Clinical trial NCT00690820: Study Investigating a Delayed-Release Pancrelipase in Patients With Pancreatic Exocrine Insufficiency (PEI) Due to Cystic Fibrosis (CF)
Official title: A Double-blind, Randomized, Multi-center, Placebo-controlled, Cross-over Study to Assess the Efficacy and Safety of Pancrelipase Delayed Release 12,000 Unit Capsules in Subjects Aged 7 - 11 With Pancreatic Exocrine Insufficiency Due to Cystic Fibrosis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Cystic Fibrosis
Pancreatic Exocrine Insufficiency
Keywords: Cystic Fibrosis
Pancreatic Exocrine Insufficiency
Lead sponsor: Solvay Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2008-06-05
Enrollment: 17
Sex: ALL
Minimum age (years): 7
Maximum age (years): 11
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

Brief summary: This study will assess the effect of pancrelipase delayed release 12,000 unit capsules on fat and nitrogen absorption in subjects 7 - 11 with pancreatic exocrine insufficiency due to Cystic Fibrosis.


Eligibility criteria: Inclusion Criteria:

* Confirmed CF diagnosis by two positive chloride sweat tests or gene analysis
* Confirmed PEI by historical Coefficient of fat Absorption \< 70% without supplementation or current or historical fecal elastase \< 50µg/stool (within the last 12 months)
* Currently receiving treatment with a commercially available pancreatic enzyme product on a stable dose for more than 3 months
* Clinically stable condition without evidence of acute respiratory disease or any other acute condition
* Stable body weight and agrees to abstain from sexual activity

Exclusion Criteria:

* Ileus or acute abdomen
* History of fibrosing colonopathy, celiac disease, gastrectomy, Crohn´s disease and small bowel surgery other than minor resection due to meconium ileus without resulting in malabsorption syndrome
* History of distal ileal obstruction syndrome within 6 months of enrollment
* Use of an immunosuppressive drug
* Any type of malignancy involving the digestive tract in the last 5 years
* Known infection with HIV

Primary outcomes:
measure: Coefficient of Fat Absorption (%)
timeFrame: 5 days
Secondary outcomes:
measure: Coefficient of Nitrogen Absorption (%)
timeFrame: 5 days
measure: Total Fat Excretion (Grams)
timeFrame: 5 days
measure: Total Stool Weight (Grams)
timeFrame: 5 days
measure: Stool Frequency
timeFrame: 5 days
measure: Percentage of Days With no Flatulence.
timeFrame: 5 days
measure: Percentage of Days With Formed/Normal Stools.
timeFrame: 5 days
measure: Percentage of Days With no Abdominal Pain.
timeFrame: 5 days
Other outcomes:
Locations:
city: Iowa City
country: United States
facility: Site 2
state: Iowa
city: Louisville
country: United States
facility: Site 5
state: Kentucky
city: Boston
country: United States
facility: Site 9
state: Massachusetts
city: Ann Arbor
country: United States
facility: Site 6
state: Michigan
city: Minneapolis
country: United States
facility: Site 4
state: Minnesota
city: Albuquerque
country: United States
facility: Site 8
state: New Mexico
city: Cincinnati
country: United States
facility: Site 1
state: Ohio
city: Oklahoma City
country: United States
facility: Site 10
state: Oklahoma
city: Oklahoma City
country: United States
facility: Site 7
state: Oklahoma
city: Hershey
country: United States
facility: Site 3
state: Pennsylvania
Study officials:
affiliation: Solvay Pharmaceuticals
name: Global Clinical Director Solvay
role: STUDY_DIRECTOR
References:
citation: Graff GR, Maguiness K, McNamara J, Morton R, Boyd D, Beckmann K, Bennett D. Efficacy and tolerability of a new formulation of pancrelipase delayed-release capsules in children aged 7 to 11 years with exocrine pancreatic insufficiency and cystic fibrosis: a multicenter, randomized, double-blind, placebo-controlled, two-period crossover, superiority study. Clin Ther. 2010 Jan;32(1):89-103. doi: 10.1016/j.clinthera.2010.01.012.
citation: Caras S, Boyd D, Zipfel L, Sander-Struckmeier S. Evaluation of stool collections to measure efficacy of PERT in subjects with exocrine pancreatic insufficiency. J Pediatr Gastroenterol Nutr. 2011 Dec;53(6):634-40. doi: 10.1097/MPG.0b013e3182281c38.