Efficacy and Tolerability of NM-BL in Patients With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis
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
2 other identifiers
interventional
35
1 country
9
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
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_1
Started May 2013
9 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
October 17, 2012
CompletedFirst Posted
Study publicly available on registry
October 19, 2012
CompletedStudy Start
First participant enrolled
May 1, 2013
CompletedPrimary Completion
Last participant's last visit for primary outcome
July 1, 2014
CompletedStudy Completion
Last participant's last visit for all outcomes
August 1, 2014
CompletedJune 7, 2016
June 1, 2016
1.2 years
October 17, 2012
June 3, 2016
Conditions
Outcome Measures
Primary Outcomes (1)
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 Outcomes (1)
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
Other Outcomes (1)
Symptomatology / Symptom Questionnaire
7 days
Study Arms (2)
Burlulipase
EXPERIMENTALBurlulipase orally, per meal
Placebo (Caramel in sterile water)
PLACEBO COMPARATORPlacebo orally, per meal
Interventions
Burlulipase oral solution will be taken with meals and snacks for 5 to 7 days
Placebo will be taken with meals and snacks for 5 to 7 days
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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%
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Nordmark Arzneimittel GmbH & Co. KGlead
- Parexelcollaborator
Study Sites (9)
Nemours Children's Clinic
Jacksonville, Florida, 32207, United States
Nemours Children's Clinic
Pensacola, Florida, 32504, United States
The University of Iowa
Iowa City, Iowa, 52242, United States
Via Cristi Hospitals Wichita, Inc.
Wichita, Kansas, 67214, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
University Hospitals Case Medical Center
Cleveland, Ohio, 44106, United States
The Children's Medical Center of Dayton
Dayton, Ohio, 45404, United States
Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033, United States
West Virginia University Research Corporation
Morgantown, West Virginia, 26506, United States
Related Publications (2)
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.
PMID: 32761612DERIVEDHeubi 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.
PMID: 27297209DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Kristin Forssmann, MD
Nordmark Arzneimittel GmbH & Co. KG
- PRINCIPAL INVESTIGATOR
James E. Heubi, MD
Children's Hospital Medical Center, Cincinnati
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- CROSSOVER
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
October 17, 2012
First Posted
October 19, 2012
Study Start
May 1, 2013
Primary Completion
July 1, 2014
Study Completion
August 1, 2014
Last Updated
June 7, 2016
Record last verified: 2016-06