Exocrine Pancreatic Insufficiency
Conditions
Keywords
Cystic Fibrosis
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.
Interventions
DRUGANG003
To evaluate four possible combinations of lipase, protease and amylase.
Sponsors
Anagram Therapeutics, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Masking description
Open-label
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Male and female subjects 18 years of age or older. 2. 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. 3. Documented history of fecal elastase \<100 µg/g stool. 4. EPI clinically controlled with minimal clinical symptoms and on a stable dose of PERT for 90 days before screening as determined by the Investigator. 5. Adequate nutritional status measured by body mass index ≥20kg/m2 for adult subjects.
Exclusion criteria
1. 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. 2. Involuntary loss of ≥10% of usual body weight within last 6 months or involuntary loss of \>5% of body weight within 1 month. 3. Requires use of naso-gastric, J-tube, G-tube, and/or enteral feeding for the study duration. 4. CF pulmonary exacerbation within 30 days prior to the Baseline SACT Period (Visit 2). 5. Subjects who cannot discontinue omega-3 supplements \>500 mg of DHA and EPA daily. 6. Subjects unable to tolerate missing a dose of PERT.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Adverse Events (AE), Serious Adverse Events (SAE) and AEs Leading to Study Discontinuation | 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. |
Countries
United States
Contacts
PRINCIPAL_INVESTIGATORMeghana Sathe, MD
University of Texas
Baseline characteristics
| Characteristic | — |
|---|---|
| Age, Continuous | 25.2 years |
| Body Mass Index (BMI) | 23.7 kg/m^2 |
| Cystic fibrosis transmembrane conductance regulator (CFTR) Modulator Therapy No | 4 Participants |
| Cystic fibrosis transmembrane conductance regulator (CFTR) Modulator Therapy Yes | 13 Participants |
| Diabetes No | 12 Participants |
| Diabetes Yes | 3 Participants |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 47 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 1 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 1 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 13 Participants |
| Sex: Female, Male Female | 7 Participants |
| Sex: Female, Male Male | 7 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk |
|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 13 | 0 / 13 | 0 / 12 | 0 / 13 |
| other Total, other adverse events | 8 / 13 | 8 / 13 | 7 / 12 | 7 / 13 |
| serious Total, serious adverse events | 0 / 13 | 0 / 13 | 0 / 12 | 0 / 13 |
Outcome results
None listed