Clinical trial NCT01710644: Efficacy and Tolerability of NM-BL in Patients With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Pancreatic Insufficiency
Cystic Fibrosis
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Lead sponsor: Nordmark Arzneimittel GmbH & Co. KG
Sponsor class: INDUSTRY
Collaborators: name: Parexel
First posted: 2012-10-19
Enrollment: 35
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

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


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%

Primary outcomes:
measure: 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%)
timeFrame: 72 hrs
Secondary outcomes:
measure: 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%)
timeFrame: 72 hrs
Other outcomes:
measure: Symptomatology / Symptom Questionnaire
timeFrame: 7 days
Locations:
city: Jacksonville
country: United States
facility: Nemours Children's Clinic
state: Florida
city: Pensacola
country: United States
facility: Nemours Children's Clinic
state: Florida
city: Iowa City
country: United States
facility: The University of Iowa
state: Iowa
city: Wichita
country: United States
facility: Via Cristi Hospitals Wichita, Inc.
state: Kansas
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Cleveland
country: United States
facility: University Hospitals Case Medical Center
state: Ohio
city: Dayton
country: United States
facility: The Children's Medical Center of Dayton
state: Ohio
city: Hershey
country: United States
facility: Penn State Milton S. Hershey Medical Center
state: Pennsylvania
city: Morgantown
country: United States
facility: West Virginia University Research Corporation
state: West Virginia
Study officials:
affiliation: Nordmark Arzneimittel GmbH & Co. KG
name: Kristin Forssmann, MD
role: STUDY_DIRECTOR
affiliation: Children's Hospital Medical Center, Cincinnati
name: James E. Heubi, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.
citation: 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.