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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ANG003 Dose Level 1 | Experimental | Single administration dose contains 20 mg lipase, 25 mg protease and 40 mg amylase. |
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| ANG003 Dose Level 2 | Experimental | Single administration dose contains 40 mg lipase, 50 mg protease and 80 mg amylase. |
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| ANG003 Dose Level 3 | Experimental | Single administration dose contains 80 mg lipase, 50 mg protease and 80 mg amylase. |
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| ANG003 Dose Level 4 | Experimental | Single administration dose contains 120 mg lipase, 75 mg protease and 120 mg amylase. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ANG003 | Drug | To evaluate four possible combinations of lipase, protease and amylase. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Adverse Events (AE), Serious Adverse Events (SAE) and AEs Leading to Study Discontinuation | AE, SAEs and AEs leading to study discontinuation measured by number of participants during study. | Assessed through study completion, up to 9 days (Day 1 thru Day 9). |
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| Measure | Description | Time Frame |
|---|---|---|
| Carbohydrate Absorption | Changes in glucose (mg/dL) as measured by continuous glucose monitoring. | Assessed through study completion, up to 9 days (Day 1 thru Day 9). |
| Protein Absorption | Measured by changes in plasma concentration of amino acids. |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Meghana Sathe, MD | University of Texas | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Arkansas for Medical Sciences | Little Rock | Arkansas | 72205 | United States | ||
| Long Beach Memorial Medical Center |
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| ID | Title | Description |
|---|---|---|
| FG000 | ANG003 Dose Level 1 | Single administration dose contains 20mg lipase, 25 mg protease and 40 mg amylase. ANG003: To evaluate four possible combinations of lipase, protease and amylase. |
| FG001 | ANG003 Dose Level 2 |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Randomized to ANG003 (Day -2) |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Mar 22, 2024 | Jul 8, 2025 |
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Open-label
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|
| Assessed through study completion, up to 9 days (Day 1 thru Day 9). |
| Fat Absorption Assessed as Cmax of DHA+EPA | Measured by concentration of plasma fatty acids. | 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) |
| Fat Absorption Assessed as Area Under the Curve (AUC-24hr) of Plamsa Docosahexaenoic Acid (DHA) and Eicosapentaenoic Acid (EPA) | Measured by concentration of plasma fatty acids. | 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) |
| 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. | Acute PAGI-SYM is based upon 7-day recall. |
| Long Beach |
| California |
| 90806 |
| United States |
| National Jewish Health | Denver | Colorado | 80206 | United States |
| Norton Children's Research Institute affiliated with University of Louisville School of Medicine | Louisville | Kentucky | 40202 | United States |
| Massachusetts General Hospital, | Boston | Massachusetts | 02114 | United States |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | United States |
| University of Michigan Medical Center | Ann Arbor | Michigan | 48109 | United States |
| Harper University Hospital / Wayne State University | Detroit | Michigan | 48201 | United States |
| University of Minnesota | Minneapolis | Minnesota | 55455 | United States |
| Dartmouth Hitchcock Medical Center | Lebanon | New Hampshire | 03756 | United States |
| Albany Medical College | Albany | New York | 12208 | United States |
| New York Medical College at Westchester Medical Center | Valhalla | New York | 10595 | United States |
| Akron Childrens Hospital | Akron | Ohio | 44308 | United States |
| University of Cincinnati | Cincinnati | Ohio | 45267 | United States |
| University Hospitals of Cleveland Medical Center | Cleveland | Ohio | 44106 | United States |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | United States |
| UPMC Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania | 15224 | United States |
| University of Texas Southwestern Medical Center | Dallas | Texas | 75235 | United States |
| University of Utah | Salt Lake City | Utah | 84132 | United States |
| Medical College of Wisconsin | Milwaukee | Wisconsin | 53226 | United States |
Single administration dose contains 40 mg lipase, 50 mg protease and 80 mg amylase.
ANG003: To evaluate four possible combinations of lipase, protease and amylase.
