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

NCT ID: NCT01710644

Last Updated: 2016-06-07

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

35 participants

Study Classification

INTERVENTIONAL

Study Start Date

2013-05-31

Study Completion Date

2014-08-31

Brief Summary

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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

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Conditions

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

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Burlulipase

Burlulipase orally, per meal

Group Type EXPERIMENTAL

Burlulipase

Intervention Type DRUG

Burlulipase oral solution will be taken with meals and snacks for 5 to 7 days

Placebo (Caramel in sterile water)

Placebo orally, per meal

Group Type PLACEBO_COMPARATOR

Placebo (Caramel in sterile water)

Intervention Type DRUG

Placebo will be taken with meals and snacks for 5 to 7 days

Interventions

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Burlulipase

Burlulipase oral solution will be taken with meals and snacks for 5 to 7 days

Intervention Type DRUG

Placebo (Caramel in sterile water)

Placebo will be taken with meals and snacks for 5 to 7 days

Intervention Type DRUG

Other Intervention Names

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NM-BL Placebo

Eligibility Criteria

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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%
Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Parexel

INDUSTRY

Sponsor Role collaborator

Nordmark Arzneimittel GmbH & Co. KG

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Kristin Forssmann, MD

Role: STUDY_DIRECTOR

Nordmark Arzneimittel GmbH & Co. KG

James E. Heubi, MD

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital Medical Center, Cincinnati

Locations

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Nemours Children's Clinic

Jacksonville, Florida, United States

Site Status

Nemours Children's Clinic

Pensacola, Florida, United States

Site Status

The University of Iowa

Iowa City, Iowa, United States

Site Status

Via Cristi Hospitals Wichita, Inc.

Wichita, Kansas, United States

Site Status

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status

University Hospitals Case Medical Center

Cleveland, Ohio, United States

Site Status

The Children's Medical Center of Dayton

Dayton, Ohio, United States

Site Status

Penn State Milton S. Hershey Medical Center

Hershey, Pennsylvania, United States

Site Status

West Virginia University Research Corporation

Morgantown, West Virginia, United States

Site Status

Countries

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United States

References

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Somaraju URR, Solis-Moya A. Pancreatic enzyme replacement therapy for people with cystic fibrosis. Cochrane Database Syst Rev. 2020 Aug 5;8(8):CD008227. doi: 10.1002/14651858.CD008227.pub4.

Reference Type DERIVED
PMID: 32761612 (View on PubMed)

Heubi JE, Schaeffer D, Ahrens RC, Sollo N, Strausbaugh S, Graff G, Jain R, Witte S, Forssmann K. Safety and Efficacy of a Novel Microbial Lipase in Patients with Exocrine Pancreatic Insufficiency due to Cystic Fibrosis: A Randomized Controlled Clinical Trial. J Pediatr. 2016 Sep;176:156-161.e1. doi: 10.1016/j.jpeds.2016.05.049. Epub 2016 Jun 11.

Reference Type DERIVED
PMID: 27297209 (View on PubMed)

Other Identifiers

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207862

Identifier Type: OTHER

Identifier Source: secondary_id

NM-BL-101

Identifier Type: -

Identifier Source: org_study_id

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