Phase 1 Study to Assess Safety and Efficacy of ANG003

Exocrine Pancreatic Insufficiency Clinical Trial

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06052293
• Other study ID #: ANG003-22-101
• Status: Recruiting
• Phase: Phase 1
• Start date: August 25, 2023
• Est. completion date: June 24, 2024

Study information

• Verified date: January 2024
• Source: Anagram Therapeutics, Inc.
• Contact: Maria Basco, MD, MPH
• Phone: 630.863.1761
• Email: maria.basco[@]ssistrategy.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 60
• Est. completion date: June 24, 2024
• Est. primary completion date: January 31, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Exocrine Pancreatic Insufficiency

Intervention

Drug:
• ANG003
To evaluate four possible combinations of lipase, protease and amylase.

Locations

Country	Name	City	State
United States	Akron Childrens Hospital	Akron	Ohio
United States	Albany Medical College	Albany	New York
United States	University of Michigan Medical Center	Ann Arbor	Michigan
United States	Boston Children's Hospital	Boston	Massachusetts
United States	Massachusetts General Hospital,	Boston	Massachusetts
United States	University of Cincinnati	Cincinnati	Ohio
United States	University Hospitals of Cleveland Medical Center	Cleveland	Ohio
United States	Nationwide Children's Hospital	Columbus	Ohio
United States	University of Texas Southwestern Medical Center	Dallas	Texas
United States	National Jewish Health	Denver	Colorado
United States	Harper University Hospital / Wayne State University	Detroit	Michigan
United States	Dartmouth Hitchcock Medical Center	Lebanon	New Hampshire
United States	University of Arkansas for Medical Sciences	Little Rock	Arkansas
United States	Long Beach Memorial Medical Center	Long Beach	California
United States	Norton Children's Research Institute affiliated with University of Louisville School of Medicine	Louisville	Kentucky
United States	Medical College of Wisconsin	Milwaukee	Wisconsin
United States	University of Minnesota	Minneapolis	Minnesota
United States	UPMC Children's Hospital of Pittsburgh	Pittsburgh	Pennsylvania
United States	University of Utah	Salt Lake City	Utah
United States	New York Medical College at Westchester Medical Center	Valhalla	New York

Sponsors (1)

Lead Sponsor	Collaborator
Anagram Therapeutics, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Adverse Events (AE), Serious Adverse Events (SAE), and AEs Leading to Study Discontinuation	AE, SAE, and AEs leading to study discontinuation measured by frequency and severity during study.	Assessed through study completion, up to 9 days (Day 1 thru Day 9).
Primary	Malabsorption Symptoms	Severity of malabsorption symptoms (e.g., abdominal discomfort or pain, bloating, heartburn, regurgitation or reflux, retching, stomach fullness, and vomiting) by patient reported outcome scale (0=None; 1=Very Mild; 2=Mild; 3=Moderate; 4=Severe; 5=Very Severe). Measured by 20 questions contained in Acute Patient Assessment of Gastrointestinal Symptoms (Acute PAGI-SYM) questionnaire.	Acute PAGI-SYM is based upon 7-day recall.
Secondary	Fat Absorption	Measured by concentration and percent of plasma fatty acids.	Assessed through study completion, up to 9 days (Day 1 thru Day 9).
Secondary	Protein Absorption	Measured by changes in plasma concentration of amino acids.	Assessed through study completion, up to 9 days (Day 1 thru Day 9).
Secondary	Carbohydrate Absorption	Changes in glucose (mg/dL) as measured by continuous glucose monitoring.	Assessed through study completion, up to 9 days (Day 1 thru Day 9).