A study of the efficacy and tolerability of pancrelipase microtablet (MT) capsules for the treatment of cystic fibrosis-dependent exocrine pancreatic insufficiency

• Register: EUCTR
• Last refreshed on: 7 April 2015
• Main ID: EUCTR2015-001219-11-Outside-EU/EEA
• Date of registration: 31/03/2015
• Prospective Registration: Yes
• Primary sponsor: Johnson & Johnson Pharmaceutical Research and Development, L.L.C.
• Public title: A study of the efficacy and tolerability of pancrelipase microtablet (MT) capsules for the treatment of cystic fibrosis-dependent exocrine pancreatic insufficiency
• Scientific title: A randomized double-blind (withdrawal) phase 3 study to evaluate the efficacy and tolerability of pancrelipase MT capsules compared with placebo in the treatment of subjects with cystic fibrosis-dependent exocrine pancreatic insufficiency
• Target sample size: 40
• Recruitment status: NA
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2015-001219-11
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no Number of treatment arms in the trial: 2

Countries of recruitment

Canada, United States

Contacts

• Name: Clinical Registry Group-JB BV
• Affiliation: Janssen Research and Development

• Name: Clinical Registry Group-JB BV
• Affiliation: Janssen Research and Development

Key inclusion & exclusion criteria

Inclusion criteria:
- 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
Are the trial subjects under 18? yes
Number of subjects for this age range: 40
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 40
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
- 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)


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Cystic fibrosis
MedDRA version: 17.1 Level: LLT Classification code 10011765 Term: Cystic fibrosis pancreas System Organ Class: 100000004850

Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]

Intervention(s)

Trade Name: PANCREASE MT
Product Name: PANCREASE MT
Pharmaceutical Form: Capsule
INN or Proposed INN: pancrelipase
Other descriptive name: PANCRELIPASE AMYLASE
Concentration unit: Other
Concentration type: up to
Concentration number: 10000-
Pharmaceutical form of the placebo: Capsule
Route of administration of the placebo: Oral use

Primary Outcome(s)

Primary end point(s): Change in the coefficient of fat absorption (COA-fat percent)

Timepoint(s) of evaluation of this end point: 72-hours stool collection in the open-label phase to the end of 72-hours stool collection in the double-blind withdrawal phase.

Main Objective: 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).

Secondary Objective: Not applicable

Secondary Outcome(s)

Secondary end point(s): - Change in Percent COA-Protein (Nitrogen)
- Percent of Patients Reporting Clinical Signs and Symptoms of Exocrine Pancreatic Insufficiency (EPI) During the Double-Blind Phase

Timepoint(s) of evaluation of this end point: - 72-hours stool collection in the open-label phase to the end of 72-hours stool collection in the double-blind withdrawal phase.
- Entire 7 days double-blind phase

Secondary ID(s)

PNCRLPCYS3001

Source(s) of Monetary Support

Johnson & Johnson Pharmaceutical Research and Development