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

Study Overview
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Brief Summary
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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

Official Title
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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

Conditions
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Pancreatic Insufficiency, Cystic Fibrosis, Digestive System Diseases, Lung Diseases, Respiratory Tract Diseases

Intervention / Treatment
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* Drug: Burlulipase
* Drug: Placebo (Caramel in sterile water)


Participation Criteria
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Eligibility Criteria
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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%

Ages Eligible for Study
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Minimum Age: 12 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Burlulipase<br>Burlulipase orally, per meal | Drug: Burlulipase<br>* Burlulipase oral solution will be taken with meals and snacks for 5 to 7 days<br>* Other names: NM-BL;|
| Placebo Comparator: Placebo (Caramel in sterile water)<br>Placebo orally, per meal | Drug: Placebo (Caramel in sterile water)<br>* Placebo will be taken with meals and snacks for 5 to 7 days<br>* Other names: Placebo;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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%) |  | 72 hrs | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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%) |  | 72 hrs |