Pancreatic InsufficiencyPhase 1/2NCT01710644
Efficacy and Tolerability of NM-BL in Patients With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis
A Phase 1/2 study testing Burlulipase in people with Pancreatic Insufficiency and Cystic Fibrosis, run by Nordmark Arzneimittel GmbH & Co. KG at 9 sites in United States.
Trial 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 Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Pancreatic Insufficiency, Cystic Fibrosis, Digestive System Diseases
- What is being tested?
- Burlulipase
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 35 participants
- Where
- 9 sitesUnited States
- Study dates
- May 2013to Aug 2014
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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%
Trial locations and contacts
9 sites in 1 country.
9 sites in 1 country
United States9 sites
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
University Hospitals Case Medical Center
Cleveland, Ohio, 44106
The Children's Medical Center of Dayton
Dayton, Ohio, 45404
Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033
The University of Iowa
Iowa City, Iowa, 52242
Nemours Children's Clinic
Jacksonville, Florida, 32207
West Virginia University Research Corporation
Morgantown, West Virginia, 26506
Nemours Children's Clinic
Pensacola, Florida, 32504
Via Cristi Hospitals Wichita, Inc.
Wichita, Kansas, 67214
About this study
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
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
- Conditions
- Pancreatic Insufficiency, Cystic Fibrosis, Digestive System Diseases, Lung Diseases, Respiratory Tract Diseases
- Being tested
- Burlulipase, Placebo (Caramel in sterile water)
- Medical subject headings
- Exocrine Pancreatic Insufficiency, Cystic Fibrosis, Digestive System Diseases, Lung Diseases, Respiratory Tract Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BurlulipaseDrug
Also called NM-BL
Burlulipase oral solution will be taken with meals and snacks for 5 to 7 days
Placebo (Caramel in sterile water)Drug
Also called Placebo
Placebo will be taken with meals and snacks for 5 to 7 days
| Study group | Role | What they receive |
|---|---|---|
| Burlulipase | Experimental | Burlulipase orally, per meal Receives: Drug: Burlulipase |
| Placebo (Caramel in sterile water) | Placebo comparator | Placebo orally, per meal Receives: Drug: Placebo (Caramel in sterile water) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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%)
Measured: 72 hrs
Secondary outcomes (1)
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%)
Measured: 72 hrs
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Oct 2012 (estimated)
- Study startMay 2013
- Primary completionJul 2014
- Study completionAug 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Pancreatic InsufficiencyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 35 participants (actual)
Sponsor and investigators
- Lead sponsor
- Nordmark Arzneimittel GmbH & Co. KG
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Parexel
- Responsible party
- Sponsor
- Investigators
- Kristin Forssmann, MD, Study director, Nordmark Arzneimittel GmbH & Co. KGJames E. Heubi, MD, Principal investigator, Children's Hospital Medical Center, Cincinnati
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01710644
- Sponsor's protocol ID
- NM-BL-101
- Other IDs
- 207862
- Status last verified
- Jun 2016
- First submitted
- 17 Oct 2012
- Last updated
- 7 Jun 2016
- Results posted
- No
- Data monitoring committee
- Yes
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.
PubMed 32761612Heubi 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.
PubMed 27297209
Study at a glance
From the registry record, last updated by the sponsor on 7 Jun 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT01710644
- Acronym / sponsor ID
- NM-BL-101
- Conditions
- Pancreatic Insufficiency, Cystic Fibrosis, Digestive System Diseases, Lung Diseases, Respiratory Tract Diseases
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 35 participants (actual)
- Lead sponsor
- Nordmark Arzneimittel GmbH & Co. KGIndustry (pharma, biotech, devices)
- Collaborators
- Parexel
- Study start
- May 2013
- Primary completion
- Jul 2014 (actual)
- Study completion
- Aug 2014 (actual)
- First posted
- 19 Oct 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Burlulipase | Experimental | Drug: BurlulipaseBurlulipase orally, per meal |
| Placebo (Caramel in sterile water) | Placebo comparator | Drug: Placebo (Caramel in sterile water)Placebo orally, per meal |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (6 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BurlulipaseAlso: NM-BLBurlulipase oral solution will be taken with meals and snacks for 5 to 7 days | Drug | Burlulipase |
| Placebo (Caramel in sterile water)Also: PlaceboPlacebo will be taken with meals and snacks for 5 to 7 days | Drug | Placebo (Caramel in sterile water) |
Primary outcome
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
1 secondary outcome
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Jun 2016.
- Derived from this study: PubMed 32761612Somaraju 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.
- Derived from this study: PubMed 27297209Heubi 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Kristin Forssmann, MD study director
Nordmark Arzneimittel GmbH & Co. KG
James E. Heubi, MD principal investigator
Children's Hospital Medical Center, Cincinnati
9 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Burlulipase in people with Pancreatic Insufficiency and Cystic Fibrosis, run by Nordmark Arzneimittel GmbH & Co. KG at 9 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 7 Jun 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01710644, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Jun 2016.