Working…
COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC: https://www.coronavirus.gov.

Get the latest research information from NIH: https://www.nih.gov/coronavirus.
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01710644
Recruitment Status : Completed
First Posted : October 19, 2012
Last Update Posted : June 7, 2016
Sponsor:
Collaborator:
Parexel
Information provided by (Responsible Party):
Nordmark Arzneimittel GmbH & Co. KG

Tracking Information
First Submitted Date  ICMJE October 17, 2012
First Posted Date  ICMJE October 19, 2012
Last Update Posted Date June 7, 2016
Study Start Date  ICMJE May 2013
Actual Primary Completion Date July 2014   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 22, 2013)
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 ]
Original Primary Outcome Measures  ICMJE
 (submitted: October 17, 2012)
To evaluate the efficacy of NM-BL compared to placebo in patients with EPI due to CF, where the primary variable is CFA%. [ Time Frame: 72 hrs ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: October 22, 2013)
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 ]
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures
 (submitted: October 22, 2013)
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
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Efficacy and Tolerability of NM-BL in Patients With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis
Official Title  ICMJE 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
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
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE
  • Pancreatic Insufficiency
  • Cystic Fibrosis
  • Digestive System Diseases
  • Lung Diseases
  • Respiratory Tract Diseases
Intervention  ICMJE
  • Drug: Burlulipase
    Burlulipase oral solution will be taken with meals and snacks for 5 to 7 days
    Other Name: NM-BL
  • Drug: Placebo (Caramel in sterile water)
    Placebo will be taken with meals and snacks for 5 to 7 days
    Other Name: Placebo
Study Arms  ICMJE
  • Experimental: Burlulipase
    Burlulipase orally, per meal
    Intervention: Drug: Burlulipase
  • Placebo Comparator: Placebo (Caramel in sterile water)
    Placebo orally, per meal
    Intervention: Drug: Placebo (Caramel in sterile water)
Publications * 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.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: August 27, 2014)
35
Original Estimated Enrollment  ICMJE
 (submitted: October 17, 2012)
28
Actual Study Completion Date  ICMJE August 2014
Actual Primary Completion Date July 2014   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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%
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 12 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01710644
Other Study ID Numbers  ICMJE NM-BL-101
207862 ( Other Identifier: Parexel )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Nordmark Arzneimittel GmbH & Co. KG
Study Sponsor  ICMJE Nordmark Arzneimittel GmbH & Co. KG
Collaborators  ICMJE Parexel
Investigators  ICMJE
Study Director: Kristin Forssmann, MD Nordmark Arzneimittel GmbH & Co. KG
Principal Investigator: James E. Heubi, MD Children's Hospital Medical Center, Cincinnati
PRS Account Nordmark Arzneimittel GmbH & Co. KG
Verification Date June 2016

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP