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

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

Study Overview

• Status: Completed
• Conditions: Digestive System Diseases; Respiratory Tract Diseases; Lung Diseases; Cystic Fibrosis; Pancreatic Insufficiency
• Intervention / Treatment: Drug: Burlulipase; Drug: Placebo (Caramel in sterile water)

• Study Type: Interventional
• Enrollment (Actual): 35
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• United States
  • Florida
    • Jacksonville, Florida, United States, 32207
      • Nemours Children's Clinic
    • Pensacola, Florida, United States, 32504
      • Nemours Children's Clinic
  • Iowa
    • Iowa City, Iowa, United States, 52242
      • The University of Iowa
  • Kansas
    • Wichita, Kansas, United States, 67214
      • Via Cristi Hospitals Wichita, Inc.
  • Ohio
    • Cincinnati, Ohio, United States, 45229
      • Cincinnati Children's Hospital Medical Center
    • Cleveland, Ohio, United States, 44106
      • University Hospitals Case Medical Center
    • Dayton, Ohio, United States, 45404
      • The Children's Medical Center of Dayton
  • Pennsylvania
    • Hershey, Pennsylvania, United States, 17033
      • Penn State Milton S. Hershey Medical Center
  • West Virginia
    • Morgantown, West Virginia, United States, 26506
      • West Virginia University Research Corporation

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 10 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Crossover Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Burlulipase; Burlulipase orally, per meal	Drug: Burlulipase; Burlulipase oral solution will be taken with meals and snacks for 5 to 7 days; Other Names: NM-BL
Placebo Comparator: Placebo (Caramel in sterile water); Placebo orally, per meal	Drug: Placebo (Caramel in sterile water); Placebo will be taken with meals and snacks for 5 to 7 days; Other Names: Placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
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 Outcome Measures

Outcome Measure	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
Symptomatology / Symptom Questionnaire	Comparison gastrointestinal parameters, recorded by the patient/carer in the Symptom Questionnaire each day during the Treatment Periods	7 days

Collaborators and Investigators

• Sponsor: Nordmark Arzneimittel GmbH & Co. KG
• Collaborators: Parexel
• Investigators: Principal Investigator: James E. Heubi, MD, Children's Hospital Medical Center, Cincinnati

Publications and helpful links

General Publications

• 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.
• 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.

Study record dates

Study Major Dates

• Study Start: May 1, 2013
• Primary Completion (Actual): July 1, 2014
• Study Completion (Actual): August 1, 2014

Study Registration Dates

• First Submitted: October 17, 2012
• First Submitted That Met QC Criteria: October 17, 2012
• First Posted (Estimate): October 19, 2012

Study Record Updates

• Last Update Posted (Estimate): June 7, 2016
• Last Update Submitted That Met QC Criteria: June 3, 2016
• Last Verified: June 1, 2016

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Infant, Newborn, Diseases; Genetic Diseases, Inborn; Pancreatic Diseases; Fibrosis; Lung Diseases; Gastrointestinal Diseases; Digestive System Diseases; Cystic Fibrosis; Respiratory Tract Diseases; Exocrine Pancreatic Insufficiency
• Other Study ID Numbers: NM-BL-101; 207862 (Other Identifier: Parexel)