Phase 2 Study to Assess the Safety and Efficacy of ANG003

Purpose

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.

Conditions

  • Exocrine Pancreatic Insufficiency (EPI)
  • Cystic Fibrosis (CF)

Eligibility

Eligible Ages
Over 12 Years
Eligible Sex
All
Accepts 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.

Study Design

Phase
Phase 2
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
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.

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
ANG003 Dose A 		All subjects will be acclimated to Creon for 14-days and then will undergo a baseline CFA analysis. If baseline CFA is >/= 80%, the subject may be randomized to ANG003 Dose A for a 21-day acclimation and subsequent CFA analysis.
  • Drug: ANG003 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])
Experimental
ANG003 Dose B 		All subjects will be acclimated to Creon for 14-days and then will undergo a baseline CFA analysis. If baseline CFA is >/= 80%, the subject may be randomized to ANG003 Dose B for a 21-day acclimation and subsequent CFA analysis.
  • Drug: ANG003 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])
Active Comparator
Creon 		All subjects will be acclimated to Creon for 14-days and then will undergo a baseline CFA analysis. If baseline CFA is >/= 80%, the subject may be randomized to Creon for an additional 21-days and subsequent CFA analysis.
  • Drug: Creon
    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.
No Intervention
Off Enzyme 		All subjects will be acclimated to Creon for 14-days and then will undergo a baseline CFA analysis. If baseline CFA is >/= 80%, the subject may be randomized to the off enzyme arm. Subjects will receive Creon for an additional 21-day and the subsequent CFA analysis will be off enzyme. Total time off enzyme will be 4-7 days.
Other
60% to <80% CFA ANG003 Dose B 		All subjects will be acclimated to Creon for 14-days and then will undergo a baseline CFA analysis. Participants with a Period A CFA result of 60% to <80% will be assigned to ANG003 Dose B.
  • Drug: ANG003 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])

Recruiting Locations

The Cystic Fibrosis Institute
Glenview, Illinois 60025
Contact:
Steven R Boas, MD
847-998-3434
sboas@wecare4lungs.com

More Details

Status
Recruiting
Sponsor
Anagram Therapeutics, Inc.

Study Contact

Evan Bailey, MD
617-466-3111
medical@anagramtx.com