Efficacy and Tolerability of NM-BL in Patients With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis

Phase 1

Completed

• Conditions: Pancreatic Insufficiency; Cystic Fibrosis; Lung Diseases; Respiratory Tract Diseases; Digestive System Diseases
• Interventions: Drug: Placebo (Caramel in sterile water); Drug: Burlulipase

• Registration Number: NCT01710644
• Lead Sponsor: Nordmark Arzneimittel GmbH & Co. KG

Brief Summary

The purpose of this Randomized, Double-blind, Multicenter, Two-period Crossover Study is to Assess the Efficacy and Tolerability of Burlulipase (NM-BL) in Patients with Exocrine Pancreatic Insufficiency due to Cystic Fibrosis

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2012-10-17
• Study First Submitted QC: 2012-10-17
• Study First Posted: 2012-10-19
• Study Start: 2013-05
• Primary Completion: 2014-07
• Study Completion: 2014-08
• Disposition First Submitted: 2016-04-04
• Disposition First Submitted QC: 2016-05-04
• Status Verified: 2016-06
• Last Update Submitted: 2016-06-03
• Disposition First Posted: 2016-06-06
• Last Update Posted: 2016-06-07

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 35

Inclusion Criteria

• Male and female patients aged ≥12 years from the date of informed consent
• Confirmed diagnosis of CF at screening
• Confirmed EPI by historical (within past 12 months) CFA <70% without use of PERTs or current fecal elastase <50 µg/g stool at screening
• Currently receiving PERT with a commercially available pancreatic enzyme
• Currently on stable treatment with proton pump inhibitors or H2 receptor antagonists
• Clinically stable condition without evidence of acute respiratory disease or any other acute condition

Exclusion Criteria

• History of fibrosing colonopathy
• History of significant bowel resection, in the opinion of the investigator, or solid organ transplant
• History of being refractory to pancreatic enzyme replacement
• Current diagnosis or history of distal intestinal obstruction syndrome
• Current diagnosis of small intestinal bacterial overgrowth, ileus or acute abdomen
• A body mass index percentile <10%

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: CROSSOVER

Arm && Interventions

Group	Intervention	Description
Placebo (Caramel in sterile water)	Placebo (Caramel in sterile water)	Placebo orally, per meal
Burlulipase	Burlulipase	Burlulipase orally, per meal

Primary Outcome Measures

Name	Time	Method
To evaluate the efficacy of NM-BL compared to placebo in patients with Exocrine Pancreatic Insufficiency (EPI) due to CF, where the primary variable is coefficient of fat absorption (CFA%)	72 hrs

Secondary Outcome Measures

Name	Time	Method
To evaluate the efficacy of NM-BL compared to placebo in patients with Exocrine Pancreatic Insufficiency (EPI)due to CF, where the primary variable is coefficient of nitrogen absorption (CNA%)	72 hrs

Trial Locations

Locations (8)

Nemours Children's Clinic; 🇺🇸 Pensacola, Florida, United States

The University of Iowa; 🇺🇸 Iowa City, Iowa, United States

The Children's Medical Center of Dayton; 🇺🇸 Dayton, Ohio, United States

Via Cristi Hospitals Wichita, Inc.; 🇺🇸 Wichita, Kansas, United States

West Virginia University Research Corporation; 🇺🇸 Morgantown, West Virginia, United States

Penn State Milton S. Hershey Medical Center; 🇺🇸 Hershey, Pennsylvania, United States

University Hospitals Case Medical Center; 🇺🇸 Cleveland, Ohio, United States

Cincinnati Children's Hospital Medical Center; 🇺🇸 Cincinnati, Ohio, United States