Cystic Fibrosis, Digestive System Diseases, Lung Diseases, Pancreatic Insufficiency, Respiratory Tract Diseases
Conditions
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
Interventions
DRUGBurlulipase
Burlulipase oral solution will be taken with meals and snacks for 5 to 7 days
DRUGPlacebo (Caramel in sterile water)
Placebo will be taken with meals and snacks for 5 to 7 days
Sponsors
Parexel
Nordmark Arzneimittel GmbH & Co. KG
Study design
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
12 Years to No maximum
Healthy volunteers
No
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%
Design outcomes
Primary
| Measure | 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
| Measure | 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 |
Other
| Measure | Time frame | Description |
|---|---|---|
| Symptomatology / Symptom Questionnaire | 7 days | Comparison gastrointestinal parameters, recorded by the patient/carer in the Symptom Questionnaire each day during the Treatment Periods |
Countries
United States
Outcome results
None listed