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

This study is currently recruiting participants. (see Contacts and Locations)
Verified June 2014 by Nordmark Arzneimittel GmbH & Co. KG
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 2, 2014
Last verified: June 2014
  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
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Masking: Double Blind (Subject, Caregiver, 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 ] [ Designated as safety issue: No ]

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 ] [ Designated as safety issue: No ]

Other Outcome Measures:
  • Symptomatology / Symptom Questionnaire [ Time Frame: 7 days ] [ Designated as safety issue: No ]
    Comparison gastrointestinal parameters, recorded by the patient/carer in the Symptom Questionnaire each day during the Treatment Periods


Estimated Enrollment: 28
Study Start Date: May 2013
Estimated Study Completion Date: October 2014
Estimated Primary Completion Date: August 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
Genders Eligible for Study:   Both
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

Contacts
Contact: Cornelia Breuer +49-4122-712-0 cornelia.breuer@nordmark-pharma.de

Locations
United States, Florida
Nemours Children's Clinic Recruiting
Jacksonville, Florida, United States, 32207
Contact: Betty DeLuca    904-697-3804    Elizabeth.Deluca@nemours.org   
Nemours Children's Clinic Recruiting
Pensacola, Florida, United States, 32504
Contact: Okan Elidemir    850-473-4784    okan.elidemir@nemours.org   
United States, Iowa
The University of Iowa Recruiting
Iowa City, Iowa, United States, 52242
Contact: Mary Teresi    319-384-7546    mary-teresi@uiowa.edu   
United States, Kansas
Via Cristi Hospitals Wichita, Inc. Recruiting
Wichita, Kansas, United States, 67214
Contact: Sheri Miller    316-268-6028    Sheri.Miller@via-christi.org   
United States, Ohio
Cincinnati Children's Hospital Medical Center Recruiting
Cincinnati, Ohio, United States, 45229
Contact: Donna Buckley    513-636-8549    donna.buckley@cchmc.org   
University Hospitals Case Medical Center Recruiting
Cleveland, Ohio, United States, 44106
Contact: David Weaver    216-983-0469    david.weaver@uhhospitals.org   
The Children's Medical Center of Dayton Recruiting
Dayton, Ohio, United States, 45404
Contact: Sandy Bartosik    937-641-4004    bartosiks@childrensdayton.org   
United States, Pennsylvania
Penn State Milton S. Hershey Medical Center Recruiting
Hershey, Pennsylvania, United States, 17033
Contact: Diane Kitch    717-531-5646    dkitch@hmc.psu.edu   
United States, West Virginia
West Virginia University Research Corporation Recruiting
Morgantown, West Virginia, United States, 26506
Contact: Sue Collins    304-293-7348    scollins@hsc.wvu.edu   
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

No publications provided

Responsible Party: Nordmark Arzneimittel GmbH & Co. KG
ClinicalTrials.gov Identifier: NCT01710644     History of Changes
Other Study ID Numbers: NM-BL-101, 207862
Study First Received: October 17, 2012
Last Updated: June 2, 2014
Health Authority: United States: Food and Drug Administration

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

ClinicalTrials.gov processed this record on July 26, 2014