Trial Outcomes & Findings for Study Investigating a Delayed-Release Pancrelipase in Patients With Pancreatic Exocrine Insufficiency Due to Cystic Fibrosis (NCT NCT00775528)

Last Updated: 2010-11-08

Results Overview

Treatment Emergent Adverse Events are defined as adverse events started at or after the first administration of study drug and include those events started prior to the first administration but which worsened after the first intake.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

19 participants

Primary outcome timeframe

10 Days

Results posted on

2010-11-08

Participant Flow

Subjects were recruited in 9 centers in US between April 2009 and June 2009. During the period of screening (Days -21 to Days -7), subjects were evaluated for eligibility. They underwent a short period of up to 7 days on their usual pancreatic enzyme supplementation before receiving the study treatment.

Nineteen subjects were consented and 18 subjects were allocated to pancrelipase delayed release capsule. One subject did not meet the fecal elastase requirement and did not receive study medication.

Participant milestones

Participant milestones
Measure	Pancrelipase Delayed-Release Capsules Target dose of 8000 lipase units/kg/day. This dose was administered in divided doses with meals/snacks.
Overall Study STARTED	19
Overall Study COMPLETED	18
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Pancrelipase Delayed-Release Capsules Target dose of 8000 lipase units/kg/day. This dose was administered in divided doses with meals/snacks.
Overall Study Protocol Violation	1

Baseline Characteristics

Study Investigating a Delayed-Release Pancrelipase in Patients With Pancreatic Exocrine Insufficiency Due to Cystic Fibrosis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Pancrelipase Delayed-Release Capsules n=18 Participants Target dose of 8000 lipase units/kg/day. This dose was administered in divided doses with meals/snacks.
Age Continuous	27.3 months STANDARD_DEVIATION 21.9 • n=5 Participants
Sex: Female, Male Female	5 Participants n=5 Participants
Sex: Female, Male Male	13 Participants n=5 Participants
Region of Enrollment United States	18 participants n=5 Participants

PRIMARY outcome

Timeframe: 10 Days

Outcome measures

Outcome measures
Measure	Pancrelipase Delayed-Release Capsules n=18 Participants Target dose of 8000 lipase units/kg/day. This dose was administered in divided doses with meals/snacks.
Number of Patients With at Least One Treatment Emergent Adverse Event (TEAE)	9 Participants

SECONDARY outcome

Timeframe: Last 3 days in a 10-day treatment period

Population: The efficacy results are presented on the Full Analysis Set meaning patients having taken the study drug and with Post-baseline efficacy data.

The stool fat content was calculated as percent fat of dry solid weight per bowel movement. Stool fat per patient was derived as mean over three bowel movements sampled (i.e. one sample per day).

Outcome measures

Outcome measures
Measure	Pancrelipase Delayed-Release Capsules n=18 Participants Target dose of 8000 lipase units/kg/day. This dose was administered in divided doses with meals/snacks.
Stool Fat (% Fat)	28.11 Percentage of fat Standard Deviation 9.86

SECONDARY outcome

Timeframe: Last 3 days in a 10-day treatment period

Population: The efficacy results are presented on the Full Analysis Set meaning patients having taken the study drug and with Post-baseline efficacy data.

The mean daily fat intake was determined as the average of daily fat intake over a 3-day period.

Outcome measures

Outcome measures
Measure	Pancrelipase Delayed-Release Capsules n=18 Participants Target dose of 8000 lipase units/kg/day. This dose was administered in divided doses with meals/snacks.
Fat Intake (g)	46.61 Grams Standard Deviation 22.34

SECONDARY outcome

Timeframe: Last 3 days in a 10-day treatment period

Population: The efficacy results are presented on the Full Analysis Set meaning patients having taken the study drug and with Post-baseline efficacy data.

The total calorie intake was determined as the average of total calorie of daily food intake intake over a 3-day period.

Outcome measures

Outcome measures
Measure	Pancrelipase Delayed-Release Capsules n=18 Participants Target dose of 8000 lipase units/kg/day. This dose was administered in divided doses with meals/snacks.
Total Calorie Intake (kcal)	1239.71 Kcal Standard Deviation 605.78

Adverse Events

Pancrelipase Delayed-Release Capsules

Serious events: 0 serious events

Other events: 9 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Pancrelipase Delayed-Release Capsules n=18 participants at risk Target dose of 8000 lipase units/kg/day. This dose was administered in divided doses with meals/snacks.
Gastrointestinal disorders Diarrhoea	5.6% 1/18 • The adverse events presented were collected from start of drug treatment to the end of the treatment period.
Gastrointestinal disorders Vomiting	5.6% 1/18 • The adverse events presented were collected from start of drug treatment to the end of the treatment period.
General disorders Pyrexia	5.6% 1/18 • The adverse events presented were collected from start of drug treatment to the end of the treatment period.
General disorders Irritability	5.6% 1/18 • The adverse events presented were collected from start of drug treatment to the end of the treatment period.
Infections and infestations Skin candida	5.6% 1/18 • The adverse events presented were collected from start of drug treatment to the end of the treatment period.
Infections and infestations Otitis media	16.7% 3/18 • The adverse events presented were collected from start of drug treatment to the end of the treatment period.
Infections and infestations Otitis media accute	5.6% 1/18 • The adverse events presented were collected from start of drug treatment to the end of the treatment period.
Infections and infestations Bronchitis	5.6% 1/18 • The adverse events presented were collected from start of drug treatment to the end of the treatment period.
Metabolism and nutrition disorders Decreased appetite	5.6% 1/18 • The adverse events presented were collected from start of drug treatment to the end of the treatment period.
Respiratory, thoracic and mediastinal disorders Cough	11.1% 2/18 • The adverse events presented were collected from start of drug treatment to the end of the treatment period.
Respiratory, thoracic and mediastinal disorders Rhinorrhoea	5.6% 1/18 • The adverse events presented were collected from start of drug treatment to the end of the treatment period.
Skin and subcutaneous tissue disorders Dermatitis diaper	5.6% 1/18 • The adverse events presented were collected from start of drug treatment to the end of the treatment period.

Additional Information

Sven Voet - Global Communication

Solvay Pharmaceuticals

Phone: +32 (0)2 509 69 77

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee At 60 days prior to submitting or presenting a manuscript or other materials relating to the Study to a publisher, reviewer, or other outside persons, the Site shall provide to Sponsor a copy and allow Sponsor 60 days to review and comment.
Publication restrictions are in place

Restriction type: OTHER