RecruitingPhase 2NCT07450547

Phase 2 Study to Assess the Safety and Efficacy of ANG003

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


Sponsor

Anagram Therapeutics, Inc.

Enrollment

113 participants

Start Date

Apr 24, 2026

Study Type

INTERVENTIONAL

Conditions

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

Min Age: 12 Years

Inclusion Criteria7

  • 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

Exclusion Criteria4

  • History of fibrosing colonopathy or recurring distal intestinal obstructive syndrome within 6 months of Screening.
  • 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.
  • Known hypersensitivity or other severe reaction to any ingredient of the investigational product (IP), Creon, or the stool marker (FD\&C Blue #2).
  • 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.

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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.


Locations(26)

Long Beach Memorial Medical Center

Long Beach, California, United States

Center for Cystic Fibrosis at Keck Medical Center of USC

Los Angeles, California, United States

National Jewish Health

Denver, Colorado, United States

University of Florida

Gainesville, Florida, United States

Central Florida Pulmonary Group

Orlando, Florida, United States

The Cystic Fibrosis Institute

Glenview, Illinois, United States

University of Iowa

Iowa City, Iowa, United States

University of Kansas Medical Center

Kansas City, Kansas, United States

University of Kentucky

Lexington, Kentucky, United States

MaineHealth Pediatric Specialty Care

Portland, Maine, United States

Massachusetts General Hospital

Boston, Massachusetts, United States

Boston Children's Hospita

Boston, Massachusetts, United States

University of Michigan, Michigan Medicine

Ann Arbor, Michigan, United States

Wayne State University Harper University Hospital

Detroit, Michigan, United States

Rutgers - Robert Wood Johnson Medical School

New Brunswick, New Jersey, United States

New York Medical College at Westchester Medical Center

Valhalla, New York, United States

Children's Hospital Medical Center of Akron

Akron, Ohio, United States

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

Cleveland, Ohio, United States

Nationwide Children's Hospital, Columbus

Columbus, Ohio, United States

Toledo Children's Hospital

Toledo, Ohio, United States

Hershey Medical Center Pennsylvania State University

Hershey, Pennsylvania, United States

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, United States

University of Texas Southwestern / Children's Health

Dallas, Texas, United States

Adult Cystic Fibrosis Center at the University of Utah

Salt Lake City, Utah, United States

University of Wisconsin

Madison, Wisconsin, United States

Froedtert & Medical College of Wisconsin

Milwaukee, Wisconsin, United States

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NCT07450547


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