Trial Outcomes & Findings for Study Investigating a Delayed-Release Pancrelipase in Patients With Pancreatic Exocrine Insufficiency (PEI) Due to Cystic Fibrosis (CF) (NCT NCT00690820)

NCT ID: NCT00690820

Last Updated: 2010-06-02

Results Overview

This coefficient is calculated from fat intake and fat excretion : 100\*\[fat intake-fat excretion\]/fat intake. Stools were collected on 3 days during the 5 days treatment period. Higher values indicate a better response.

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 17 participants
• Primary outcome timeframe: 5 days
• Results posted on: 2010-06-02

Participant Flow

Subjects were recruited in 10 centers in US between June 2008 and October 2008. Before the randomization, subjects were evaluated for eligibility. They underwent a short period of up to 14 days on their usual pancreatic enzyme supplementation.

Twenty three subjects had given their consent and 17 subjects were randomly allocated to pancrelipase/placebo or placebo/pancrelipase. One subject did not complete the first period of the treatment (consent withdrawal).

Participant milestones

Measure	Placebo/Pancrelipase Placebo period followed by Pancrelipase delayed release 12000 units period	Pancrelipase/Placebo Pancrelipase delayed release 12000 units period followed by Placebo period
Period 1 STARTED	8	9
Period 1 COMPLETED	8	8
Period 1 NOT COMPLETED	0	1
Period 2 STARTED	8	8
Period 2 COMPLETED	8	8
Period 2 NOT COMPLETED	0	0

Reasons for withdrawal

Measure	Placebo/Pancrelipase Placebo period followed by Pancrelipase delayed release 12000 units period	Pancrelipase/Placebo Pancrelipase delayed release 12000 units period followed by Placebo period
Period 1 Withdrawal by Subject	0	1

Baseline Characteristics

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

Baseline characteristics by cohort

Measure	Placebo/Pancrelipase n=8 Participants Placebo period followed by Pancrelipase delayed release 12000 units period	Pancrelipase/Placebo n=9 Participants Pancrelipase delayed release 12000 units period followed by Placebo period	Total n=17 Participants Total of all reporting groups
Age Continuous	8.9 years STANDARD_DEVIATION 1.1 • n=93 Participants	8.7 years STANDARD_DEVIATION 1.4 • n=4 Participants	8.8 years STANDARD_DEVIATION 1.3 • n=27 Participants
Sex: Female, Male Female	1 Participants n=93 Participants	4 Participants n=4 Participants	5 Participants n=27 Participants
Sex: Female, Male Male	7 Participants n=93 Participants	5 Participants n=4 Participants	12 Participants n=27 Participants
Region of Enrollment United States	8 participants n=93 Participants	9 participants n=4 Participants	17 participants n=27 Participants

PRIMARY outcome

Timeframe: 5 days

Population: The analysis was done on the Full Analysis Sample defined as the randomized subjects with at least one post-baseline efficacy measurement.

This coefficient is calculated from fat intake and fat excretion : 100*[fat intake-fat excretion]/fat intake. Stools were collected on 3 days during the 5 days treatment period. Higher values indicate a better response.

Outcome measures

Measure	Placebo n=16 Participants Placebo treatment	Pancrelipase n=16 Participants Pancrelipase delayed release 12000 units treatment
Coefficient of Fat Absorption (%)	47.41 Percentage Standard Deviation 16.84	82.81 Percentage Standard Deviation 8.29

SECONDARY outcome

Timeframe: 5 days

Population: The analysis was done on the Full Analysis Sample defined as the randomized subjects with at least one post-baseline efficacy measurement.

This coefficient is calculated from nitrogen intake and nitrogen excretion : 100*[nitrogen intake-nitrogen excretion]/nitrogen intake. Stools were collected on 3 days during the 5 days treatment period. Higher values indicate a better response.

