Phase 1 Study to Assess Safety and Efficacy of ANG003

NCT06052293 | Completed Phase 1 Results DMC FDA Drug

• 1 other identifier: ANG003-22-101
• Study Type: interventional
• Target: 57
• Locations: 1 country
• Sites: 20

Brief Summary

Randomized, parallel, active-treatment Phase 1 study of a single dose of orally administered ANG003 with a test meal in adult subjects with cystic fibrosis-related exocrine pancreatic insufficiency. The study's overall objectives are to evaluate the safety, tolerability and effect of four dose levels of ANG003.

Conditions

Exocrine Pancreatic Insufficiency

Keywords

Cystic Fibrosis

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Adverse Events (AE), Serious Adverse Events (SAE) and AEs Leading to Study Discontinuation

  AE, SAEs and AEs leading to study discontinuation measured by number of participants during study.

  Assessed through study completion, up to 9 days (Day 1 thru Day 9).

Other Outcomes (5)

• Carbohydrate Absorption

  Assessed through study completion, up to 9 days (Day 1 thru Day 9).

• Protein Absorption

  Assessed through study completion, up to 9 days (Day 1 thru Day 9).

• Fat Absorption Assessed as Cmax of DHA+EPA

  DHA+EPA measured at baseline t0, 1h, 2h, 4h, 6h, 8h, 10-12h, and 24h post on Baseline SACT (Day 1) and ANG003 SACT (Day 5)

Study Arms (4)

ANG003 Dose Level 1

EXPERIMENTAL

Single administration dose contains 20 mg lipase, 25 mg protease and 40 mg amylase.

Drug: ANG003

ANG003 Dose Level 2

EXPERIMENTAL

Single administration dose contains 40 mg lipase, 50 mg protease and 80 mg amylase.

Drug: ANG003

ANG003 Dose Level 3

EXPERIMENTAL

Single administration dose contains 80 mg lipase, 50 mg protease and 80 mg amylase.

Drug: ANG003

ANG003 Dose Level 4

EXPERIMENTAL

Single administration dose contains 120 mg lipase, 75 mg protease and 120 mg amylase.

Drug: ANG003

Interventions

ANG003 DRUG

To evaluate four possible combinations of lipase, protease and amylase.

Also known as: Dose Level 1, Dose Level 2, Dose Level 3, and Dose Level 4.

ANG003 Dose Level 1; ANG003 Dose Level 2; ANG003 Dose Level 3; ANG003 Dose Level 4

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male and female subjects 18 years of age or older.
• Confirmed diagnosis of CF defined as: a) CF signs and symptoms AND b) Two CF-causing mutations on genetic testing or sweat chloride \>60 mEq/L.
• Documented history of fecal elastase \<100 µg/g stool.
• EPI clinically controlled with minimal clinical symptoms and on a stable dose of PERT for 90 days before screening as determined by the Investigator.
• Adequate nutritional status measured by body mass index ≥20kg/m2 for adult subjects.

You may not qualify if:

• Subjects with diabetes mellitus who are unable to refrain from short-acting and rapid-acting insulin on Days 1 and 5 for a daily total of 6 hours.
• Involuntary loss of ≥10% of usual body weight within last 6 months or involuntary loss of \>5% of body weight within 1 month.
• Requires use of naso-gastric, J-tube, G-tube, and/or enteral feeding for the study duration.
• CF pulmonary exacerbation within 30 days prior to the Baseline SACT Period (Visit 2).
• Subjects who cannot discontinue omega-3 supplements \>500 mg of DHA and EPA daily.
• Subjects unable to tolerate missing a dose of PERT.

Sponsors & Collaborators

• Anagram Therapeutics, Inc.: lead

Study Sites (20)

University of Arkansas for Medical Sciences

Little Rock, Arkansas, 72205, United States

Location

Long Beach Memorial Medical Center

Long Beach, California, 90806, United States

Location

National Jewish Health

Denver, Colorado, 80206, United States

Location

Norton Children's Research Institute affiliated with University of Louisville School of Medicine

Louisville, Kentucky, 40202, United States

Location

Massachusetts General Hospital,

Boston, Massachusetts, 02114, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

University of Michigan Medical Center

Ann Arbor, Michigan, 48109, United States

Location

Harper University Hospital / Wayne State University

Detroit, Michigan, 48201, United States

Location

University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

Dartmouth Hitchcock Medical Center

Lebanon, New Hampshire, 03756, United States

Location

Albany Medical College

Albany, New York, 12208, United States

Location

New York Medical College at Westchester Medical Center

Valhalla, New York, 10595, United States

Location

Akron Childrens Hospital

Akron, Ohio, 44308, United States

Location

University of Cincinnati

Cincinnati, Ohio, 45267, United States

Location

University Hospitals of Cleveland Medical Center

Cleveland, Ohio, 44106, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

UPMC Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, 15224, United States

Location

University of Texas Southwestern Medical Center

Dallas, Texas, 75235, United States

Location

University of Utah

Salt Lake City, Utah, 84132, United States

Location

Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

MeSH Terms

Conditions

Exocrine Pancreatic Insufficiency; Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Results Point of Contact

• Title: Marcie Clarkin
• Organization: Anagram Therapeutics, Inc.

mclarkin@anagramtx.com

6174663111

Study Officials

• Meghana Sathe, MD

  University of Texas

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Masking Details: Open-label
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 9, 2023
• First Posted: September 25, 2023
• Study Start: August 25, 2023
• Primary Completion: July 8, 2024
• Study Completion: July 8, 2024
• Last Updated: April 27, 2026
• Results First Posted: April 27, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (20)