Phase 1 Study to Assess Safety and Efficacy of ANG003

NCT ID: NCT06052293

Last Updated: 2024-08-13

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

51 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-08-25

Study Completion Date

2024-07-08

Brief Summary

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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.

Detailed Description

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The Phase 1 study was designed to compare a 24h Baseline Substrate Absorption Challenge Test (SACT) period with no enzymes to a 24h ANG003 SACT period with enzymes. Eligible subjects will be randomly assigned with equal allocation to one of four active dose levels of lipase, protease and amylase. Approximately 48 to 60 eligible subjects are planned to be enrolled in the study with 12 to 15 subjects assigned to each dose level from up to 21 investigational sites.

Conditions

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Exocrine Pancreatic Insufficiency

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Open-label

Study Groups

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ANG003 Dose Level 1

Single administration starting dose contains lipase, protease and amylase.

Group Type EXPERIMENTAL

ANG003

Intervention Type DRUG

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

ANG003 Dose Level 2

Single administration dose contains 2x lipase, 2x protease and 2x amylase starting dose.

Group Type EXPERIMENTAL

ANG003

Intervention Type DRUG

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

ANG003 Dose Level 3

Single administration dose contains 4x lipase, 2x protease and 2x amylase starting dose.

Group Type EXPERIMENTAL

ANG003

Intervention Type DRUG

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

ANG003 Dose Level 4

Single administration dose contains 6x lipase, 3x protease and 3x amylase starting dose.

Group Type EXPERIMENTAL

ANG003

Intervention Type DRUG

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

Interventions

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ANG003

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

Intervention Type DRUG

Other Intervention Names

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Dose Level 1, Dose Level 2, Dose Level 3, and Dose Level 4.

Eligibility Criteria

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Inclusion Criteria

1. Male and female subjects 18 years of age or older.
2. 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.
3. Documented history of fecal elastase \<100 µg/g stool.
4. EPI clinically controlled with minimal clinical symptoms and on a stable dose of PERT for 90 days before screening as determined by the Investigator.
5. Adequate nutritional status measured by body mass index ≥20kg/m2 for adult subjects.

Exclusion Criteria

1. 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.
2. Involuntary loss of ≥10% of usual body weight within last 6 months or involuntary loss of \>5% of body weight within 1 month.
3. Requires use of naso-gastric, J-tube, G-tube, and/or enteral feeding for the study duration.
4. CF pulmonary exacerbation within 30 days prior to the Baseline SACT Period (Visit 2).
5. Subjects who cannot discontinue omega-3 supplements \>500 mg of DHA and EPA daily.
6. Subjects unable to tolerate missing a dose of PERT.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Anagram Therapeutics, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Meghana Sathe, MD

Role: PRINCIPAL_INVESTIGATOR

University of Texas

Locations

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University of Arkansas for Medical Sciences

Little Rock, Arkansas, United States

Site Status

Long Beach Memorial Medical Center

Long Beach, California, United States

Site Status

National Jewish Health

Denver, Colorado, United States

Site Status

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

Louisville, Kentucky, United States

Site Status

Massachusetts General Hospital,

Boston, Massachusetts, United States

Site Status

Boston Children's Hospital

Boston, Massachusetts, United States

Site Status

University of Michigan Medical Center

Ann Arbor, Michigan, United States

Site Status

Harper University Hospital / Wayne State University

Detroit, Michigan, United States

Site Status

University of Minnesota

Minneapolis, Minnesota, United States

Site Status

Dartmouth Hitchcock Medical Center

Lebanon, New Hampshire, United States

Site Status

Albany Medical College

Albany, New York, United States

Site Status

New York Medical College at Westchester Medical Center

Valhalla, New York, United States

Site Status

Akron Childrens Hospital

Akron, Ohio, United States

Site Status

University of Cincinnati

Cincinnati, Ohio, United States

Site Status

University Hospitals of Cleveland Medical Center

Cleveland, Ohio, United States

Site Status

Nationwide Children's Hospital

Columbus, Ohio, United States

Site Status

UPMC Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States

Site Status

University of Texas Southwestern Medical Center

Dallas, Texas, United States

Site Status

University of Utah

Salt Lake City, Utah, United States

Site Status

Medical College of Wisconsin

Milwaukee, Wisconsin, United States

Site Status

Countries

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United States

Other Identifiers

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ANG003-22-101

Identifier Type: -

Identifier Source: org_study_id

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