Phase 2 Study Evaluating Rapcabtagene Autoleucel in Participants With Severe Active GPA or MPA

Официальное название

A Phase 2, Randomized, Open-label, Controlled Study to Evaluate the Efficacy and Safety of Rapcabtagene Autoleucel Versus Comparator in Participants With Severe Active Granulomatosis With Polyangiitis (GPA) or Microscopic Polyangiitis (MPA)

Обзор

The purpose of this study is to evaluate the efficacy and safety of rapcabtagene autoleucel versus comparator in participants with severe active Granulomatosis with Polyangiitis (GPA) or Microscopic Polyangiitis (MPA)

Подробное описание

This is a Phase 2, randomized, assessor-blinded active controlled study. This study comprises two cohorts:

* A lead-in cohort enrolling participants to receive rapcabtagene autoleucel * A randomized cohort with participants receiving either rapcabtagene autoleucel or comparator.

After end of study (EOS), participants who received rapcabtagene autoleucel infusion will enter a long-term follow-up (LTFU) period lasting up to 15 years after rapcabtagene autoleucel infusion. This LTFU will be described in a separate study protocol.

Вмешательства

• Биопрепарат Rapcabtagene autoleucel
  Single infusion of rapcabtagene autoleucel
• Другое Active Comparator
  Active comparator option as per protocol
• Препарат Glucocorticoids
  Concomitant glucocorticoids as per protocol

Первичные конечные точки

• Event-free survival (EFS) [Срок оценки: From randomization until the occurrence of an EFS event, up to approx. 4 years after randomization]

Вторичные конечные точки (6)

• Percentage of patients achieving complete remission [Срок оценки: Up to Week 13]
• Adjusted annual cumulative GC dose between Randomization and analysis cutoff date [Срок оценки: From randomization until the occurrence of an EFS event, up to approx. 4 years after randomization]
• ANCA seronegativity and sustaining ANCA seronegativity until the analysis cutoff date [Срок оценки: From randomization until the occurrence of an EFS event, up to approx. 4 years after randomization]
• Change from baseline in estimated glomerular filtration rate (eGFR) at Week 39 [Срок оценки: Up to Week 39]
• Change from baseline in symptoms of GPA/ MPA using the AAV-PRO at Week 39 [Срок оценки: Up to Week 39]
• Change from baseline in Patient- Reported Outcome Measurement Information System (PROMIS)- Fatigue 7a at Week 91. [Срок оценки: Up to Week 91]

Критерии участия

Критерии включения

• Men and women, aged ≥18 and ≤ 75 years with a diagnosis of GPA or MPA according to the American College of Rheumatology/ European League Against Rheumatism 2022 (ACR/EULAR 2022) classification criteria
• Positive test for ANCA-autoantibodies
• GPA and MPA participants with severe active disease

Критерии исключения

• Any condition that could prevent a complete washout of medications or could otherwise make the participant ineligible for anti-CD19 CAR-T therapy and further participation in the study
• Hypersensitivity and/or contraindications to any product to be given to the participant as part of the study protocol
• Other systemic autoimmune diseases requiring therapy
• Any medical conditions that are not related to GPA/MPA that would jeopardize the ability of the participant to tolerate CD19 CAR-T cell therapy
• Inadequate organ function

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 · 11 центров

• University Of Colorado — Aurora
• Mayo Clinic Jacksonville — Jacksonville
• Northwestern University — Chicago
• Massachusetts General Hospital — Boston
• Michigan Med University of Michigan — Ann Arbor
• University of Minnesota — Minneapolis
• Mayo Clinic Rochester — Rochester
• Oregon Health Sciences University — Portland
• … и ещё 3 центра

Япония · 10 центров

• Novartis Investigative Site — Sapporo
• Novartis Investigative Site — Kobe
• Novartis Investigative Site — Sendai
• Novartis Investigative Site — Suita
• Novartis Investigative Site — Bunkyo Ku
• Novartis Investigative Site — Shinjuku-ku
• Novartis Investigative Site — Chiba
• Novartis Investigative Site — Fukuoka
• … и ещё 2 центра

Бразилия · 4 центра

• Novartis Investigative Site — Salvador
• Novartis Investigative Site — Barretos
• Novartis Investigative Site — São Paulo
• Novartis Investigative Site — São Paulo

Великобритания · 3 центра

• Novartis Investigative Site — Cambridge
• Novartis Investigative Site — London
• Novartis Investigative Site — Manchester

Израиль · 2 центра

• Novartis Investigative Site — Haifa
• Novartis Investigative Site — Ramat Gan

Сингапур · 2 центра

• Novartis Investigative Site — Singapore
• Novartis Investigative Site — Singapore

Швейцария · 2 центра

• Novartis Investigative Site — Basel
• Novartis Investigative Site — Bern

Саудовская Аравия · 1 центр

• Novartis Investigative Site — Riyadh

Идентификаторы

NCT: NCT06868290 · CYTB323I12201

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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