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

Nordmark Pharma

Status and phase

Completed

Phase 2

Phase 1

Conditions

Pancreatic Insufficiency

Lung Diseases

Respiratory Tract Diseases

Digestive System Diseases

Cystic Fibrosis

Treatments

Drug: Placebo (Caramel in sterile water)

Drug: Burlulipase

Study type

Interventional

Funder types

Industry

Identifiers

NCT01710644

NM-BL-101

207862 (Other Identifier)

Details and patient eligibility

About

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

Enrollment

35 patients

Sex

All

Ages

12+ years old

Volunteers

No Healthy Volunteers

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%