A Study Evaluating the Safety, Tolerability, and Activity of ICM-203 in Subjects With Knee Osteoarthritis.

Официальное название

A Single Dose Escalation Study of Intra-Articular ICM-203 in Subjects With Kellgren-Lawrence Grade 2 or Grade 3 Osteoarthritis of the Knee

Обзор

The purpose of this study is to determine the safety, tolerability, and activity of ICM-203, a recombinant adeno-associated viral (AAV) vector that expresses a therapeutic gene that promotes cartilage formation, reduces joint inflammation and pain, as well as improves joint physical function, by injecting escalating doses of ICM-203 into the knee of subjects with mild to moderate knee osteoarthritis (OA). Approximately 6 to 18 subjects will be enrolled into 3 successive dose-escalating groups in a 3+3 study design, whereby 3 study subjects in each group will be dosed sequentially with ICM-203 and 3 additional subjects will be dosed at the same dose level if a dose limiting toxicity (DLT) occurs in any of the first 3 subjects.

Вмешательства

• Генная терапия ICM-203
  Intra-articular injection

Первичные конечные точки

• Treatment-Emergent Adverse Events (TEAEs) [Срок оценки: Up to Week 52]
• Knee pain [Срок оценки: Up to Week 52]
• Knee function [Срок оценки: Up to Week 52]
• Analgesic use [Срок оценки: Up to Week 52]
• Magnetic Resonance Imaging Osteoarthritis Knee Score (MOAKS) [Срок оценки: Up to Week 52]
• Joint space width [Срок оценки: Up to Week 52]

Вторичные конечные точки (3)

• Humoral response to AAV5.2 capsid [Срок оценки: Up to Week 52]
• Cellular immune response to AAV5.2 capsid [Срок оценки: Up to Week 52]
• Systemic biodistribution of ICM-203 [Срок оценки: Up to Week 52]

Критерии участия

Критерии включения

• Body mass index (BMI) of 18.0 to 40.0 kg/m2, inclusive, at the time of screening.
• Kellgren-Lawrence grade 2 or grade 3 OA of target knee.
• Target knee pain between 4 and 9, inclusive, on an NRS ranging from 0 (no pain) to 10 (worst pain imaginable), at the time of screening.
• KOOS function in daily living score >25, a measure of knee function ranging from 0 (extreme problems) to 100 (no problems), at the time of screening .
• A stable treatment regimen for symptomatic relief of OA, including NSAIDs, for 4 weeks prior to screening.

Критерии исключения

• History of rheumatoid arthritis, psoriatic arthritis, gout, pseudogout, autoimmune OA, chondrocalcinosis, hemochromatosis, villonodular synovitis, and synovial chondromatosis or other disorder that in the opinion of the Investigator could cause inflammation of the knee.
• Injection of steroid, hyaluronate or other agent, into the target knee less than 90 days prior to day 1.
• Major injury to the target knee, such as torn ligament or severe sprain, within 12 months of screening.
• Disability so severe that the subject cannot comply with the study requirements, including knee symptoms that result in significant difficulty or inability to walk.
• Surgery on the target knee within 180 days prior to day 1.
• Total knee arthroplasty or other knee surgery planned in the next 12 months.
• Active joint infection or other concurrent medical or psychiatric condition that, in the opinion of the Investigator, would make the subject unsuitable for the study.

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

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Нерандомизированное

Модель

Последовательный дизайн

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Список центров уточняется — проверьте первичный протокол.

Идентификаторы

NCT: NCT05454566 · ICM 20-1003

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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