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

Primary Purpose

Pancreatic Insufficiency, Cystic Fibrosis, Digestive System Diseases

Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Burlulipase
Placebo (Caramel in sterile water)
Sponsored by
Nordmark Arzneimittel GmbH & Co. KG
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Pancreatic Insufficiency

Eligibility Criteria

12 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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%

Sites / Locations

  • Nemours Children's Clinic
  • Nemours Children's Clinic
  • The University of Iowa
  • Via Cristi Hospitals Wichita, Inc.
  • Cincinnati Children's Hospital Medical Center
  • University Hospitals Case Medical Center
  • The Children's Medical Center of Dayton
  • Penn State Milton S. Hershey Medical Center
  • West Virginia University Research Corporation

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Burlulipase

Placebo (Caramel in sterile water)

Arm Description

Burlulipase orally, per meal

Placebo orally, per meal

Outcomes

Primary Outcome Measures

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%)

Secondary Outcome Measures

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%)

Full Information

First Posted
October 17, 2012
Last Updated
June 3, 2016
Sponsor
Nordmark Arzneimittel GmbH & Co. KG
Collaborators
Parexel
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1. Study Identification

Unique Protocol Identification Number
NCT01710644
Brief Title
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

2. Study Status

Record Verification Date
June 2016
Overall Recruitment Status
Completed
Study Start Date
May 2013 (undefined)
Primary Completion Date
July 2014 (Actual)
Study Completion Date
August 2014 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Nordmark Arzneimittel GmbH & Co. KG
Collaborators
Parexel

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Pancreatic Insufficiency, Cystic Fibrosis, Digestive System Diseases, Lung Diseases, Respiratory Tract Diseases

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Crossover Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
35 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Burlulipase
Arm Type
Experimental
Arm Description
Burlulipase orally, per meal
Arm Title
Placebo (Caramel in sterile water)
Arm Type
Placebo Comparator
Arm Description
Placebo orally, per meal
Intervention Type
Drug
Intervention Name(s)
Burlulipase
Other Intervention Name(s)
NM-BL
Intervention Description
Burlulipase oral solution will be taken with meals and snacks for 5 to 7 days
Intervention Type
Drug
Intervention Name(s)
Placebo (Caramel in sterile water)
Other Intervention Name(s)
Placebo
Intervention Description
Placebo will be taken with meals and snacks for 5 to 7 days
Primary Outcome Measure Information:
Title
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%)
Time Frame
72 hrs
Secondary Outcome Measure Information:
Title
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%)
Time Frame
72 hrs
Other Pre-specified Outcome Measures:
Title
Symptomatology / Symptom Questionnaire
Description
Comparison gastrointestinal parameters, recorded by the patient/carer in the Symptom Questionnaire each day during the Treatment Periods
Time Frame
7 days

10. Eligibility

Sex
All
Minimum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
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%
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Kristin Forssmann, MD
Organizational Affiliation
Nordmark Arzneimittel GmbH & Co. KG
Official's Role
Study Director
First Name & Middle Initial & Last Name & Degree
James E. Heubi, MD
Organizational Affiliation
Children's Hospital Medical Center, Cincinnati
Official's Role
Principal Investigator
Facility Information:
Facility Name
Nemours Children's Clinic
City
Jacksonville
State/Province
Florida
ZIP/Postal Code
32207
Country
United States
Facility Name
Nemours Children's Clinic
City
Pensacola
State/Province
Florida
ZIP/Postal Code
32504
Country
United States
Facility Name
The University of Iowa
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
Country
United States
Facility Name
Via Cristi Hospitals Wichita, Inc.
City
Wichita
State/Province
Kansas
ZIP/Postal Code
67214
Country
United States
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
University Hospitals Case Medical Center
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44106
Country
United States
Facility Name
The Children's Medical Center of Dayton
City
Dayton
State/Province
Ohio
ZIP/Postal Code
45404
Country
United States
Facility Name
Penn State Milton S. Hershey Medical Center
City
Hershey
State/Province
Pennsylvania
ZIP/Postal Code
17033
Country
United States
Facility Name
West Virginia University Research Corporation
City
Morgantown
State/Province
West Virginia
ZIP/Postal Code
26506
Country
United States

12. IPD Sharing Statement

Citations:
PubMed Identifier
32761612
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.
Results Reference
derived
PubMed Identifier
27297209
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.
Results Reference
derived

Learn more about this trial

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

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