| FG002 | ANG003 Dose Level 3 | Single administration dose contains 80 mg lipase, 50 mg protease and 80 mg amylase. ANG003: To evaluate four possible combinations of lipase, protease and amylase. |
| FG003 | ANG003 Dose Level 4 | Single administration dose contains 120 mg lipase, 75 mg protease and 120 mg amylase. ANG003: To evaluate four possible combinations of lipase, protease and amylase. |
| COMPLETED |
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| NOT COMPLETED |
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| Baseline SACT (Day 1) |
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| ANG003 SACT (Day 5) |
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| ID | Title | Description |
|---|---|---|
| BG000 | ANG003 Dose Level 1 | Single administration dose contains 20 mg lipase, 25 mg protease and 40 mg amylase. ANG003: To evaluate four possible combinations of lipase, protease and amylase. |
| BG001 | ANG003 Dose Level 2 | Single administration dose contains 40 mg lipase, 50 mg protease and 80 mg amylase. ANG003: To evaluate four possible combinations of lipase, protease and amylase. |
| BG002 | ANG003 Dose Level 3 | Single administration dose contains 80 mg lipase, 50 mg protease and 80 mg amylase. ANG003: To evaluate four possible combinations of lipase, protease and amylase. |
| BG003 | ANG003 Dose Level 4 | Single administration dose contains 120 mg lipase, 75 mg protease and 120 mg amylase. ANG003: To evaluate four possible combinations of lipase, protease and amylase. |
| BG004 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Full Range | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Cystic fibrosis transmembrane conductance regulator (CFTR) Modulator Therapy | Count of Participants | Participants |
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| Diabetes | Count of Participants | Participants |
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| Body Mass Index (BMI) | Mean | Full Range | kg/m^2 |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number of Participants With Adverse Events (AE), Serious Adverse Events (SAE) and AEs Leading to Study Discontinuation | AE, SAEs and AEs leading to study discontinuation measured by number of participants during study. | Posted | Number | Participants | Assessed through study completion, up to 9 days (Day 1 thru Day 9). |
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| Other Pre-specified | Carbohydrate Absorption | Changes in glucose (mg/dL) as measured by continuous glucose monitoring. | Not Posted | Assessed through study completion, up to 9 days (Day 1 thru Day 9). | Participants | ||||||||||||||||||||||||||||||||||||||||
| Other Pre-specified | Protein Absorption | Measured by changes in plasma concentration of amino acids. | Not Posted | Assessed through study completion, up to 9 days (Day 1 thru Day 9). | Participants | ||||||||||||||||||||||||||||||||||||||||
| Other Pre-specified | Fat Absorption Assessed as Cmax of DHA+EPA | Measured by concentration of plasma fatty acids. | DHA+EPA Cmax ANG003 SACT (Day 5) - Baseline SACT (Day 1) | Posted | Mean | Standard Deviation | ug/mL | 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) |
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| Other Pre-specified | Fat Absorption Assessed as Area Under the Curve (AUC-24hr) of Plamsa Docosahexaenoic Acid (DHA) and Eicosapentaenoic Acid (EPA) | Measured by concentration of plasma fatty acids. | DHA+EPA Positive Peak AUC-24hr ANG003 SACT (Day 5) - Baseline SACT (Day 1) | Posted | Mean | Standard Deviation | ug*hr/mL | 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) |
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| Other Pre-specified | 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. | Not Posted | Acute PAGI-SYM is based upon 7-day recall. | Participants |
Reporting of AEs occurred from Day 1 to Day 9/EOS
Subjects were required to be off their Standard of Care (SoC) Pancreatic Enzyme Replacement Therapy (PERT) regimen for 24 hours prior to the start of each Substrate Absorption Challenge Test (SACT) period (Day -1 and 4) and for an additional 24 hours (Day 1 and 5) during each SACT period for a total of 48 consecutive hours Off SoC PERT. Subjects received one dose of ANG003 on Day 5.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | ANG003 Dose Level 1 | Single administration dose contains 20 mg lipase, 25 mg protease and 40 mg amylase. ANG003: To evaluate four possible combinations of lipase, protease and amylase. | 0 | 13 | 0 | 13 | 8 | 13 |
| EG001 | ANG003 Dose Level 2 | Single administration dose contains 40 mg lipase, 50 mg protease and 80 mg amylase. ANG003: To evaluate four possible combinations of lipase, protease and amylase. | 0 | 13 | 0 | 13 | 8 | 13 |
| EG002 | ANG003 Dose Level 3 | Single administration dose contains 80 mg lipase, 50 mg protease and 80 mg amylase. ANG003: To evaluate four possible combinations of lipase, protease and amylase. | 0 | 12 | 0 | 12 | 7 | 12 |
| EG003 | ANG003 Dose Level 4 | Single administration dose contains 120 mg lipase, 75 mg protease and 120 mg amylase. ANG003: To evaluate four possible combinations of lipase, protease and amylase. | 0 | 13 | 0 | 13 | 7 | 13 |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal Discomfort | Gastrointestinal disorders | MedDRA 26.1 | Non-systematic Assessment |
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| Abdominal Distension | Gastrointestinal disorders | MedDRA 26.1 | Non-systematic Assessment |
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| Abdominal Pain | Gastrointestinal disorders | MedDRA 26.1 | Non-systematic Assessment |
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| Abdominal Pain Upper | Gastrointestinal disorders | MedDRA 26.1 | Non-systematic Assessment |
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| Constipation | Gastrointestinal disorders | MedDRA 26.1 | Non-systematic Assessment |
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| Diarrhoea | Gastrointestinal disorders | MedDRA 26.1 | Non-systematic Assessment |
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| Dyspepsia | Gastrointestinal disorders | MedDRA 26.1 | Non-systematic Assessment |
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| Faeces Soft | Gastrointestinal disorders | MedDRA 26.1 | Non-systematic Assessment |
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| Flatulence | Gastrointestinal disorders | MedDRA 26.1 | Non-systematic Assessment |
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| Frequent Bowel Movements | Gastrointestinal disorders | MedDRA 26.1 | Non-systematic Assessment |
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| Gastrointestinal Sounds Abnormal | Gastrointestinal disorders | MedDRA 26.1 | Non-systematic Assessment |
