A Phase II, Randomized, Investigator-Blinded, Parallel-Group, Pilot Study Evaluating the Safety, Palatability and Efficacy of Four Doses of Pancrelipase Microtablets in the Treatment of Infants and Toddlers with Cystic Fibrosis-Related Pancreatic Insufficiency and Fat Malabsorptio

Phase 1

• Conditions: Cystic Fibrosis-Related Pancreatic Insufficiency and Fat Malabsorption

• Registration Number: EUCTR2005-001438-34-BE
• Lead Sponsor: McNeil Consumer and Specialty Pharmaceuticals

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2005-07-15
• Last Update Posted: 2 October 2017

Recruitment & Eligibility

• Status: A
• Sex: All
• Target Recruitment: 20

Inclusion Criteria

1. Male and/or female infants/toddlers 6 to 24 months of age.
2. Diagnosis of CF confirmed by genetic testing including chromosomal analysis and/or clinical criteria including abnormal results of pilocarpine iontophoresis sweat testing consistent with diagnosis of CF.
3. Historical documentation of abnormal coefficient of fat absorption (COA)
4. Subject’s parent or legal guardian must sign informed consent.
5. Stable patient requiring enzyme therapy for management of steatorrhoea in subjects with defined diagnosis as set forth by criteria 2 and 3
6. Historical documentation of abnormal coefficient of fat absorption (COA) or presence of <15 micrograms fecal elastase/gram stool consistent with diagnosis of severe pancreatic insufficiency.
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Stable antibiotic therapy for small bowel overgrowth or chronic pulmonary infection.
2. Hypersensitivity to porcine products.
3. Use of prokinetics including metoclopramide or cisapride.
4. Concurrent nasogastric tube feeding for supplemental enteral nutrition.
5. Concomitant steroid therapy.
6. Exacerbation of chronic lung infections.
7. Use of concomitant H2 blockers or proton pump inhibitors as concomitant therapy. 8. Use of herbal supplements.
9. Clinically significant vomiting, malnutrition, or severe dehydration.
10. Severe constipation and intestinal resection.
11. Uncorrected electrolyte disorders (such as hypokalaemia, hypocalcaemia, hypomagnesaemia).
12. Known chromosome abnormality or congenital anomalies of the gastrointestinal tract, heart or liver; including gastrointestinal tract abnormalities.
13. Clinically significant disease that could interfere with the adequate assessment of the study drug.
14. HIV infection.
15. History of drug sensitivity to any of the study medication ingredients;
16. Subject in any other investigational drug study within the previous 30 days of consent.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: The objectives of this study are to evaluate preliminary safety, palatability and efficacy of pancrelipase microtablets to improve steatorrhoea in infants and toddlers with cystic fibrosis (CF).;Secondary Objective: None stated.;Primary end point(s): The primary endpoints will be:<br><br>• the change in coefficient of fecal fat absorption (COA) measured from baseline to the end of study period.<br>• assessment of daily palatability using a 4-point scale.<br>• assessment of percentage of CO2 expired by a 13C-medium chain triglyceride breath test as a measure of exogenous lipase activity and as a surrogate marker of lipase activity and pharmacodynamic effect of pancreatic enzyme therapy.