Phase 2 Study to Assess the Safety and Efficacy of ANG003

NCT07450547 | Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: ANG003-25-2012025-524569-25-00
• Study Type: interventional
• Target: 113
• Locations: 1 country
• Sites: 26

Brief 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.

Conditions

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

Keywords

Cystic Fibrosis (CF); EPI

Outcome Measures

Primary Outcomes (2)

• Adverse events (AEs); Treatment-emergent AEs; Serious AEs; Discontinuation due to AEs; Clinical laboratory values

  Adverse events (AEs); Treatment-emergent AEs; Serious AEs; Discontinuation due to AEs; Clinical laboratory values

  From Visit 1 to Visit 5, anticipated average 80 days

• Coefficient of Fat Absorption (CFA)

  Mean change from baseline CFA (i.e., Period B CFA minus Period A CFA) assessed for ANG003 Dose A, ANG003 Dose B, and Creon amongst subjects with baseline CFA ≥80%.

  Period A (baseline) and Period B 72 hour CFA collection

Secondary Outcomes (5)

• Plasma docosahexaenoic acid (DHA) and eicosapentaenoic acid (EPA) (ug/mL)

  Period A (baseline) and Period B time 0, 1, 2, 4, 6, 8, 10 and 24 hour collections

• Coefficient of Nitrogen Absorption (CNA)

  Period A (baseline) and Period B 72 hour CNA collection

• % of subjects with GI symptoms

  From Visit 2 to Visit 5, anticipated average 59 days

• Cumulative stool weight

  Period A (baseline) and Period B 72 hour collection

• Number of Stools per Day

  Period A (baseline) and Period B 72 hour collection

Other Outcomes (3)

• Correlation between DHA/EPA and CFA

  Period A (baseline) Period B 72 hour CFA and 24 hour DHA/EPA collection

• Coefficient of Fat Absorption (CFA) for subjects with a baseline CFA of 60% to <80% (analyzed separately)

  Period A (baseline) and Period B 72 hour CFA collection

• Mean Period B CFA for all subjects randomized to the Off Enzyme arm with a baseline CFA ≥80%

  Period B 72 hour CFA collection

Study Arms (5)

ANG003 Dose A

EXPERIMENTAL

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

ANG003 Dose B

EXPERIMENTAL

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

Creon

ACTIVE COMPARATOR

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

Off Enzyme

NO INTERVENTION

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.

60% to <80% CFA ANG003 Dose B

OTHER

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

Interventions

ANG003 Dose A DRUG

160,000 u\* lipase, 105,000 u\* protease, and 11,600 u\* amylase per dose (\*units are comparable to United States Pharmacopeiea \[USP\])

ANG003 Dose A

ANG003 Dose B DRUG

240,000 u\* lipase, 105,000 u\* protease, and 11,600 u\* amylase per dose (\*units are comparable to United States Pharmacopeiea \[USP\])

60% to <80% CFA ANG003 Dose B; ANG003 Dose B

Creon DRUG

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.

Creon

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Anagram Therapeutics, Inc.: lead

Study Sites (26)

Long Beach Memorial Medical Center

Long Beach, California, 90806, United States

RECRUITING

Center for Cystic Fibrosis at Keck Medical Center of USC

Los Angeles, California, 90033, United States

NOT YET RECRUITING

National Jewish Health

Denver, Colorado, 80206, United States

NOT YET RECRUITING

University of Florida

Gainesville, Florida, 32610, United States

NOT YET RECRUITING

Central Florida Pulmonary Group

Orlando, Florida, 32803, United States

NOT YET RECRUITING

The Cystic Fibrosis Institute

Glenview, Illinois, 60025, United States

RECRUITING

University of Iowa

Iowa City, Iowa, 52242, United States

NOT YET RECRUITING

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

NOT YET RECRUITING

University of Kentucky

Lexington, Kentucky, 40506, United States

RECRUITING

MaineHealth Pediatric Specialty Care

Portland, Maine, 04102, United States

NOT YET RECRUITING

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

NOT YET RECRUITING

Boston Children's Hospita

Boston, Massachusetts, 02115, United States

NOT YET RECRUITING

University of Michigan, Michigan Medicine

Ann Arbor, Michigan, 48109, United States

RECRUITING

Wayne State University Harper University Hospital

Detroit, Michigan, 48201, United States

NOT YET RECRUITING

Rutgers - Robert Wood Johnson Medical School

New Brunswick, New Jersey, 08903, United States

RECRUITING

New York Medical College at Westchester Medical Center

Valhalla, New York, 10595, United States

NOT YET RECRUITING

Children's Hospital Medical Center of Akron

Akron, Ohio, 44308, United States

RECRUITING

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

Cleveland, Ohio, 44106, United States

NOT YET RECRUITING

Nationwide Children's Hospital, Columbus

Columbus, Ohio, 43205, United States

NOT YET RECRUITING

Toledo Children's Hospital

Toledo, Ohio, 43606, United States

RECRUITING

Hershey Medical Center Pennsylvania State University

Hershey, Pennsylvania, 17033, United States

RECRUITING

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, 15224, United States

NOT YET RECRUITING

University of Texas Southwestern / Children's Health

Dallas, Texas, 75207, United States

RECRUITING

Adult Cystic Fibrosis Center at the University of Utah

Salt Lake City, Utah, 84112, United States

NOT YET RECRUITING

University of Wisconsin

Madison, Wisconsin, 53792, United States

RECRUITING

Froedtert & Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States

NOT YET RECRUITING

MeSH Terms

Conditions

Exocrine Pancreatic Insufficiency; Cystic Fibrosis

Interventions

Pancrelipase

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

Intervention Hierarchy (Ancestors)

Lipase; Carboxylic Ester Hydrolases; Esterases; Hydrolases; Enzymes; Enzymes and Coenzymes; Pancreatic Extracts; Tissue Extracts; Complex Mixtures

Central Study Contacts

Evan Bailey, MD

CONTACT

617-466-3111; medical@anagramtx.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Masking Details: 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.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 9, 2026
• First Posted: March 4, 2026
• Study Start: April 24, 2026
• Primary Completion (Estimated): July 1, 2027
• Study Completion (Estimated): July 1, 2027
• Last Updated: August 4, 2026

Record last verified: 2026-08

Locations

US (26)