Back to resultsPhase 1 Study to Assess Safety and Efficacy of ANG003
Primary Purpose
Exocrine Pancreatic Insufficiency
Status
Recruiting
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
ANG003
Sponsored by
About this trial
This is an interventional treatment trial for Exocrine Pancreatic Insufficiency focused on measuring Cystic Fibrosis
Eligibility Criteria
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.
Sites / Locations
- University of Michigan Medical CenterRecruiting
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Arm Type
Experimental
Experimental
Experimental
Experimental
Arm Label
ANG003 Dose Level 1
ANG003 Dose Level 2
ANG003 Dose Level 3
ANG003 Dose Level 4
Arm Description
Single administration starting dose contains lipase, protease and amylase.
Single administration dose contains 2x lipase, 2x protease and 2x amylase starting dose.
Single administration dose contains 4x lipase, 2x protease and 2x amylase starting dose.
Single administration dose contains 6x lipase, 3x protease and 3x amylase starting dose.
Outcomes
Primary Outcome Measures
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.
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.
Secondary Outcome Measures
Fat Absorption
Measured by concentration and percent of plasma fatty acids.
Protein Absorption
Measured by changes in plasma concentration of amino acids.
Carbohydrate Absorption
Changes in glucose (mg/dL) as measured by continuous glucose monitoring.
Full Information
NCT ID
NCT06052293
First Posted
August 9, 2023
Last Updated
September 18, 2023
Sponsor
Anagram Therapeutics, Inc.
1. Study Identification
Unique Protocol Identification Number
NCT06052293
Brief Title
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
2. Study Status
Record Verification Date
September 2023
Overall Recruitment Status
Recruiting
Study Start Date
August 25, 2023 (Actual)
Primary Completion Date
January 31, 2024 (Anticipated)
Study Completion Date
June 24, 2024 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Anagram Therapeutics, Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Exocrine Pancreatic Insufficiency
Keywords
Cystic Fibrosis
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Masking Description
Open-label
Allocation
Randomized
Enrollment
60 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
ANG003 Dose Level 1
Arm Type
Experimental
Arm Description
Single administration starting dose contains lipase, protease and amylase.
Arm Title
ANG003 Dose Level 2
Arm Type
Experimental
Arm Description
Single administration dose contains 2x lipase, 2x protease and 2x amylase starting dose.
Arm Title
ANG003 Dose Level 3
Arm Type
Experimental
Arm Description
Single administration dose contains 4x lipase, 2x protease and 2x amylase starting dose.
Arm Title
ANG003 Dose Level 4
Arm Type
Experimental
Arm Description
Single administration dose contains 6x lipase, 3x protease and 3x amylase starting dose.
Intervention Type
Drug
Intervention Name(s)
ANG003
Other Intervention Name(s)
Dose Level 1, Dose Level 2, Dose Level 3, and Dose Level 4.
Intervention Description
To evaluate four possible combinations of lipase, protease and amylase.
Primary Outcome Measure Information:
Title
Adverse Events (AE), Serious Adverse Events (SAE), and AEs Leading to Study Discontinuation
Description
AE, SAE, and AEs leading to study discontinuation measured by frequency and severity during study.
Time Frame
Assessed through study completion, up to 9 days (Day 1 thru Day 9).
Title
Malabsorption Symptoms
Description
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.
Time Frame
Acute PAGI-SYM is based upon 7-day recall.
Secondary Outcome Measure Information:
Title
Fat Absorption
Description
Measured by concentration and percent of plasma fatty acids.
Time Frame
Assessed through study completion, up to 9 days (Day 1 thru Day 9).
Title
Protein Absorption
Description
Measured by changes in plasma concentration of amino acids.
Time Frame
Assessed through study completion, up to 9 days (Day 1 thru Day 9).
Title
Carbohydrate Absorption
Description
Changes in glucose (mg/dL) as measured by continuous glucose monitoring.
Time Frame
Assessed through study completion, up to 9 days (Day 1 thru Day 9).
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Maria Basco, MD, MPH
Phone
630.863.1761
Email
maria.basco@ssistrategy.com
First Name & Middle Initial & Last Name or Official Title & Degree
Marcie Clarkin, RN,BS
Phone
617.466.3111
Email
mclarkin@anagramtx.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Meghana Sathe, MD
Organizational Affiliation
University of Texas
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of Michigan Medical Center
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Samya Nasr, MD
Phone
734-764-4123
Email
snasr@med.umich.edu
First Name & Middle Initial & Last Name & Degree
Dawn Kruse, RN
Phone
734.615.3266
Email
dmkruse@med.umich.edu
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Phase 1 Study to Assess Safety and Efficacy of ANG003
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