Trial Outcomes & Findings for Study Investigating a Delayed-Release Pancrelipase in Patients With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis (NCT NCT00510484)

NCT ID: NCT00510484

Last Updated: 2010-06-03

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: 35 participants
• Primary outcome timeframe: 5 days
• Results posted on: 2010-06-03

Participant Flow

Subjects were recruited in 10 centers in US between November 2007 and January 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. Subjects were to take 4000 lipase units/g fat intake during both cross-over periods.

Two subjects prematurely withdrew before the randomization (1 withdrew consent and 1 for protocol violation). 32 subjects were randomly allocated to pancrelipase/placebo or placebo/pancrelipase. 1 subject did not complete the second period of the treatment. 1 subject was randomized twice and only the second randomization was used for analyses.

Participant milestones

Measure	Placebo/Pancrelipase	Pancrelipase/Placebo
Period 1 STARTED	16	16
Period 1 COMPLETED	16	16
Period 1 NOT COMPLETED	0	0
Wash-out Period STARTED	16	16
Wash-out Period COMPLETED	16	15
Wash-out Period NOT COMPLETED	0	1
Period 2 STARTED	16	15
Period 2 COMPLETED	16	15
Period 2 NOT COMPLETED	0	0

Reasons for withdrawal

Measure	Placebo/Pancrelipase	Pancrelipase/Placebo
Wash-out Period Adverse Event	0	1

Baseline Characteristics

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

Baseline characteristics by cohort

Measure	Placebo/Pancrelipase n=16 Participants	Pancrelipase/Placebo n=16 Participants	Total n=32 Participants Total of all reporting groups
Age Continuous	22.2 years STANDARD_DEVIATION 7.8 • n=5 Participants	22.8 years STANDARD_DEVIATION 6.5 • n=7 Participants	22.5 years STANDARD_DEVIATION 7.1 • n=5 Participants
Sex: Female, Male Female	4 Participants n=5 Participants	7 Participants n=7 Participants	11 Participants n=5 Participants
Sex: Female, Male Male	12 Participants n=5 Participants	9 Participants n=7 Participants	21 Participants n=5 Participants
Region of Enrollment United States	16 participants n=5 Participants	16 participants n=7 Participants	32 participants n=5 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	Pancrelipase n=31 Participants	Placebo n=31 Participants
Coefficient of Fat Absorption (%)	88.62 Percentage Standard Deviation 6.62	49.78 Percentage Standard Deviation 18.33

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	Pancrelipase n=31 Participants	Placebo n=31 Participants
Coefficient of Nitrogen Absorption (%)	85.06 Percentage Standard Deviation 6.38	49.99 Percentage Standard Deviation 17.14

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	Pancrelipase n=31 Participants	Placebo n=31 Participants
Total Fat Excretion (Grams)	57.7 Grams Standard Deviation 46.1	243.2 Grams Standard Deviation 112.7

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	Pancrelipase n=31 Participants	Placebo n=31 Participants
Total Stool Weight (Grams)	630.1 Grams Standard Deviation 306.9	1582.5 Grams Standard Deviation 664.5

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	Pancrelipase n=32 Participants	Placebo n=31 Participants
Stool Frequency	1.75 Number per day Standard Deviation 0.81	2.88 Number per day Standard Deviation 1.18

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	Pancrelipase n=32 Participants	Placebo n=31 Participants
Percentage of Days With no Flatulence.	41.6 Percentage of days Standard Deviation 41.9	23.1 Percentage of days Standard Deviation 35.1

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 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	Pancrelipase n=32 Participants	Placebo n=31 Participants
Percentage of Days With no Abdominal Pain.	90.3 Percentage of days Standard Deviation 16.8	59.1 Percentage of days Standard Deviation 43.2

OTHER_PRE_SPECIFIED 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	Pancrelipase n=32 Participants	Placebo n=31 Participants
Percentage of Days With Formed/Normal Stools.	75.0 Percentage of days Standard Deviation 30.0	23.9 Percentage of days Standard Deviation 27.8

Adverse Events

Pancrelipase

Serious events: 0 serious events

Other events: 14 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 20 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Pancrelipase n=32 participants at risk	Placebo n=31 participants at risk
Gastrointestinal disorders Faeces abnormal	3.1% 1/32	19.4% 6/31
Gastrointestinal disorders Flatulence, Bloating and Distension	9.4% 3/32	25.8% 8/31
Gastrointestinal disorders Gastrointestinal and abdominal pain (Excl oral and throat)	9.4% 3/32	32.3% 10/31
Investigations Physical Examination Procedures	3.1% 1/32	6.5% 2/31
Nervous system disorders Headaches NEC	6.2% 2/32	22.6% 7/31
Nervous system disorders Neurological signs and symptoms NEC	6.2% 2/32	0.00% 0/31
Respiratory, thoracic and mediastinal disorders Coughing and Associated Symptoms	6.2% 2/32	0.00% 0/31

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 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