Trial Outcomes & Findings for A Study of the Efficacy and Tolerability of Pancrelipase Microtablet (MT) Capsules for the Treatment of Cystic Fibrosis-dependent Exocrine Pancreatic Insufficiency (NCT NCT00662675)
NCT ID: NCT00662675
Last Updated: 2014-05-09
Results Overview
Change in the coefficient of fat absorption (percent COA-fat) from the 72-hour inpatient period in the open-label phase to the 72-hour period inpatient period in the double-blind (withdrawal) phase.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
40 participants
Primary outcome timeframe
72-hours stool collection in the open-label phase to the end of 72-hours stool collection in the double-blind withdrawal phase.
Results posted on
2014-05-09
Participant Flow
The initial (screening) dose of PANCREASE MT was based on the average dose of pancreatic enzyme replacement therapy (PERT) taken for the 3 days immediately before entry into the study in combination with a high-fat diet. This PERT was continued until all screening test results were received and the subject met all inclusion/exclusion criteria.
Participant milestones
| Measure |
Placebo
Matching placebo capsules taken by mouth per meal or snack
|
PANCREASE MT
Pancrease MT 10.5 or MT 21 capsules taken by mouth per meal or snack
|
|---|---|---|
|
Overall Study
STARTED
|
20
|
20
|
|
Overall Study
COMPLETED
|
20
|
20
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Study of the Efficacy and Tolerability of Pancrelipase Microtablet (MT) Capsules for the Treatment of Cystic Fibrosis-dependent Exocrine Pancreatic Insufficiency
Baseline characteristics by cohort
| Measure |
Placebo
n=20 Participants
Matching placebo capsules taken by mouth per meal or snack
|
PANCREASE MT
n=20 Participants
Pancrease MT 10.5 or MT 21 capsules taken by mouth per meal or snack
|
Total
n=40 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
23.4 years
STANDARD_DEVIATION 11.58 • n=5 Participants
|
24 years
STANDARD_DEVIATION 13.44 • n=7 Participants
|
23.7 years
STANDARD_DEVIATION 12.39 • n=5 Participants
|
|
Sex: Female, Male
Female
|
7 Participants
n=5 Participants
|
11 Participants
n=7 Participants
|
18 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
13 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
22 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
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
19 Participants
n=5 Participants
|
17 Participants
n=7 Participants
|
36 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
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 72-hours stool collection in the open-label phase to the end of 72-hours stool collection in the double-blind withdrawal phase.Population: Intent-to-Treat (ITT)
Change in the coefficient of fat absorption (percent COA-fat) from the 72-hour inpatient period in the open-label phase to the 72-hour period inpatient period in the double-blind (withdrawal) phase.
Outcome measures
| Measure |
Placebo
n=20 Participants
Matching placebo capsules taken by mouth per meal or snack
|
PANCREASE MT
n=20 Participants
Pancrease MT 10.5 or MT 21 capsules taken by mouth per meal or snack
|
|---|---|---|
|
Change in the Coefficient of Fat Absorption (COA-fat Percent)
|
-34.1 percentage COA-fat
Standard Deviation 23.03
|
-1.5 percentage COA-fat
Standard Deviation 5.88
|
SECONDARY outcome
Timeframe: 72-hours stool collection in the open-label phase to the end of 72-hours stool collection in the double-blind withdrawal phase.Population: ITT
The change in percent COA-protein from the stool collection period in double-blind phase to open-label phase
Outcome measures
| Measure |
Placebo
n=20 Participants
Matching placebo capsules taken by mouth per meal or snack
|
PANCREASE MT
n=20 Participants
Pancrease MT 10.5 or MT 21 capsules taken by mouth per meal or snack
|
|---|---|---|
|
Change in Percent COA-Protein (Nitrogen)
|
-26.5 percentage COA-protein
Standard Deviation 15.3
|
1.3 percentage COA-protein
Standard Deviation 4.71
|
SECONDARY outcome
Timeframe: Entire 7 days double-blind phasePopulation: ITT
Percent of patients reporting nausea, vomiting, bloating, diarrhea, oily/greasy stools, and abdominal pain signs and symptoms reported as Adverse events during the double-blind phase.
