A Study of the Efficacy and Tolerability of Pancrelipase Microtablet (MT) Capsules for the Treatment of Cystic Fibrosis-dependent Exocrine Pancreatic Insufficiency

NCT00662675 | Completed Phase 3 Results

• 1 other identifier: CR014719
• Study Type: interventional
• Target: 40
• Locations: 2 countries
• Sites: 11

Brief Summary

The purpose of this study is to assess the effectiveness and safety of oral pancrelipase MT in the treatment of adult and pediatric/adolescent cystic fibrosis (CF) patients with clinical symptoms of exocrine pancreatic insufficiency (EPI).

Conditions

Exocrine Pancreatic Insufficiency; Steatorrhea; Malabsorption Syndromes; Cystic Fibrosis

Keywords

Exocrine pancreatic insufficiency; Steatorrhea; Malabsorption syndromes; Cystic fibrosis; Pediatrics; Adult; Pancrelipase

Outcome Measures

Primary Outcomes (1)

• Change in the Coefficient of Fat Absorption (COA-fat Percent)

  Change in the coefficient of fat absorption (percent COA-fat) from the 72-hour inpatient period in the open-label phase to the 72-hour period inpatient period in the double-blind (withdrawal) phase.

  72-hours stool collection in the open-label phase to the end of 72-hours stool collection in the double-blind withdrawal phase.

Secondary Outcomes (2)

• Change in Percent COA-Protein (Nitrogen)

  72-hours stool collection in the open-label phase to the end of 72-hours stool collection in the double-blind withdrawal phase.

• Percent of Patients Reporting Clinical Signs and Symptoms of Exocrine Pancreatic Insufficiency (EPI) During the Double-Blind Phase

  Entire 7 days double-blind phase

Study Arms (2)

001

EXPERIMENTAL

Pancrease MT 10.5 or MT 21 Pancrease MT capsules for maximum dose of 10 000 lipase units / Kg / day

Drug: Pancrease MT 10.5, or MT 21

002

EXPERIMENTAL

Placebo for Pancrease MT 10.5 or MT 21 Capsules with Pancrease MT excipients without the active enzymes

Drug: Placebo for Pancrease MT 10.5 or MT 21

Interventions

Pancrease MT 10.5, or MT 21 DRUG

Pancrease MT capsules for maximum dose of 10,000 lipase units / Kg / day

001

Placebo for Pancrease MT 10.5 or MT 21 DRUG

Capsules with Pancrease MT excipients without the active enzymes

002

Eligibility Criteria

• Age: 7 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Have a diagnosis of CF documented by sweat chloride results (\>60 mmol/L) and require pancreatic enzyme replacement therapy (PERT) to control clinical symptoms of EPI (nausea, vomiting, bloating, diarrhea, and abdominal pain) with a history of excess fat in the feces
• Have documentation of an abnormal COA-fat and a fecal elastase result of \<100 micrograms fecal elastase/gram stool
• Must be on a stable diet and dose of pancreatic enzyme supplementation that has provided satisfactory symptom control for at least the past 1 month

You may not qualify if:

• No extreme physical wasting with loss of weight and muscle mass
• No severe, acute, or chronic pulmonary disease unrelated to complications of CF
• No worsening of pulmonary disease in past 30 days
• No use of drugs known to affect blood uric acid concentrations (e.g., aspirin, diflunisal, allopurinol, probenecid, thiazide diuretics, phenylbutazone, sulfinpyrazone)
• No known congenital (present at birth) abnormalities of the gastrointestinal tract, heart, or liver
• No distal intestinal obstruction syndrome (DIOS)

Sponsors & Collaborators

• Johnson & Johnson Pharmaceutical Research & Development, L.L.C.: lead

Study Sites (11)

Unknown Facility

Long Beach, California, United States

Location

Unknown Facility

Los Angeles, California, United States

Location

Unknown Facility

Orlando, Florida, United States

Location

Unknown Facility

Louisville, Kentucky, United States

Location

Unknown Facility

Las Vegas, Nevada, United States

Location

Unknown Facility

Long Branch, New Jersey, United States

Location

Unknown Facility

Cincinnati, Ohio, United States

Location

Unknown Facility

Cleveland, Ohio, United States

Location

Unknown Facility

Oklahoma City, Oklahoma, United States

Location

Unknown Facility

Pittsburgh, Pennsylvania, United States

Location

Unknown Facility

Vancouver, British Columbia, Canada

Location

Related Links

• A randomized double-blind (withdrawal) Phase 3 study to evaluate the efficacy and tolerability of PANCREASE MT capsules compared with placebo in the treatment of subjects with cystic fibrosis-dependent exocrine pancreatic insufficiency

MeSH Terms

Conditions

Exocrine Pancreatic Insufficiency; Steatorrhea; Malabsorption Syndromes; Cystic Fibrosis

Interventions

MT 21

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Intestinal Diseases; Gastrointestinal Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Results Point of Contact

• Title: Director, Clinical Team Leader
• Organization: Johnson & Johnson Pharmaceutical Research & Development, LLC

609-730-3158

Study Officials

• Johnson & Johnson Pharmaceutical Research & Development, L.L. C. Clinical Trial

  Johnson & Johnson Pharmaceutical Research & Development, L.L.C.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 17, 2008
• First Posted: April 21, 2008
• Study Start: August 1, 2008
• Primary Completion: February 1, 2009
• Study Completion: February 1, 2009
• Last Updated: May 9, 2014
• Results First Posted: March 17, 2010

Record last verified: 2014-04

Locations

US (10); CA (1)