Outcome measures

Measure	Placebo n=16 Participants Placebo treatment	Pancrelipase n=16 Participants Pancrelipase delayed release 12000 units treatment
Coefficient of Nitrogen Absorption (%)	44.98 Percentage Standard Deviation 20.47	80.33 Percentage Standard Deviation 7.92

SECONDARY outcome

Timeframe: 5 days

Population: The analysis was done on the Full Analysis Sample defined as the randomized subjects with at least one post-baseline efficacy measurement.

Total amount of fat excreted during the stool collection period. Stools were collected on 3 days during the 5 days treatment period. Lower values indicate a better response.

Outcome measures

Measure	Placebo n=16 Participants Placebo treatment	Pancrelipase n=16 Participants Pancrelipase delayed release 12000 units treatment
Total Fat Excretion (Grams)	182.9 Grams Standard Deviation 68.0	58.1 Grams Standard Deviation 27.5

SECONDARY outcome

Timeframe: 5 days

Population: The analysis was done on the Full Analysis Sample defined as the randomized subjects with at least one post-baseline efficacy measurement.

Total weight of the stools collected during the stool collection period. Stools were collected on 3 days during the 5 days treatment period. Lower values indicate a better response.

Outcome measures

Measure	Placebo n=16 Participants Placebo treatment	Pancrelipase n=16 Participants Pancrelipase delayed release 12000 units treatment
Total Stool Weight (Grams)	436.5 Grams Standard Deviation 158.4	161.4 Grams Standard Deviation 57.5

SECONDARY outcome

Timeframe: 5 days

Population: The analysis was done on the Full Analysis Sample defined as the randomized subjects with at least one post-baseline efficacy measurement.

Stool frequency is the average of the daily number of stools recorded during the treatment period. Lower values indicate a better response.

Outcome measures

Measure	Placebo n=16 Participants Placebo treatment	Pancrelipase n=16 Participants Pancrelipase delayed release 12000 units treatment
Stool Frequency	3.46 Number per day Standard Deviation 1.06	1.88 Number per day Standard Deviation 0.82

SECONDARY outcome

Timeframe: 5 days

Population: The analysis was done on the Full Analysis Sample defined as the randomized subjects with at least one post-baseline efficacy measurement.

The percentage of days with no flatulence is calculated from the diary during the treatment period: 100*(number of days with no flatulence/number of days recorded in diary). Higher values indicate a better response.

Outcome measures

Measure	Placebo n=16 Participants Placebo treatment	Pancrelipase n=16 Participants Pancrelipase delayed release 12000 units treatment
Percentage of Days With no Flatulence.	36.3 Percentage of days Standard Deviation 36.9	45.0 Percentage of days Standard Deviation 42.1

SECONDARY outcome

Timeframe: 5 days

Population: The analysis was done on the Full Analysis Sample defined as the randomized subjects with at least one post-baseline efficacy measurement.

The percentage of days with formed/normal stools is calculated from the diary during the treatment period: 100*(number of days with formed/normal stools/number of days with any stool). Higher values indicate a better response.

Outcome measures

Measure	Placebo n=16 Participants Placebo treatment	Pancrelipase n=16 Participants Pancrelipase delayed release 12000 units treatment
Percentage of Days With Formed/Normal Stools.	38.9 Percentage of days Standard Deviation 33.0	77.7 Percentage of days Standard Deviation 27.0

SECONDARY outcome

Timeframe: 5 days

Population: The analysis was done on the Full Analysis Sample defined as the randomized subjects with at least one post-baseline efficacy measurement.

The percentage of days with no abdominal pain is calculated from the diary during the treatment period: 100*(number of days with no abdominal pain / number of days recorded in diary). Higher values indicate a better response.

