Phase 1 Study to Assess Safety and Efficacy of ANG003
Purpose
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.
Condition
- Exocrine Pancreatic Insufficiency
Eligibility
- Eligible Ages
- Over 18 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- 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
- 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.
Study Design
- Phase
- Phase 1
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Treatment
- Masking
- None (Open Label)
- Masking Description
- Open-label
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental ANG003 Dose Level 1 |
Single administration starting dose contains lipase, protease and amylase. |
|
|
Experimental ANG003 Dose Level 2 |
Single administration dose contains 2x lipase, 2x protease and 2x amylase starting dose. |
|
|
Experimental ANG003 Dose Level 3 |
Single administration dose contains 4x lipase, 2x protease and 2x amylase starting dose. |
|
|
Experimental ANG003 Dose Level 4 |
Single administration dose contains 6x lipase, 3x protease and 3x amylase starting dose. |
|
Recruiting Locations
University of Arkansas for Medical Sciences
Little Rock, Arkansas 72205
Little Rock, Arkansas 72205
Long Beach Memorial Medical Center
Long Beach, California 90806
Long Beach, California 90806
National Jewish Health
Denver, Colorado 80206
Denver, Colorado 80206
Massachusetts General Hospital,
Boston, Massachusetts 02114
Boston, Massachusetts 02114
Boston Children's Hospital
Boston, Massachusetts 02115
Boston, Massachusetts 02115
University of Michigan Medical Center
Ann Arbor, Michigan 48109
Ann Arbor, Michigan 48109
Harper University Hospital / Wayne State University
Detroit, Michigan 48201
Detroit, Michigan 48201
University of Minnesota
Minneapolis, Minnesota 55455
Minneapolis, Minnesota 55455
Albany Medical College
Albany, New York 12208
Albany, New York 12208
New York Medical College at Westchester Medical Center
Valhalla, New York 10595
Valhalla, New York 10595
Akron Childrens Hospital
Akron, Ohio 44308
Akron, Ohio 44308
University Hospitals of Cleveland Medical Center
Cleveland, Ohio 44106
Cleveland, Ohio 44106
Nationwide Children's Hospital
Columbus, Ohio 43205
Columbus, Ohio 43205
University of Texas Southwestern Medical Center
Dallas, Texas 75235
Dallas, Texas 75235
University of Utah
Salt Lake City, Utah 84132
Salt Lake City, Utah 84132
Medical College of Wisconsin
Milwaukee, Wisconsin 53226
Milwaukee, Wisconsin 53226
More Details
- NCT ID
- NCT06052293
- Status
- Recruiting
- Sponsor
- Anagram Therapeutics, Inc.
Detailed Description
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.