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NCT07450547PHASE2Recruiting

Phase 2 Study to Assess the Safety and Efficacy of ANG003

Anagram Therapeutics, Inc.

Start Date

4/24/2026

Completion Date

7/1/2027

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 Criteria

Age Range: 12 years to No maximum

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.

Interventions

DRUG

ANG003 Dose A

DRUG

ANG003 Dose B

DRUG

Creon

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Conditions

Exocrine Pancreatic Insufficiency (EPI)Cystic Fibrosis (CF)

Locations

Long Beach Memorial Medical Center

Long Beach, California 90806

United States

Center for Cystic Fibrosis at Keck Medical Center of USC

Los Angeles, California 90033

United States

National Jewish Health

Denver, Colorado 80206

United States

University of Florida

Gainesville, Florida 32610

United States

Central Florida Pulmonary Group

Orlando, Florida 32803

United States

The Cystic Fibrosis Institute

Glenview, Illinois 60025

United States

University of Iowa

Iowa City, Iowa 52242

United States

University of Kansas Medical Center

Kansas City, Kansas 66160

United States

University of Kentucky

Lexington, Kentucky 40506

United States

MaineHealth Pediatric Specialty Care

Portland, Maine 04102

United States

Massachusetts General Hospital

Boston, Massachusetts 02114

United States

Boston Children's Hospita

Boston, Massachusetts 02115

United States

University of Michigan, Michigan Medicine

Ann Arbor, Michigan 48109

United States

Wayne State University Harper University Hospital

Detroit, Michigan 48201

United States

Rutgers - Robert Wood Johnson Medical School

New Brunswick, New Jersey 08903

United States

New York Medical College at Westchester Medical Center

Valhalla, New York 10595

United States

Children's Hospital Medical Center of Akron

Akron, Ohio 44308

United States

Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center

Cleveland, Ohio 44106

United States

Nationwide Children's Hospital, Columbus

Columbus, Ohio 43205

United States

Toledo Children's Hospital

Toledo, Ohio 43606

United States

Hershey Medical Center Pennsylvania State University

Hershey, Pennsylvania 17033

United States

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania 15224

United States

University of Texas Southwestern / Children's Health

Dallas, Texas 75207

United States

Adult Cystic Fibrosis Center at the University of Utah

Salt Lake City, Utah 84112

United States

University of Wisconsin

Madison, Wisconsin 53792

United States

Froedtert & Medical College of Wisconsin

Milwaukee, Wisconsin 53226

United States