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| ID | Type | Description | Link |
|---|---|---|---|
| 2025-524569-25-00 | EU Trial (CTIS) Number |
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In this study, ANG003, a pancreatic enzyme replacement therapy (PERT; commonly called "enzymes"), is being investigated as a potential treatment for exocrine pancreatic insufficiency (EPI). People with EPI due to Cystic Fibrosis (CF) may be eligible to participate in this study. The primary objective of this study is to evaluate the safety of ANG003 and see if it works as well compared to Creon, an approved PERT.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ANG003 Dose A | Experimental | All subjects will be acclimated to Creon for 14-days and then will undergo a baseline CFA analysis. If baseline CFA is >/= 80%, the subject may be randomized to ANG003 Dose A for a 21-day acclimation and subsequent CFA analysis. |
|
| ANG003 Dose B | Experimental | All subjects will be acclimated to Creon for 14-days and then will undergo a baseline CFA analysis. If baseline CFA is >/= 80%, the subject may be randomized to ANG003 Dose B for a 21-day acclimation and subsequent CFA analysis. |
|
| Creon | Active Comparator | All subjects will be acclimated to Creon for 14-days and then will undergo a baseline CFA analysis. If baseline CFA is >/= 80%, the subject may be randomized to Creon for an additional 21-days and subsequent CFA analysis. |
|
| Off Enzyme | No Intervention | All subjects will be acclimated to Creon for 14-days and then will undergo a baseline CFA analysis. If baseline CFA is >/= 80%, the subject may be randomized to the off enzyme arm. Subjects will receive Creon for an additional 21-day and the subsequent CFA analysis will be off enzyme. Total time off enzyme will be 4-7 days. | |
| 60% to <80% CFA ANG003 Dose B | Other |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ANG003 Dose A | Drug | 160,000 u* lipase, 105,000 u* protease, and 11,600 u* amylase per dose (*units are comparable to United States Pharmacopeiea [USP]) |
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| Measure | Description | Time Frame |
|---|---|---|
| Adverse events (AEs); Treatment-emergent AEs; Serious AEs; Discontinuation due to AEs; Clinical laboratory values | Adverse events (AEs); Treatment-emergent AEs; Serious AEs; Discontinuation due to AEs; Clinical laboratory values | From Visit 1 to Visit 5, anticipated average 80 days |
| Coefficient of Fat Absorption (CFA) | Mean change from baseline CFA (i.e., Period B CFA minus Period A CFA) assessed for ANG003 Dose A, ANG003 Dose B, and Creon amongst subjects with baseline CFA ≥80%. | Period A (baseline) and Period B 72 hour CFA collection |
| Measure | Description | Time Frame |
|---|---|---|
| Plasma docosahexaenoic acid (DHA) and eicosapentaenoic acid (EPA) (ug/mL) | Mean change from baseline for each treatment group amongst subjects with baseline CFA ≥80%. | Period A (baseline) and Period B time 0, 1, 2, 4, 6, 8, 10 and 24 hour collections |
| Coefficient of Nitrogen Absorption (CNA) |
| Measure | Description | Time Frame |
|---|---|---|
| Correlation between DHA/EPA and CFA | Mean change from baseline for each treatment group with a baseline CFA ≥80% | Period A (baseline) Period B 72 hour CFA and 24 hour DHA/EPA collection |
| Coefficient of Fat Absorption (CFA) for subjects with a baseline CFA of 60% to <80% (analyzed separately) |
Inclusion Criteria:
Male and female subjects aged ≥12 years from the date of informed consent in the US and aged ≥18 years in the EU
Confirmed and documented diagnosis of CF
Diagnosed with severe EPI as defined by a fecal elastase ≤50 μg/g stool measured at Screening by a central laboratory.
Subject has controlled EPI and taking a stable dose of pancreatic enzyme replacement therapy (PERT) for 90 days prior to Screening.
Adequate nutritional status measured by body mass index (BMI) defined by:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Evan Bailey, MD | Contact | 617-466-3111 | medical@anagramtx.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Long Beach Memorial Medical Center | Recruiting | Long Beach | California | 90806 | United States |
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A centralized, blinded safety assessor will be responsible for conducting independent reviews of key safety data to ensure objective and consistent evaluation across all study sites.
