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

This study has been completed.
Sponsor:
Collaborator:
Parexel
Information provided by (Responsible Party):
Nordmark Arzneimittel GmbH & Co. KG
ClinicalTrials.gov Identifier:
NCT01710644
First received: October 17, 2012
Last updated: June 3, 2016
Last verified: June 2016
  Purpose
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

Condition Intervention Phase
Pancreatic Insufficiency Cystic Fibrosis Digestive System Diseases Lung Diseases Respiratory Tract Diseases Drug: Burlulipase Drug: Placebo (Caramel in sterile water) Phase 1 Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Nordmark Arzneimittel GmbH & Co. KG:

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%) [ Time Frame: 72 hrs ]

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%) [ Time Frame: 72 hrs ]

Other Outcome Measures:
  • Symptomatology / Symptom Questionnaire [ Time Frame: 7 days ]
    Comparison gastrointestinal parameters, recorded by the patient/carer in the Symptom Questionnaire each day during the Treatment Periods


Enrollment: 35
Study Start Date: May 2013
Study Completion Date: August 2014
Primary Completion Date: July 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Burlulipase
Burlulipase orally, per meal
Drug: Burlulipase
Burlulipase oral solution will be taken with meals and snacks for 5 to 7 days
Other Name: 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 Name: Placebo

  Eligibility

Ages Eligible for Study:   12 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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%
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01710644

Locations
United States, Florida
Nemours Children's Clinic
Jacksonville, Florida, United States, 32207
Nemours Children's Clinic
Pensacola, Florida, United States, 32504
United States, Iowa
The University of Iowa
Iowa City, Iowa, United States, 52242
United States, Kansas
Via Cristi Hospitals Wichita, Inc.
Wichita, Kansas, United States, 67214
United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
University Hospitals Case Medical Center
Cleveland, Ohio, United States, 44106
The Children's Medical Center of Dayton
Dayton, Ohio, United States, 45404
United States, Pennsylvania
Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, United States, 17033
United States, West Virginia
West Virginia University Research Corporation
Morgantown, West Virginia, United States, 26506
Sponsors and Collaborators
Nordmark Arzneimittel GmbH & Co. KG
Parexel
Investigators
Study Director: Kristin Forssmann, MD Nordmark Arzneimittel GmbH & Co. KG
Principal Investigator: James E. Heubi, MD Children's Hospital Medical Center, Cincinnati
  More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Nordmark Arzneimittel GmbH & Co. KG
ClinicalTrials.gov Identifier: NCT01710644     History of Changes
Other Study ID Numbers: NM-BL-101
207862 ( Other Identifier: Parexel )
Study First Received: October 17, 2012
Last Updated: June 3, 2016

Additional relevant MeSH terms:
Fibrosis
Cystic Fibrosis
Lung Diseases
Respiratory Tract Diseases
Exocrine Pancreatic Insufficiency
Digestive System Diseases
Gastrointestinal Diseases
Pathologic Processes
Pancreatic Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases

ClinicalTrials.gov processed this record on August 18, 2017