Outcome measures

Measure	Placebo n=16 Participants Placebo treatment	Pancrelipase n=16 Participants Pancrelipase delayed release 12000 units treatment
Percentage of Days With no Abdominal Pain.	65.2 Percentage of days Standard Deviation 28.9	85.3 Percentage of days Standard Deviation 21.1

Adverse Events

Placebo

Serious events: 0 serious events

Other events: 9 other events

Deaths: 0 deaths

Pancrelipase

Serious events: 0 serious events

Other events: 5 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Placebo n=16 participants at risk Placebo treatment	Pancrelipase n=17 participants at risk Pancrelipase delayed release 12000 units treatment
Gastrointestinal disorders Rectal Discharge	6.2% 1/16 • The adverse events were collected from Screening to the end of the second period of the cross-over trial. A safety follow-up call was performed 5-7 days after the last visit to the clinic. Only Treatment Emergent Adverse events are presented.	0.00% 0/17 • The adverse events were collected from Screening to the end of the second period of the cross-over trial. A safety follow-up call was performed 5-7 days after the last visit to the clinic. Only Treatment Emergent Adverse events are presented.
Gastrointestinal disorders Diarrhoea	12.5% 2/16 • The adverse events were collected from Screening to the end of the second period of the cross-over trial. A safety follow-up call was performed 5-7 days after the last visit to the clinic. Only Treatment Emergent Adverse events are presented.	0.00% 0/17 • The adverse events were collected from Screening to the end of the second period of the cross-over trial. A safety follow-up call was performed 5-7 days after the last visit to the clinic. Only Treatment Emergent Adverse events are presented.
Gastrointestinal disorders Faecal Volume Increased	6.2% 1/16 • The adverse events were collected from Screening to the end of the second period of the cross-over trial. A safety follow-up call was performed 5-7 days after the last visit to the clinic. Only Treatment Emergent Adverse events are presented.	0.00% 0/17 • The adverse events were collected from Screening to the end of the second period of the cross-over trial. A safety follow-up call was performed 5-7 days after the last visit to the clinic. Only Treatment Emergent Adverse events are presented.
Gastrointestinal disorders Flatulence	12.5% 2/16 • The adverse events were collected from Screening to the end of the second period of the cross-over trial. A safety follow-up call was performed 5-7 days after the last visit to the clinic. Only Treatment Emergent Adverse events are presented.	0.00% 0/17 • The adverse events were collected from Screening to the end of the second period of the cross-over trial. A safety follow-up call was performed 5-7 days after the last visit to the clinic. Only Treatment Emergent Adverse events are presented.
Gastrointestinal disorders Abdominal Pain	25.0% 4/16 • The adverse events were collected from Screening to the end of the second period of the cross-over trial. A safety follow-up call was performed 5-7 days after the last visit to the clinic. Only Treatment Emergent Adverse events are presented.	5.9% 1/17 • The adverse events were collected from Screening to the end of the second period of the cross-over trial. A safety follow-up call was performed 5-7 days after the last visit to the clinic. Only Treatment Emergent Adverse events are presented.
Gastrointestinal disorders Frequent Bowel Movements	6.2% 1/16 • The adverse events were collected from Screening to the end of the second period of the cross-over trial. A safety follow-up call was performed 5-7 days after the last visit to the clinic. Only Treatment Emergent Adverse events are presented.	0.00% 0/17 • The adverse events were collected from Screening to the end of the second period of the cross-over trial. A safety follow-up call was performed 5-7 days after the last visit to the clinic. Only Treatment Emergent Adverse events are presented.
Gastrointestinal disorders Nausea	6.2% 1/16 • The adverse events were collected from Screening to the end of the second period of the cross-over trial. A safety follow-up call was performed 5-7 days after the last visit to the clinic. Only Treatment Emergent Adverse events are presented.	0.00% 0/17 • The adverse events were collected from Screening to the end of the second period of the cross-over trial. A safety follow-up call was performed 5-7 days after the last visit to the clinic. Only Treatment Emergent Adverse events are presented.
Gastrointestinal disorders Vomiting	0.00% 0/16 • The adverse events were collected from Screening to the end of the second period of the cross-over trial. A safety follow-up call was performed 5-7 days after the last visit to the clinic. Only Treatment Emergent Adverse events are presented.	11.8% 2/17 • The adverse events were collected from Screening to the end of the second period of the cross-over trial. A safety follow-up call was performed 5-7 days after the last visit to the clinic. Only Treatment Emergent Adverse events are presented.
