A Randomized, Double-blind, Multicenter, Two-period Crossover Study to Assess the Efficacy and Tolerability of NM-BL (Burlulipase) in Patients With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis

Nordmark Arzneimittel GmbH & Co. KG8 sites in 1 country35 target enrollmentMay 2013

ConditionsPancreatic Insufficiency Cystic Fibrosis Digestive System Diseases Lung Diseases Respiratory Tract Diseases

InterventionsBurlulipase Placebo (Caramel in sterile water)

DrugsBurlulipase Placebo (Caramel in sterile water)

Overview

Phase: Phase 1
Intervention: Burlulipase
Conditions: Pancreatic Insufficiency
Sponsor: Nordmark Arzneimittel GmbH & Co. KG
Enrollment: 35
Locations: 8
Primary Endpoint: 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%)
Status: Completed
Last Updated: 9 years ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

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

Registry

clinicaltrials.gov

Start Date

May 2013

End Date

August 2014

Last Updated

9 years ago

Study Type

Interventional

Study Design

Crossover

Sex

All

Investigators

Sponsor

Nordmark Arzneimittel GmbH & Co. KG

Responsible Party

Sponsor

Eligibility Criteria

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%

Arms & Interventions

Burlulipase

Burlulipase orally, per meal

Intervention: Burlulipase

Placebo (Caramel in sterile water)

Placebo orally, per meal

Intervention: Placebo (Caramel in sterile water)

Outcomes

Primary Outcomes

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%)

Time Frame: 72 hrs

Secondary Outcomes

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)