Study to Evaluate the Efficacy, Safety, and Tolerability of Efzofitimod in Patients With Systemic Sclerosis (SSc)-Related Interstitial Lung Disease (ILD) (SSc-ILD)

Официальное название

Randomized, Double-blind, Placebo-controlled Proof-of-Concept (PoC) Study to Evaluate the Efficacy, Safety, and Tolerability of Efzofitimod in Patients With Systemic Sclerosis (SSc)-Related Interstitial Lung Disease (ILD) (SSc-ILD)

Обзор

This is a 2-Part study with Part A, a double-blind, randomized, placebo-controlled, PoC study to evaluate the efficacy, safety, and tolerability of efzofitimod in patients with SSc-ILD. The primary objective of the study is to evaluate the PoC for efficacy in a population with SSc-ILD. While improvement of ILD is the outcome of interest, the study will also evaluate changes in the skin. After initial screening (up to 4 weeks), approximately 25 eligible participants will be randomized 2:2:1 to 1 of 2 active (experimental) dose arms or placebo, administered every 4 weeks up to and including Week 20. Part B is an optional open-label extension to Part A in which participants can receive 450 mg efzofitimod every 4 weeks for 6 doses.

Вмешательства

• Препарат efzofitimod 450 mg
  IV infusion over approximately 60 minutes every 4 weeks
• Препарат efzofitimod 270 mg
  IV infusion over approximately 60 minutes every 4 weeks
• Препарат Placebo
  IV infusion over approximately 60 minutes every 4 weeks

Первичные конечные точки

• Absolute change from baseline in forced vital capacity (FVC) in mL [Срок оценки: 24 weeks]
• Annual rate of decline in FVC in mL [Срок оценки: 24 weeks]
• Annual rate of decline in FVC in percent predicted [Срок оценки: 24 weeks]
• Change in HRCT fibrosis score [Срок оценки: Baseline to Week 24]

Критерии участия

Критерии включения

• Diagnosis of SSc based on ACR/ EULAR criteria (2013)
• Overall duration of SSc < 84 months from the first non-Raynaud symptom manifestation prior to Day 1
• HRCT obtained at the Screening Visit or within the 3 months prior to Screening consistent with SSc-ILD (adjudicated by a central reader) AND with pulmonary involvement > 10%
• Clinical presentation at Screening consistent with lcSSc (up to 40% of patients) or dcSSc
• MMF of ≥ 2 gm/day (or equivalent doses of other mycophenolate based compounds) for 3 months prior to Day 1 OR When documented intolerance to mycophenolates (in discussion with the Medical Monitor): treatment with maximum tolerated dose of MMF is acceptable, if < 2 gm/day, provided the cumulative duration of dosing has exceeded 3 months, OR An adequate dose and duration of an alternate immunosuppressant with a stable dose for the 4 weeks prior to baseline is also allowed.

Критерии исключения

• Pulmonary disease with FVC %pred ≤ 45% OR DLco %pred ≤ 30%; FEV1/FVC ratio < 0.7
• Participants with pulmonary artery hypertension on parenteral therapy or with clinical evidence of right heart failure
• HRCT obtained in the 3 months prior to Screening consistent with other confounding pathology.
• Treatment with corticosteroids (> 10 mg/day of prednisone or equivalent) within 2 weeks prior to Day 1
• Treatment with more than 1 immunosuppressant (e.g., MMF, methotrexate \[MTX\], azathioprine \[AZA\], or leflunomide)
• Any treatment in the 12 months prior to Day 1 with any of the following: rituximab, intravenous immune globulin (IVIG), tocilizumab, cyclophosphamide, pirfenidone, tyrosine-kinase inhibitors (e.g., imatinib, nilotinib, dasatinib)
• Rheumatic autoimmune disease other than SSc, Is an active, heavy smoker of tobacco/nicotine-containing products
• History of (anti-Jo-1) anti-synthetase syndrome or Jo-1 positive at Screening

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

Тройное слепое

Основная цель

Лечение

Центры проведения

США · 15 центров

• aTyr Investigative Site — Los Angeles
• aTyr Investigative Site — San Diego
• aTyr Investigative Site — Miami
• aTyr Investigative Site — Chicago
• aTyr Investigative Site — Chicago
• aTyr Investigative Site — Chicago
• aTyr Investigative Site — New Orleans
• aTyr Investigative Site — New York
• … и ещё 7 центров

Идентификаторы

NCT: NCT05892614 · ATYR1923-C-005

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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