Phase 1 Study to Assess Safety and Efficacy of ANG003
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| ClinicalTrials.gov Identifier: NCT06052293 |
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Recruitment Status :
Recruiting
First Posted : September 25, 2023
Last Update Posted : January 5, 2024
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Sponsor:
Anagram Therapeutics, Inc.
Information provided by (Responsible Party):
Anagram Therapeutics, Inc.
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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.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Exocrine Pancreatic Insufficiency | Drug: ANG003 | Phase 1 |
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.
| Study Type : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 60 participants |
| Allocation: | Randomized |
| Intervention Model: | Parallel Assignment |
| Masking: | None (Open Label) |
| Masking Description: | Open-label |
| Primary Purpose: | Treatment |
| 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 |
| Actual Study Start Date : | August 25, 2023 |
| Estimated Primary Completion Date : | January 31, 2024 |
| Estimated Study Completion Date : | June 24, 2024 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Cystic fibrosis
MedlinePlus related topics:
Cystic Fibrosis
| Arm | Intervention/treatment |
|---|---|
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Experimental: ANG003 Dose Level 1
Single administration starting dose contains lipase, protease and amylase.
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Drug: ANG003
To evaluate four possible combinations of lipase, protease and amylase.
Other Name: Dose Level 1, Dose Level 2, Dose Level 3, and Dose Level 4. |
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Experimental: ANG003 Dose Level 2
Single administration dose contains 2x lipase, 2x protease and 2x amylase starting dose.
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Drug: ANG003
To evaluate four possible combinations of lipase, protease and amylase.
Other Name: Dose Level 1, Dose Level 2, Dose Level 3, and Dose Level 4. |
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Experimental: ANG003 Dose Level 3
Single administration dose contains 4x lipase, 2x protease and 2x amylase starting dose.
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Drug: ANG003
To evaluate four possible combinations of lipase, protease and amylase.
Other Name: Dose Level 1, Dose Level 2, Dose Level 3, and Dose Level 4. |
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Experimental: ANG003 Dose Level 4
Single administration dose contains 6x lipase, 3x protease and 3x amylase starting dose.
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Drug: ANG003
To evaluate four possible combinations of lipase, protease and amylase.
Other Name: Dose Level 1, Dose Level 2, Dose Level 3, and Dose Level 4. |
Primary Outcome Measures :
- 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, SAE, and AEs leading to study discontinuation measured by frequency and severity during study.
- 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.
Secondary Outcome Measures :
- Fat Absorption [ Time Frame: Assessed through study completion, up to 9 days (Day 1 thru Day 9). ]Measured by concentration and percent of plasma fatty acids.
- 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.
- 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.
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| Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
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.
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT06052293
Contacts
| Contact: Maria Basco, MD, MPH | 630.863.1761 | maria.basco@ssistrategy.com | |
| Contact: Marcie Clarkin, RN,BS | 617.466.3111 | mclarkin@anagramtx.com |
Locations
Show 20 study locations
Sponsors and Collaborators
Anagram Therapeutics, Inc.
Investigators
| Principal Investigator: | Meghana Sathe, MD | University of Texas |
| Responsible Party: | Anagram Therapeutics, Inc. |
| ClinicalTrials.gov Identifier: | NCT06052293 |
| Other Study ID Numbers: |
ANG003-22-101 |
| First Posted: | September 25, 2023 Key Record Dates |
| Last Update Posted: | January 5, 2024 |
| Last Verified: | January 2024 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | No |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
Keywords provided by Anagram Therapeutics, Inc.:
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Cystic Fibrosis |
Additional relevant MeSH terms:
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Exocrine Pancreatic Insufficiency Pancreatic Diseases Digestive System Diseases |