All subjects will be acclimated to Creon for 14-days and then will undergo a baseline CFA analysis. Participants with a Period A CFA result of 60% to <80% will be assigned to ANG003 Dose B. |
|
| ANG003 Dose B | Drug | 240,000 u* lipase, 105,000 u* protease, and 11,600 u* amylase per dose (*units are comparable to United States Pharmacopeiea [USP]) |
|
| Creon | Drug | The commercially available pPERT Creon will be the active comparator in the study. Subjects will be instructed to take Creon according to the package insert and PI direction. |
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Mean change from baseline for each treatment group amongst subjects with baseline CFA ≥80%. |
| Period A (baseline) and Period B 72 hour CNA collection |
| % of subjects with GI symptoms | Mean change from baseline for each treatment group amongst subjects with baseline CFA ≥80%. | From Visit 2 to Visit 5, anticipated average 59 days |
| Cumulative stool weight | Mean change from baseline for each treatment group amongst subjects with baseline CFA ≥80%. | Period A (baseline) and Period B 72 hour collection |
| Number of Stools per Day | Mean change from baseline for each treatment group amongst subjects with baseline CFA ≥80%. | Period A (baseline) and Period B 72 hour collection |
Mean change from baseline for each treatment group with a baseline CFA 60% to <80% |
| Period A (baseline) and Period B 72 hour CFA collection |
| Mean Period B CFA for all subjects randomized to the Off Enzyme arm with a baseline CFA ≥80% | Period B CFA Off Enzyme in subjects with a baseline CFA ≥80% | Period B 72 hour CFA collection |
| Center for Cystic Fibrosis at Keck Medical Center of USC | Not yet recruiting | Los Angeles | California | 90033 | United States |
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| National Jewish Health | Not yet recruiting | Denver | Colorado | 80206 | United States |
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| University of Florida | Not yet recruiting | Gainesville | Florida | 32610 | United States |
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| Central Florida Pulmonary Group | Not yet recruiting | Orlando | Florida | 32803 | United States |
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| The Cystic Fibrosis Institute | Recruiting | Glenview | Illinois | 60025 | United States |
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| University of Iowa | Not yet recruiting | Iowa City | Iowa | 52242 | United States |
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| University of Kansas Medical Center | Not yet recruiting | Kansas City | Kansas | 66160 | United States |
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| University of Kentucky | Not yet recruiting | Lexington | Kentucky | 40506 | United States |
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| MaineHealth Pediatric Specialty Care | Not yet recruiting | Portland | Maine | 04102 | United States |
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| Massachusetts General Hospital | Not yet recruiting | Boston | Massachusetts | 02114 | United States |
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| Boston Children's Hospita | Not yet recruiting | Boston | Massachusetts | 02115 | United States |
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| University of Michigan, Michigan Medicine | Recruiting | Ann Arbor | Michigan | 48109 | United States |
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| Wayne State University Harper University Hospital | Not yet recruiting | Detroit | Michigan | 48201 | United States |
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| Rutgers - Robert Wood Johnson Medical School | Not yet recruiting | New Brunswick | New Jersey | 08903 | United States |
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| New York Medical College at Westchester Medical Center | Not yet recruiting | Valhalla | New York | 10595 | United States |
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| Children's Hospital Medical Center of Akron | Not yet recruiting | Akron | Ohio | 44308 | United States |
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| Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center | Not yet recruiting | Cleveland | Ohio | 44106 | United States |
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| Nationwide Children's Hospital, Columbus | Not yet recruiting | Columbus | Ohio | 43205 | United States |
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| Toledo Children's Hospital | Recruiting | Toledo | Ohio | 43606 | United States |
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| Hershey Medical Center Pennsylvania State University | Recruiting | Hershey | Pennsylvania | 17033 | United States |
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| University of Pittsburgh Medical Center | Not yet recruiting | Pittsburgh | Pennsylvania | 15224 | United States |
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| University of Texas Southwestern / Children's Health | Not yet recruiting | Dallas | Texas | 75207 | United States |
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| Adult Cystic Fibrosis Center at the University of Utah | Not yet recruiting | Salt Lake City | Utah | 84112 | United States |
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| University of Wisconsin | Recruiting | Madison | Wisconsin | 53792 | United States |
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| Froedtert & Medical College of Wisconsin | Not yet recruiting | Milwaukee | Wisconsin | 53226 | United States |
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| ID | Term |
|---|---|
| D010188 | Exocrine Pancreatic Insufficiency |
| D003550 | Cystic Fibrosis |
| ID | Term |
|---|---|
| D010182 | Pancreatic Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007232 | Infant, Newborn, Diseases |
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| ID | Term |
|---|---|
| D020799 | Pancrelipase |
| ID | Term |
|---|---|
| D008049 | Lipase |
| D002265 | Carboxylic Ester Hydrolases |
| D004950 | Esterases |
| D006867 | Hydrolases |
| D004798 | Enzymes |
| D045762 | Enzymes and Coenzymes |
| D010184 | Pancreatic Extracts |
| D014020 | Tissue Extracts |
| D045424 | Complex Mixtures |
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