Phase 2 Study to Assess the Safety and Efficacy of ANG003

Официальное название

A Phase 2, Multicenter, Randomized, Active-controlled Study to Assess the Safety and Efficacy of ANG003 at Two Different Dose Levels in Subjects With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis

Обзор

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.

Вмешательства

• Препарат ANG003 Dose A
  160,000 u\* lipase, 105,000 u\* protease, and 11,600 u\* amylase per dose (\*units are comparable to United States Pharmacopeiea \[USP\])
• Препарат ANG003 Dose B
  240,000 u\* lipase, 105,000 u\* protease, and 11,600 u\* amylase per dose (\*units are comparable to United States Pharmacopeiea \[USP\])
• Препарат Creon
  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.

Первичные конечные точки

• Adverse events (AEs); Treatment-emergent AEs; Serious AEs; Discontinuation due to AEs; Clinical laboratory values [Срок оценки: From Visit 1 to Visit 5, anticipated average 80 days]
• Coefficient of Fat Absorption (CFA) [Срок оценки: Period A (baseline) and Period B 72 hour CFA collection]

Вторичные конечные точки (5)

• Plasma docosahexaenoic acid (DHA) and eicosapentaenoic acid (EPA) (ug/mL) [Срок оценки: Period A (baseline) and Period B time 0, 1, 2, 4, 6, 8, 10 and 24 hour collections]
• Coefficient of Nitrogen Absorption (CNA) [Срок оценки: Period A (baseline) and Period B 72 hour CNA collection]
• % of subjects with GI symptoms [Срок оценки: From Visit 2 to Visit 5, anticipated average 59 days]
• Cumulative stool weight [Срок оценки: Period A (baseline) and Period B 72 hour collection]
• Number of Stools per Day [Срок оценки: Period A (baseline) and Period B 72 hour collection]

Критерии участия

Критерии включения

• 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:

• BMI ≥25th percentile for children aged 12-17 years
• BMI ≥18.5 kg/m2 for ≥18 years of age

Критерии исключения

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Параллельные группы

Маскирование

Простое слепое

Основная цель

Лечение

Центры проведения

США · 26 центров

• Long Beach Memorial Medical Center — Long Beach
• Center for Cystic Fibrosis at Keck Medical Center of USC — Los Angeles
• National Jewish Health — Denver
• University of Florida — Gainesville
• Central Florida Pulmonary Group — Orlando
• The Cystic Fibrosis Institute — Glenview
• University of Iowa — Iowa City
• University of Kansas Medical Center — Kansas City
• … и ещё 18 центров

Идентификаторы

NCT: NCT07450547 · ANG003-25-201 · 2025-524569-25-00

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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