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| Gastrooesophageal Reflux Disease | Gastrointestinal disorders | MedDRA 26.1 | Non-systematic Assessment |
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| Haematochezia | Gastrointestinal disorders | MedDRA 26.1 | Non-systematic Assessment |
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| Nausea | Gastrointestinal disorders | MedDRA 26.1 | Non-systematic Assessment |
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| Steatorrhoea | Gastrointestinal disorders | MedDRA 26.1 | Non-systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA 26.1 | Non-systematic Assessment |
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| Infusion Site Bruising | General disorders | MedDRA 26.1 | Non-systematic Assessment |
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| Infusion Site Pain | General disorders | MedDRA 26.1 | Non-systematic Assessment |
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| Medical Device Site Phlebitis | General disorders | MedDRA 26.1 | Non-systematic Assessment |
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| COVID-19 | Infections and infestations | MedDRA 26.1 | Non-systematic Assessment |
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| Tinea Infection | Infections and infestations | MedDRA 26.1 | Non-systematic Assessment |
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| Contusion | Injury, poisoning and procedural complications | MedDRA 26.1 | Non-systematic Assessment |
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| Procedural Vomiting | Injury, poisoning and procedural complications | MedDRA 26.1 | Non-systematic Assessment |
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| Skin Laceration | Injury, poisoning and procedural complications | MedDRA 26.1 | Non-systematic Assessment |
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| Alanine Aminotransferase Increased | Investigations | MedDRA 26.1 | Non-systematic Assessment |
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| Aspartate Aminotransferase Increased | Investigations | MedDRA 26.1 | Non-systematic Assessment |
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| Blood Creatine Increased | Investigations | MedDRA 26.1 | Non-systematic Assessment |
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| Blood Triglycerides Increased | Investigations | MedDRA 26.1 | Non-systematic Assessment |
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| Crystal Urine Present | Investigations | MedDRA 26.1 | Non-systematic Assessment |
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| Eosinophil Count Increased | Investigations | MedDRA 26.1 | Non-systematic Assessment |
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| Glucose Urine | Investigations | MedDRA 26.1 | Non-systematic Assessment |
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| Glycosylated Haemoglobin Increased | Investigations | MedDRA 26.1 | Non-systematic Assessment |
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| Insulin C-peptide Increased | Investigations | MedDRA 26.1 | Non-systematic Assessment |
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| Low Density Lipoprotein Increased | Investigations | MedDRA 26.1 | Non-systematic Assessment |
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| Monocyte Count Increased | Investigations | MedDRA 26.1 | Non-systematic Assessment |
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| Protein Urine | Investigations | MedDRA 26.1 | Non-systematic Assessment |
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| Protein Urine Present | Investigations | MedDRA 26.1 | Non-systematic Assessment |
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| Urine Ketone Body Present | Investigations | MedDRA 26.1 | Non-systematic Assessment |
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| Vitamin E Decreased | Investigations | MedDRA 26.1 | Non-systematic Assessment |
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| Vitamin K Decreased | Investigations | MedDRA 26.1 | Non-systematic Assessment |
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| White Blood Cell Count Decreased | Investigations | MedDRA 26.1 | Non-systematic Assessment |
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| Hypoglycaemia | Metabolism and nutrition disorders | MedDRA 26.1 | Non-systematic Assessment |
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| Arthritis | Musculoskeletal and connective tissue disorders | MedDRA 26.1 | Non-systematic Assessment |
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| Headache | Nervous system disorders | MedDRA 26.1 | Non-systematic Assessment |
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| Oropharyngeal Pain | Respiratory, thoracic and mediastinal disorders | MedDRA 26.1 | Non-systematic Assessment |
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| Rash | Skin and subcutaneous tissue disorders | MedDRA 26.1 | Non-systematic Assessment |
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| Skin Irritation | Skin and subcutaneous tissue disorders | MedDRA 26.1 | Non-systematic Assessment |
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| Flushing | Vascular disorders | MedDRA 26.1 | Non-systematic Assessment |
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| Orthostatic Hypotension | Vascular disorders | MedDRA 26.1 | Non-systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Marcie Clarkin | Anagram Therapeutics, Inc. | 6174663111 | mclarkin@anagramtx.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Aug 18, 2024 | Jul 8, 2025 | SAP_001.pdf |
| 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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| Male |
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| Not Hispanic or Latino |
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| Unknown or Not Reported |
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| Asian |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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| No |
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| No |
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| Any SEA |
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| Any Treatment-Related TEAE |
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Single administration dose contains 120 mg lipase, 75 mg protease and 120 mg amylase
ANG003: To evaluate four possible combinations of lipase, protease and amylase.
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Single administration dose contains 120 mg lipase, 75 mg protease and 120 mg amylase. ANG003: To evaluate four possible combinations of lipase, protease and amylase. |
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