A Phase 1 Study of FT819 in B-cell Mediated Autoimmune Disease

Обзор

This is a phase 1 study designed to evaluate the safety, pharmacokinetics (PK), and anti-B-cell activity of FT819 following treatment with or without auxiliary medicinal product (AMP) in participants with moderate-to-severe active systemic lupus erythematosus (SLE) with or without nephritis, antineutrophilic cytoplasmic antibody (ANCA)-associated vasculitis (AAV), idiopathic inflammatory myositis (IIM), and systemic sclerosis (SSc). The study will consist of a dose-escalation stage, followed by an expansion stage to further evaluate the safety and activity of FT819.

Вмешательства

• Препарат FT819
  FT819 will be administered as intravenous (IV) infusion at planned dose levels.
• Препарат Fludarabine
  Fludarabine will be administered as an IV infusion at planned dose levels.
• Препарат Cyclophosphamide
  Cyclophosphamide will be administered as an IV infusion at planned dose levels.
• Препарат Bendamustine
  Bendamustine will be administered as an IV infusion at planned dose levels.

Первичные конечные точки

• Number of participants with treatment-emergent adverse events (TEAEs) [Срок оценки: Up to approximately 2 years]
• Number of participants with serious TEAEs [Срок оценки: Up to approximately 2 years]
• Number of participants with dose-limiting toxicities (DLTs) [Срок оценки: Up to approximately 29 days]

Вторичные конечные точки (4)

• Plasma concentration of FT819 [Срок оценки: At designated time points up to approximately 29 days]
• Impact of treatment on quality of life [Срок оценки: Up to approximately 2 years]
• Disease Activity [Срок оценки: Up to approximately 2 years]
• Disease Activity in Participants with Lupus Nephritis [Срок оценки: Up to approximately 2 years]

Критерии участия

Критерии включения

• Age: 12 to 70 years old.
• Diagnosis: Must have active B-cell mediated autoimmune disease (SLE, AAV, IIM, or SSc) confirmed by standard criteria.
• Disease Severity: Moderate to severe, requiring at least two prior treatments that were ineffective.
• Health Status: Adequate organ function to tolerate treatment.
• Consent: Able to provide informed consent or assent/obtain parental consent and comply with study procedures.

Критерии исключения

• Pregnancy/Breastfeeding: Women must not be pregnant or nursing.
• Severe Organ Dysfunction: Significant heart, lung, liver, or kidney impairment.
• Active Infections: No recent or ongoing serious infections.
• Recent Cancer or Prior Cell Therapy: No active/recent malignancies, prior CAR T-cell therapy, or organ transplant.
• Allergies: No known allergies to study treatments.
• Weight Restriction: Must weigh at least 50 kg (110 lbs).

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

США · 16 центров

• Wallace Rheumatic Center — Beverly Hills
• Providence Medical Foundation — Fullerton
• University of California Irvine — Irvine
• Children's Hospital Los Angeles Division Of Rheumatology — Los Angeles
• University of California San Francisco — San Francisco
• University of Florida — Gainesville
• Nicklaus Children's Hospital — Miami
• University of Louisville — Louisville
• … и ещё 8 центров

Великобритания · 3 центра

• Manchester University NHS Foundation Trust — Manchester
• Cambridge University Hospitals NHS Foundation Trust — Cambridge
• University College of London Hospitals NHS Trust (UCLH) — London

Франция · 1 центр

• Hôpital La Pitié Salpêtrière — Paris

Швеция · 1 центр

• Uppsala University — Uppsala

Идентификаторы

NCT: NCT06308978 · FT819-102

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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