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

Cystic Fibrosis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01710644
• Other study ID #: NM-BL-101
• Secondary ID: 207862
• Status: Completed
• Phase: Phase 1/Phase 2
• First received: October 17, 2012
• Last updated: June 3, 2016
• Start date: May 2013
• Est. completion date: August 2014

Study information

• Verified date: June 2016
• Source: Nordmark Arzneimittel GmbH & Co. KG
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 35
• Est. completion date: August 2014
• Est. primary completion date: July 2014
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 12 Years and older

Eligibility

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

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Crossover Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Cystic Fibrosis
• Digestive System Diseases
• Exocrine Pancreatic Insufficiency
• Fibrosis
• Gastrointestinal Diseases
• Lung Diseases
• Pancreatic Insufficiency
• Respiratory Tract Diseases

Intervention

Drug:
• Burlulipase
Burlulipase oral solution will be taken with meals and snacks for 5 to 7 days
• Placebo (Caramel in sterile water)
Placebo will be taken with meals and snacks for 5 to 7 days

Locations

Country	Name	City	State
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio
United States	University Hospitals Case Medical Center	Cleveland	Ohio
United States	The Children's Medical Center of Dayton	Dayton	Ohio
United States	Penn State Milton S. Hershey Medical Center	Hershey	Pennsylvania
United States	The University of Iowa	Iowa City	Iowa
United States	Nemours Children's Clinic	Jacksonville	Florida
United States	West Virginia University Research Corporation	Morgantown	West Virginia
United States	Nemours Children's Clinic	Pensacola	Florida
United States	Via Cristi Hospitals Wichita, Inc.	Wichita	Kansas

Sponsors (2)

Lead Sponsor	Collaborator
Nordmark Arzneimittel GmbH & Co. KG	Parexel

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Symptomatology / Symptom Questionnaire	Comparison gastrointestinal parameters, recorded by the patient/carer in the Symptom Questionnaire each day during the Treatment Periods	7 days	No
Primary	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	No
Secondary	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	No