Outcome measures
| Measure |
Placebo
n=20 Participants
Matching placebo capsules taken by mouth per meal or snack
|
PANCREASE MT
n=20 Participants
Pancrease MT 10.5 or MT 21 capsules taken by mouth per meal or snack
|
|---|---|---|
|
Percent of Patients Reporting Clinical Signs and Symptoms of Exocrine Pancreatic Insufficiency (EPI) During the Double-Blind Phase
% of subjects with Abdominal pain
|
30 Percent of participants
|
15 Percent of participants
|
|
Percent of Patients Reporting Clinical Signs and Symptoms of Exocrine Pancreatic Insufficiency (EPI) During the Double-Blind Phase
% of subjects with Bloating
|
15 Percent of participants
|
5 Percent of participants
|
|
Percent of Patients Reporting Clinical Signs and Symptoms of Exocrine Pancreatic Insufficiency (EPI) During the Double-Blind Phase
% of subjects with Diarrhea
|
20 Percent of participants
|
0 Percent of participants
|
|
Percent of Patients Reporting Clinical Signs and Symptoms of Exocrine Pancreatic Insufficiency (EPI) During the Double-Blind Phase
% of subjects with Greasy stools
|
15 Percent of participants
|
0 Percent of participants
|
|
Percent of Patients Reporting Clinical Signs and Symptoms of Exocrine Pancreatic Insufficiency (EPI) During the Double-Blind Phase
% of subjects with Vomiting
|
0 Percent of participants
|
5 Percent of participants
|
|
Percent of Patients Reporting Clinical Signs and Symptoms of Exocrine Pancreatic Insufficiency (EPI) During the Double-Blind Phase
% of subjects with at least one EPI symptoms
|
55 Percent of participants
|
20 Percent of participants
|
Adverse Events
Placebo
Serious events: 0 serious events
Other events: 12 other events
Deaths: 0 deaths
PANCREASE MT
Serious events: 0 serious events
Other events: 8 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Placebo
n=20 participants at risk
Matching placebo capsules taken by mouth per meal or snack
|
PANCREASE MT
n=20 participants at risk
Pancrease MT 10.5 or MT 21 capsules taken by mouth per meal or snack
|
|---|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
15.0%
3/20 • During the 7 days double-blind withdrawal phase
|
10.0%
2/20 • During the 7 days double-blind withdrawal phase
|
|
Gastrointestinal disorders
Abdominal pain upper
|
15.0%
3/20 • During the 7 days double-blind withdrawal phase
|
5.0%
1/20 • During the 7 days double-blind withdrawal phase
|
|
Gastrointestinal disorders
Diarrhoea
|
20.0%
4/20 • During the 7 days double-blind withdrawal phase
|
0.00%
0/20 • During the 7 days double-blind withdrawal phase
|
|
Gastrointestinal disorders
Flatulence
|
15.0%
3/20 • During the 7 days double-blind withdrawal phase
|
5.0%
1/20 • During the 7 days double-blind withdrawal phase
|
|
Gastrointestinal disorders
Abnormal faeces
|
15.0%
3/20 • During the 7 days double-blind withdrawal phase
|
0.00%
0/20 • During the 7 days double-blind withdrawal phase
|
|
Gastrointestinal disorders
Abdominal discomfort
|
5.0%
1/20 • During the 7 days double-blind withdrawal phase
|
0.00%
0/20 • During the 7 days double-blind withdrawal phase
|
|
Gastrointestinal disorders
Abdominal distension
|
5.0%
1/20 • During the 7 days double-blind withdrawal phase
|
0.00%
0/20 • During the 7 days double-blind withdrawal phase
|
|
Gastrointestinal disorders
Dyspepsia
|
0.00%
0/20 • During the 7 days double-blind withdrawal phase
|
5.0%
1/20 • During the 7 days double-blind withdrawal phase
|
|
Gastrointestinal disorders
Gastric disorder
|
0.00%
0/20 • During the 7 days double-blind withdrawal phase
|
5.0%
1/20 • During the 7 days double-blind withdrawal phase
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/20 • During the 7 days double-blind withdrawal phase
|
5.0%
1/20 • During the 7 days double-blind withdrawal phase
|
|
General disorders
Fatigue
|
10.0%
2/20 • During the 7 days double-blind withdrawal phase
|
0.00%
0/20 • During the 7 days double-blind withdrawal phase
|
|
General disorders
Asthenia
|
5.0%
1/20 • During the 7 days double-blind withdrawal phase
|
0.00%
0/20 • During the 7 days double-blind withdrawal phase
|
|
General disorders
Feeling cold
|
5.0%
1/20 • During the 7 days double-blind withdrawal phase
|
0.00%
0/20 • During the 7 days double-blind withdrawal phase
|
|
General disorders
Thirst
|
5.0%
1/20 • During the 7 days double-blind withdrawal phase
|
0.00%
0/20 • During the 7 days double-blind withdrawal phase
|
|
Infections and infestations
Influenza
|
0.00%
0/20 • During the 7 days double-blind withdrawal phase
|
5.0%
1/20 • During the 7 days double-blind withdrawal phase
|
|
Metabolism and nutrition disorders
Decreased appetite
|
5.0%
1/20 • During the 7 days double-blind withdrawal phase
|
0.00%
0/20 • During the 7 days double-blind withdrawal phase
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
5.0%
1/20 • During the 7 days double-blind withdrawal phase
|
0.00%
0/20 • During the 7 days double-blind withdrawal phase
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
5.0%
1/20 • During the 7 days double-blind withdrawal phase
|
0.00%
0/20 • During the 7 days double-blind withdrawal phase
|
|
Nervous system disorders
Headache
|
0.00%
0/20 • During the 7 days double-blind withdrawal phase
|
5.0%
1/20 • During the 7 days double-blind withdrawal phase
|
|
Psychiatric disorders
Anxiety
|
0.00%
0/20 • During the 7 days double-blind withdrawal phase
|
5.0%
1/20 • During the 7 days double-blind withdrawal phase
|
|
Reproductive system and breast disorders
Dysmenorrhoea
|
0.00%
0/20 • During the 7 days double-blind withdrawal phase
|
5.0%
1/20 • During the 7 days double-blind withdrawal phase
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory disorder
|
0.00%
0/20 • During the 7 days double-blind withdrawal phase
|
5.0%
1/20 • During the 7 days double-blind withdrawal phase
|
|
Vascular disorders
Pallor
|
5.0%
1/20 • During the 7 days double-blind withdrawal phase
|
0.00%
0/20 • During the 7 days double-blind withdrawal phase
|
Additional Information
Director, Clinical Team Leader
Johnson & Johnson Pharmaceutical Research & Development, LLC
Phone: 609-730-3158
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: LTE60