A Phase 2, Multicenter, Randomized, Active-controlled Study to Assess the Safety and Efficacy of ANG003 at Two Different Dose Levels in Subjects With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis
Status: Recruiting
Location: See all (26) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
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.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 12
Healthy Volunteers: f
View:
• Male and female subjects aged ≥12 years from the date of informed consent in the US and aged ≥18 years in the EU
• Confirmed and documented diagnosis of CF
• Diagnosed with severe EPI as defined by a fecal elastase ≤50 μg/g stool measured at Screening by a central laboratory.
• Subject has controlled EPI and taking a stable dose of pancreatic enzyme replacement therapy (PERT) for 90 days prior to Screening.
• Adequate nutritional status measured by body mass index (BMI) defined by:
‣ BMI ≥25th percentile for children aged 12-17 years
⁃ BMI ≥18.5 kg/m2 for ≥18 years of age
Locations
United States
California
Long Beach Memorial Medical Center
RECRUITING
Long Beach
Center for Cystic Fibrosis at Keck Medical Center of USC
NOT_YET_RECRUITING
Los Angeles
Colorado
National Jewish Health
NOT_YET_RECRUITING
Denver
Florida
University of Florida
NOT_YET_RECRUITING
Gainesville
Central Florida Pulmonary Group
NOT_YET_RECRUITING
Orlando
Iowa
University of Iowa
NOT_YET_RECRUITING
Iowa City
Illinois
The Cystic Fibrosis Institute
RECRUITING
Glenview
Kansas
University of Kansas Medical Center
NOT_YET_RECRUITING
Kansas City
Kentucky
University of Kentucky
NOT_YET_RECRUITING
Lexington
Massachusetts
Boston Children's Hospita
NOT_YET_RECRUITING
Boston
Massachusetts General Hospital
NOT_YET_RECRUITING
Boston
Maine
MaineHealth Pediatric Specialty Care
NOT_YET_RECRUITING
Portland
Michigan
University of Michigan, Michigan Medicine
NOT_YET_RECRUITING
Ann Arbor
Wayne State University Harper University Hospital
NOT_YET_RECRUITING
Detroit
New Jersey
Rutgers - Robert Wood Johnson Medical School
NOT_YET_RECRUITING
New Brunswick
New York
New York Medical College at Westchester Medical Center
NOT_YET_RECRUITING
Valhalla
Ohio
Children's Hospital Medical Center of Akron
NOT_YET_RECRUITING
Akron
Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center
NOT_YET_RECRUITING
Cleveland
Nationwide Children's Hospital, Columbus
NOT_YET_RECRUITING
Columbus
Toledo Children's Hospital
NOT_YET_RECRUITING
Toledo
Pennsylvania
Hershey Medical Center Pennsylvania State University
RECRUITING
Hershey
University of Pittsburgh Medical Center
NOT_YET_RECRUITING
Pittsburgh
Texas
University of Texas Southwestern / Children's Health
NOT_YET_RECRUITING
Dallas
Utah
Adult Cystic Fibrosis Center at the University of Utah
NOT_YET_RECRUITING
Salt Lake City
Wisconsin
University of Wisconsin
NOT_YET_RECRUITING
Madison
Froedtert & Medical College of Wisconsin
NOT_YET_RECRUITING
Milwaukee
Contact Information
Primary
Evan Bailey, MD
medical@anagramtx.com
617-466-3111
Time Frame
Start Date:2026-04-24
Estimated Completion Date:2027-07
Participants
Target number of participants:113
Treatments
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.
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.
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.
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.