|
Metabolism and nutrition disorders
Abnormal loss of weight
|
6.2%
4/65 • Number of events 4 • 6 months
|
1.6%
1/63 • Number of events 1 • 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
3.1%
2/65 • Number of events 2 • 6 months
|
9.5%
6/63 • Number of events 6 • 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
3.1%
2/65 • Number of events 2 • 6 months
|
0.00%
0/63 • 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Haemoptysis
|
3.1%
2/65 • Number of events 2 • 6 months
|
3.2%
2/63 • Number of events 2 • 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
3.1%
2/65 • Number of events 2 • 6 months
|
4.8%
3/63 • Number of events 3 • 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
3.1%
2/65 • Number of events 2 • 6 months
|
1.6%
1/63 • Number of events 1 • 6 months
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: OTHER