Investigations Blood glucose increased	6.2% 1/16 • The adverse events were collected from Screening to the end of the second period of the cross-over trial. A safety follow-up call was performed 5-7 days after the last visit to the clinic. Only Treatment Emergent Adverse events are presented.	5.9% 1/17 • The adverse events were collected from Screening to the end of the second period of the cross-over trial. A safety follow-up call was performed 5-7 days after the last visit to the clinic. Only Treatment Emergent Adverse events are presented.
Investigations Weight Decrease	6.2% 1/16 • The adverse events were collected from Screening to the end of the second period of the cross-over trial. A safety follow-up call was performed 5-7 days after the last visit to the clinic. Only Treatment Emergent Adverse events are presented.	0.00% 0/17 • The adverse events were collected from Screening to the end of the second period of the cross-over trial. A safety follow-up call was performed 5-7 days after the last visit to the clinic. Only Treatment Emergent Adverse events are presented.
Nervous system disorders Headache	0.00% 0/16 • The adverse events were collected from Screening to the end of the second period of the cross-over trial. A safety follow-up call was performed 5-7 days after the last visit to the clinic. Only Treatment Emergent Adverse events are presented.	11.8% 2/17 • The adverse events were collected from Screening to the end of the second period of the cross-over trial. A safety follow-up call was performed 5-7 days after the last visit to the clinic. Only Treatment Emergent Adverse events are presented.
Nervous system disorders Dizziness	6.2% 1/16 • The adverse events were collected from Screening to the end of the second period of the cross-over trial. A safety follow-up call was performed 5-7 days after the last visit to the clinic. Only Treatment Emergent Adverse events are presented.	0.00% 0/17 • The adverse events were collected from Screening to the end of the second period of the cross-over trial. A safety follow-up call was performed 5-7 days after the last visit to the clinic. Only Treatment Emergent Adverse events are presented.
Skin and subcutaneous tissue disorders Cold sweat	6.2% 1/16 • The adverse events were collected from Screening to the end of the second period of the cross-over trial. A safety follow-up call was performed 5-7 days after the last visit to the clinic. Only Treatment Emergent Adverse events are presented.	0.00% 0/17 • The adverse events were collected from Screening to the end of the second period of the cross-over trial. A safety follow-up call was performed 5-7 days after the last visit to the clinic. Only Treatment Emergent Adverse events are presented.
Skin and subcutaneous tissue disorders Rash	6.2% 1/16 • The adverse events were collected from Screening to the end of the second period of the cross-over trial. A safety follow-up call was performed 5-7 days after the last visit to the clinic. Only Treatment Emergent Adverse events are presented.	0.00% 0/17 • The adverse events were collected from Screening to the end of the second period of the cross-over trial. A safety follow-up call was performed 5-7 days after the last visit to the clinic. Only Treatment Emergent Adverse events are presented.
Skin and subcutaneous tissue disorders Rash Macular	6.2% 1/16 • The adverse events were collected from Screening to the end of the second period of the cross-over trial. A safety follow-up call was performed 5-7 days after the last visit to the clinic. Only Treatment Emergent Adverse events are presented.	0.00% 0/17 • The adverse events were collected from Screening to the end of the second period of the cross-over trial. A safety follow-up call was performed 5-7 days after the last visit to the clinic. Only Treatment Emergent Adverse events are presented.
Skin and subcutaneous tissue disorders Urticaria	0.00% 0/16 • The adverse events were collected from Screening to the end of the second period of the cross-over trial. A safety follow-up call was performed 5-7 days after the last visit to the clinic. Only Treatment Emergent Adverse events are presented.	5.9% 1/17 • The adverse events were collected from Screening to the end of the second period of the cross-over trial. A safety follow-up call was performed 5-7 days after the last visit to the clinic. Only Treatment Emergent Adverse events are presented.

Additional Information

Sven Voet - Global Communication

Solvay Pharmaceuticals

Phone: +32(0)2 509 69 77

Email: sven.voet@solvay.com

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 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