Clinical trial NCT00775528: Study Investigating a Delayed-Release Pancrelipase in Patients With Pancreatic Exocrine Insufficiency Due to Cystic Fibrosis
Official title: An Open Label, Multi-center, Study to Assess the Safety and Tolerability of Pancrelipase Delayed Release Capsules in Infants and Children Less Than 7 Years of Age 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: 2008-10-20
Enrollment: 19
Sex: ALL
Minimum age (years): 1
Maximum age (years): 6
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This study will assess the safety and tolerability of pancrelipase delayed release capsules in subjects up to 6 years of age with Pancreatic Exocrine Insufficiency (PEI) due to Cystic Fibrosis.


Eligibility criteria: Inclusion Criteria:

* Confirmed Cystic Fibrosis (CF) diagnosis by two positive chloride sweat tests or gene analysis
* Current or historical human fecal elastase \< 50µg/gstool
* Weight greater than 3.75 kg
* Age 1 month to 6 years
* 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

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: Number of Patients With at Least One Treatment Emergent Adverse Event (TEAE)
timeFrame: 10 Days
Secondary outcomes:
measure: Stool Fat (% Fat)
timeFrame: Last 3 days in a 10-day treatment period
measure: Fat Intake (g)
timeFrame: Last 3 days in a 10-day treatment period
measure: Total Calorie Intake (kcal)
timeFrame: Last 3 days in a 10-day treatment period
Other outcomes:
Locations:
city: Boise
country: United States
facility: Site 11
state: Idaho
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: Detroit
country: United States
facility: Site 12
state: Michigan
city: Minneapolis
country: United States
facility: Site 4
state: Minnesota
city: Long Branch
country: United States
facility: Site 13
state: New Jersey
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, McNamara J, Royall J, Caras S, Forssmann K. Safety and tolerability of a new formulation of pancrelipase delayed-release capsules (CREON) in children under seven years of age with exocrine pancreatic insufficiency due to cystic fibrosis: an open-label, multicentre, single-treatment-arm study. Clin Drug Investig. 2010;30(6):351-64. doi: 10.2165/11533390-000000000-00000.