Clinical trial NCT06052293: Phase 1 Study to Assess Safety and Efficacy of ANG003
Official title: A Phase 1 Open-Label, Multicenter Study to Assess the Safety and Efficacy of ANG003 in Patients With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Exocrine Pancreatic Insufficiency
Keywords: Cystic Fibrosis
Lead sponsor: Anagram Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2023-09-25
Enrollment: 51
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Adverse Events (AE), Serious Adverse Events (SAE), and AEs Leading to Study Discontinuation
timeFrame: Assessed through study completion, up to 9 days (Day 1 thru Day 9).
measure: Malabsorption Symptoms
timeFrame: Acute PAGI-SYM is based upon 7-day recall.
Secondary outcomes:
measure: Fat Absorption
timeFrame: Assessed through study completion, up to 9 days (Day 1 thru Day 9).
measure: Protein Absorption
timeFrame: Assessed through study completion, up to 9 days (Day 1 thru Day 9).
measure: Carbohydrate Absorption
timeFrame: Assessed through study completion, up to 9 days (Day 1 thru Day 9).
Other outcomes:
Locations:
city: Little Rock
country: United States
facility: University of Arkansas for Medical Sciences
state: Arkansas
city: Long Beach
country: United States
facility: Long Beach Memorial Medical Center
state: California
city: Denver
country: United States
facility: National Jewish Health
state: Colorado
city: Louisville
country: United States
facility: Norton Children's Research Institute affiliated with University of Louisville School of Medicine
state: Kentucky
city: Boston
country: United States
facility: Massachusetts General Hospital,
state: Massachusetts
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan Medical Center
state: Michigan
city: Detroit
country: United States
facility: Harper University Hospital / Wayne State University
state: Michigan
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
city: Lebanon
country: United States
facility: Dartmouth Hitchcock Medical Center
state: New Hampshire
city: Albany
country: United States
facility: Albany Medical College
state: New York
city: Valhalla
country: United States
facility: New York Medical College at Westchester Medical Center
state: New York
city: Akron
country: United States
facility: Akron Childrens Hospital
state: Ohio
city: Cincinnati
country: United States
facility: University of Cincinnati
state: Ohio
city: Cleveland
country: United States
facility: University Hospitals of Cleveland Medical Center
state: Ohio
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
city: Pittsburgh
country: United States
facility: UPMC Children's Hospital of Pittsburgh
state: Pennsylvania
city: Dallas
country: United States
facility: University of Texas Southwestern Medical Center
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah
state: Utah
city: Milwaukee
country: United States
facility: Medical College of Wisconsin
state: Wisconsin
Study officials:
affiliation: University of Texas
name: Meghana Sathe, MD
role: PRINCIPAL_INVESTIGATOR
References: