Phase 2 Study to Assess the Safety and Efficacy of ANG003
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age12+
SponsorAnagram Therapeutics, Inc.
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
Disqualifiers
History of fibrosing colonopathy or recurring distal intestinal obstructive syndrome within 6 months of Screening.
History of lung or liver transplant, listing for organ transplant or significant bowel resection within the last 6 months. Subjects with a history of resection that does not result in short bowel syndrome are eligible.
Known hypersensitivity or other severe reaction to any ingredient of the investigational product (IP), Creon, or the stool marker (FD&C Blue #2).
Any chronic diarrheal illness unrelated to pancreatic insufficiency, actively being treated for small intestinal bacterial overgrowth, requiring use of naso-gastric, J-tube, G-tube, and/or enteral feeding for the study duration, Clostridioides difficile infection within 6 months prior to Screening, or severe constipation defined as <1 bowel movement/week.
Trial design
Design model
Parallel
Treatments tested in this trial
ANG003 Dose A
Drug160,000 u\* lipase, 105,000 u\* protease, and 11,600 u\* amylase per dose (\*units are comparable to United States Pharmacopeiea \[USP\])
ANG003 Dose B
Drug240,000 u\* lipase, 105,000 u\* protease, and 11,600 u\* amylase per dose (\*units are comparable to United States Pharmacopeiea \[USP\])
Creon
DrugThe 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.
Treatment groups
113 Participants
are divided into 5 treatment groupsGroup A: ANG003 Dose AExperimental treatment 1 intervention
Group B: ANG003 Dose BExperimental treatment 1 intervention
Group C: CreonActive comparator 1 intervention
Group D: Off EnzymeNo intervention 0 interventions
Group E: 60% to <80% CFA ANG003 Dose BOther 1 intervention
Trial outcomes
Primary outcomes
1
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
Time frame
From Visit 1 to Visit 5, anticipated average 80 days2
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%.
Time frame
Period A (baseline) and Period B 72 hour CFA collectionSecondary outcomes
1
Plasma docosahexaenoic acid (DHA) and eicosapentaenoic acid (EPA) (ug/mL)
Mean change from baseline for each treatment group amongst subjects with baseline CFA ≥80%.
Time frame
Period A (baseline) and Period B time 0, 1, 2, 4, 6, 8, 10 and 24 hour collections2
Coefficient of Nitrogen Absorption (CNA)
Mean change from baseline for each treatment group amongst subjects with baseline CFA ≥80%.
Time frame
Period A (baseline) and Period B 72 hour CNA collection3
% of subjects with GI symptoms
Mean change from baseline for each treatment group amongst subjects with baseline CFA ≥80%.
Time frame
From Visit 2 to Visit 5, anticipated average 59 days4
Cumulative stool weight
Mean change from baseline for each treatment group amongst subjects with baseline CFA ≥80%.
Time frame
Period A (baseline) and Period B 72 hour collectionOther outcomes
1
Correlation between DHA/EPA and CFA
Mean change from baseline for each treatment group with a baseline CFA ≥80%
Time frame
Period A (baseline) Period B 72 hour CFA and 24 hour DHA/EPA collection2
Coefficient of Fat Absorption (CFA) for subjects with a baseline CFA of 60% to <80% (analyzed separately)
Mean change from baseline for each treatment group with a baseline CFA 60% to \<80%
Time frame
Period A (baseline) and Period B 72 hour CFA collection3
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%
Time frame
Period B 72 hour CFA collectionSponsors and contacts
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Source ClinicalTrials.gov