Cystic Fibrosis (CF), Exocrine Pancreatic Insufficiency (EPI)
Conditions
Keywords
Cystic Fibrosis (CF), EPI
Brief summary
In this study, ANG003, a pancreatic enzyme replacement therapy (PERT; commonly called "enzymes"), is being investigated as a potential treatment for exocrine pancreatic insufficiency (EPI). People with EPI due to Cystic Fibrosis (CF) may be eligible to participate in this study. The primary objective of this study is to evaluate the safety of ANG003 and see if it works as well compared to Creon, an approved PERT.
Interventions
DRUGANG003 Dose A
160,000 u\* lipase, 105,000 u\* protease, and 11,600 u\* amylase per dose (\*units are comparable to United States Pharmacopeiea \[USP\])
DRUGANG003 Dose B
240,000 u\* lipase, 105,000 u\* protease, and 11,600 u\* amylase per dose (\*units are comparable to United States Pharmacopeiea \[USP\])
DRUGCreon
The commercially available pPERT Creon will be the active comparator in the study. Subjects will be instructed to take Creon according to the package insert and PI direction.
Sponsors
Anagram Therapeutics, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
SINGLE (Outcomes Assessor)
Masking description
A centralized, blinded safety assessor will be responsible for conducting independent reviews of key safety data to ensure objective and consistent evaluation across all study sites.
Eligibility
Sex/Gender
ALL
Age
12 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Male and female subjects aged ≥12 years from the date of informed consent in the US and aged ≥18 years in the EU 2. Confirmed and documented diagnosis of CF 3. Diagnosed with severe EPI as defined by a fecal elastase ≤50 μg/g stool measured at Screening by a central laboratory. 4. Subject has controlled EPI and taking a stable dose of pancreatic enzyme replacement therapy (PERT) for 90 days prior to Screening. 5. Adequate nutritional status measured by body mass index (BMI) defined by: 1. BMI ≥25th percentile for children aged 12-17 years 2. BMI ≥18.5 kg/m2 for ≥18 years of age
Exclusion criteria
1. History of fibrosing colonopathy or recurring distal intestinal obstructive syndrome within 6 months of Screening. 2. History of lung or liver transplant, listing for organ transplant or significant bowel resection within the last 6 months. Subjects with a history of resection that does not result in short bowel syndrome are eligible. 3. Known hypersensitivity or other severe reaction to any ingredient of the investigational product (IP), Creon, or the stool marker (FD\&C Blue #2). 4. Any chronic diarrheal illness unrelated to pancreatic insufficiency, actively being treated for small intestinal bacterial overgrowth, requiring use of naso-gastric, J-tube, G-tube, and/or enteral feeding for the study duration, Clostridioides difficile infection within 6 months prior to Screening, or severe constipation defined as \<1 bowel movement/week.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Adverse events (AEs); Treatment-emergent AEs; Serious AEs; Discontinuation due to AEs; Clinical laboratory values | From Visit 1 to Visit 5, anticipated average 80 days | Adverse events (AEs); Treatment-emergent AEs; Serious AEs; Discontinuation due to AEs; Clinical laboratory values |
| Coefficient of Fat Absorption (CFA) | Period A (baseline) and Period B 72 hour CFA collection | Mean change from baseline CFA (i.e., Period B CFA minus Period A CFA) assessed for ANG003 Dose A, ANG003 Dose B, and Creon amongst subjects with baseline CFA ≥80%. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Plasma docosahexaenoic acid (DHA) and eicosapentaenoic acid (EPA) (ug/mL) | Period A (baseline) and Period B time 0, 1, 2, 4, 6, 8, 10 and 24 hour collections | Mean change from baseline for each treatment group amongst subjects with baseline CFA ≥80%. |
| Coefficient of Nitrogen Absorption (CNA) | Period A (baseline) and Period B 72 hour CNA collection | Mean change from baseline for each treatment group amongst subjects with baseline CFA ≥80%. |
| % of subjects with GI symptoms | From Visit 2 to Visit 5, anticipated average 59 days | Mean change from baseline for each treatment group amongst subjects with baseline CFA ≥80%. |
| Cumulative stool weight | Period A (baseline) and Period B 72 hour collection | Mean change from baseline for each treatment group amongst subjects with baseline CFA ≥80%. |
| Number of Stools per Day | Period A (baseline) and Period B 72 hour collection | Mean change from baseline for each treatment group amongst subjects with baseline CFA ≥80%. |
Countries
United States
Contacts
CONTACTEvan Bailey, MD
Outcome results
None listed