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NCT Number: NCT06052293

Phase 1 Study to Assess Safety and Efficacy of ANG003

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.

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Key information

Conditions

Exocrine Pancreatic Insufficiency Congenital, Hereditary, and Neonatal Diseases and Abnormalities Cystic Fibrosis Digestive System Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases Lung Diseases Pancreatic Diseases Respiratory Tract Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

University of Arkansas for Medical Sciences, Little Rock, Arkansas, United States

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Male and female subjects 18 years of age or older.
  • 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.
  • Documented history of fecal elastase <100 µg/g stool.
  • EPI clinically controlled with minimal clinical symptoms and on a stable dose of PERT for 90 days before screening as determined by the Investigator.
  • 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.
  • Involuntary loss of ≥10% of usual body weight within last 6 months or involuntary loss of >5% of body weight within 1 month.
  • Requires use of naso-gastric, J-tube, G-tube, and/or enteral feeding for the study duration.
  • CF pulmonary exacerbation within 30 days prior to the Baseline SACT Period (Visit 2).
  • Subjects who cannot discontinue omega-3 supplements >500 mg of DHA and EPA daily.
  • Subjects unable to tolerate missing a dose of PERT.

Treatment and study plan

ANG003

Drug

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

Other names: Dose Level 1, Dose Level 2, Dose Level 3, and Dose Level 4.

Primary outcomes

  1. Number of Participants With Adverse Events (AE), Serious Adverse Events (SAE) and AEs Leading to Study Discontinuation

    Time frame: Assessed through study completion, up to 9 days (Day 1 thru Day 9).

    AE, SAEs and AEs leading to study discontinuation measured by number of participants during study.

Other outcomes

  1. Carbohydrate Absorption

    Time frame: Assessed through study completion, up to 9 days (Day 1 thru Day 9).

    Changes in glucose (mg/dL) as measured by continuous glucose monitoring.

  2. Protein Absorption

    Time frame: Assessed through study completion, up to 9 days (Day 1 thru Day 9).

    Measured by changes in plasma concentration of amino acids.

  3. Fat Absorption Assessed as Cmax of DHA+EPA

    Time frame: DHA+EPA measured at baseline t0, 1h, 2h, 4h, 6h, 8h, 10-12h, and 24h post on Baseline SACT (Day 1) and ANG003 SACT (Day 5)

    Measured by concentration of plasma fatty acids.

  4. Fat Absorption Assessed as Area Under the Curve (AUC-24hr) of Plamsa Docosahexaenoic Acid (DHA) and Eicosapentaenoic Acid (EPA)

    Time frame: DHA+EPA measured at baseline t0, 1h, 2h, 4h, 6h, 8h, 10-12h, and 24h post on Baseline SACT (Day 1) and ANG003 SACT (Day 5)

    Measured by concentration of plasma fatty acids.

  5. Malabsorption Symptoms

    Time frame: Acute PAGI-SYM is based upon 7-day recall.

    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.

Sponsors and collaborators

Lead sponsor

Anagram Therapeutics, Inc.

Industry

Registry information

Official study 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

Important dates

Study start
2023
Primary completion
2024
Study completion
2024
First posted
Sep 25, 2023
Registry last updated
Apr 27, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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