Osteogenesis Imperfecta Trial of AGA2115 for ADUlts With COL1A1 and/or COL1A2 GeNetic Variations (IDUN)

Официальное название

A Phase 2, Multi-center, Randomized, Double-blind, Placebo-controlled, Dose-ranging Study to Evaluate the Safety and Efficacy of AGA2115 in Adults With Type I, III, or IV Osteogenesis Imperfecta (OI)

Обзор

This study will determine the effect of treatment of AGA2115 in adults with Type I, III, or IV osteogenesis imperfecta versus placebo.

Подробное описание

This Phase 2 dose-ranging study will evaluate the safety and efficacy of AGA2115 at a range of doses in adults with Type I, III, or IV osteogenesis imperfecta. The study will last 27 months with a 24-month treatment period and a 3-month follow-up period. During the first 12 months of the study, participants will be randomized 1:1:1:1 to receive either placebo or one of three dose levels of AGA2115 doses; treatment assignment will be double-blind. Months 12 to 24 will be open-label, and all participants will receive AGA2115. Participants will attend visits where safety and efficacy parameters will be evaluated.

Вмешательства

• Препарат AGA2115
  Subcutaneous injection
• Другое Placebo
  Subcutaneous injection

Первичные конечные точки

• Percent change from Baseline at Month 12 in lumbar spine BMD [Срок оценки: 12 months]

Вторичные конечные точки (5)

• Percent change from Baseline at Month 3 and 6 in lumbar spine, total hip, femoral neck, one-third distal radius, and total body (minus head) BMD [Срок оценки: 3 and 6 months]
• Percent change from Baseline at Month 12 in total hip, femoral neck, one-third distal radius, and total body (minus head) BMD [Срок оценки: 12 months]
• Percent change from Baseline at Week 1 and Month 1, 3, 6, 9, and 12 in P1NP and CTX-1 [Срок оценки: Week 1, Month 1, 3, 6, 9, and 12]
• Percentage of participants with fractures during the double-blind treatment period [Срок оценки: up to 12 months]
• Annualized fracture rate for incident fractures occurring during the double-blind treatment period (total, long-bone, vertebral, peripheral) [Срок оценки: up to 12 months]

Критерии участия

Критерии включения

• Male or female adults (aged 18 to 75 years inclusive) with a clinical diagnosis of osteogenesis imperfecta Type I, III, or IV with documented genetic testing confirmation of genetic variations in the COL1A1 or COL1A2 genes
• Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in the protocol
• BMD T-score of ≤ -1.0 at the lumbar spine, total hip, or femoral neck

Критерии исключения

• Vitamin D deficiency
• Concomitant uncontrolled diseases or conditions that could affect bone metabolism such as hypo-/hyperparathyroidism, hypo-/hyperthyroidism, abnormal thyroid function or thyroid disease, or other endocrine disorders
• Current hyper- or hypocalcemia
• History of rickets or osteomalacia or any skeletal condition (other than OI) leading to long-bone deformities and/or increased risk of fractures
• Treatment with bisphosphonates within the past 6 months
• Treatment with teriparatide, abaloparatide, strontium ranelate, or hormone replacement therapy within the past 12 months
• Treatment with denosumab (or denosumab biosimilars) within the past 2 years
• Treatment with anti-sclerostin antibody medications (romosozumab, setrusumab, blosozumab) at any time
• History of myocardial infarction or stroke (or other cardiovascular associated event deemed significant) within the past 12 months
• Malignancy within the last 5 years
• Pregnant or breastfeeding women, or women planning to become pregnant during the study
• Participation in any clinical study within the past 12 months during which the participant was administered any IP (participant must also agree not to enroll in any other clinical study concurrently in which IP is administered)

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

Двойное слепое

Основная цель

Лечение

Центры проведения

США · 9 центров

• Phoenix Children's — Phoenix
• Yale University School of Medicine — New Haven
• Nemours/Alfred I. duPont Hospital for Children — Wilmington
• Indiana University School of Medicine, Department of Medicine and Pediatrics Division of E — Indianapolis
• Washington University School of Medicine in St. Louis — St Louis
• New Mexico Clinical Research & Osteoporosis Center, Inc. (NMCROC) — Albuquerque
• The Ohio State University Wexner Medical Center (OSUWMC) — Columbus
• Oregon Health & Science University (OHSU) - The Harold Schnitzer Diabetes Health Center (H — Portland
• … и ещё 1 центр

Австралия · 4 центра

• Monash University-Monash Medical Centre (MMC) — Melbourne
• Royal Melbourne Hospital — Parkville
• Royal North Shore Hospital (RNSH) — Saint Leonards
• Adults Westmead Hospital — Westmead

Великобритания · 4 центра

• Cambridge University Hospitals NHS Foundation Trust-Addenbrooke's Hospital — Cambridge
• Lothian Health Board, Western General Hospital — Edinburgh
• Royal National Orthopaedic Hospital NHS Trust — London
• Oxford University Hospitals NHS Foundation Trust — Oxford

Нидерланды · 3 центра

• Leiden University Medical Center (LUMC) - Centrum voor botkwaliteit — Leiden
• Erasmus MC — Rotterdam
• Isala ziekenhuizen — Zwolle

Дания · 2 центра

• Aarhus Universitetshospital - Medicinsk Endokrinologisk Afdeling (MEA) Ambulatoriet - Tage — Aarhus
• Odense Universitetshospital — Odense

Франция · 2 центра

• Hopital Edouard Herriot — Lyon
• Assistance Publique-Hopitaux de Paris (AP-HP) - Hopital Lariboisiere — Paris

Аргентина · 1 центр

• Instituto de Investigaciones Metabolicas Dr. Zanchetta - Sede Centro — Buenos Aires

Канада · 1 центр

• University Health Network - Toronto General Hospital - Osteoporosis Clinic — Toronto

Идентификаторы

NCT: NCT07062588 · ACT24-001

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

Открыть это исследование на ClinicalTrials.gov ↗