Phase 1 Study to Assess Safety and Efficacy of ANG003

Anagram Therapeutics

Status and phase: Completed; Phase 1

Conditions: Exocrine Pancreatic Insufficiency

Treatments: Drug: ANG003

Study type: Interventional

Funder types: Industry

Identifiers

NCT06052293

ANG003-22-101

Details and patient eligibility

About

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.

Full 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.

Enrollment: 51 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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51 participants in 4 patient groups

ANG003 Dose Level 1

Experimental group

Description:

Single administration starting dose contains lipase, protease and amylase.

Treatment:

Drug: ANG003

ANG003 Dose Level 2

Experimental group

Description:

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

Treatment:

Drug: ANG003

ANG003 Dose Level 3

Experimental group

Description:

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

Treatment:

Drug: ANG003

ANG003 Dose Level 4

Experimental group

Description:

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

Treatment:

Drug: ANG003