A Phase 1b/2 Study of CAR T Cell Therapy Targeting CD19 and BCMA in Participants With Relapsed or Refractory AL Amyloidosis.

Официальное название

A Phase 1b/2 Study of AZD0120 (Also Known as GC012F), a Chimeric Antigen Receptor T Cell Therapy Targeting CD19 and B Cell Maturation Antigen in Participants With Relapsed or Refractory AL Amyloidosis.

Обзор

Open-label Phase 1b/2 study with primary objective of this study is to evaluate the safety, tolerability and efficacy of AZD0120 in participants with light chain (AL) amyloidosis.

Вмешательства

• Препарат AZD0120
  Participants will receive AZD0120 via intravenous (IV) infusion.

Первичные конечные точки

• Phase 1b: Number of Participants With incidence and severity of Treatment-emergent Adverse Events [Срок оценки: Through study completion, a minimum of 6 months]
• Phase 2: Proportion of Participants Achieving Complete Response [Срок оценки: Through study completion, a minimum of 6 months]

Вторичные конечные точки (2)

• Phase 1b: Levels of AZD0120 in blood over time in participants with AL amyloidosis [Срок оценки: Through study completion, a minimum of 6 months]
• Phase 2: Percentage of participants achieving hematologic response [Срок оценки: Through study completion, a minimum of 6 months]

Критерии участия

Критерии включения

• Confirmed histopathological diagnosis of AL amyloidosis
• One or more organs currently or historically impacted by AL amyloidosis according to consensus guidelines
• Measurable hematologic disease: dFLC > 20 mg/L or serum M-protein > 5g/L
• Relapsed or refractory disease with a need for additional therapy after at least 1 line of anti-plasma cell-directed therapy.
• ECOG performance status of 0 to 2
• Must be able and willing to adhere to the study visit schedule and other protocol requirements
• Women of child-bearing potential (WCBP) must have a negative serum and/or urine pregnancy test result prior to treatment. All sexually active WCBP and all sexually active male subjects must agree to use highly effective methods of birth control throughout the study.

Критерии исключения

• Have any other form of amyloidosis other than AL amyloidosis
• Mayo Stage IIIb AL amyloidosis
• Oxygen saturation < 95% on room air
• Systolic blood pressure <100mmHg
• NYHA class III or IV
• Extensive GI involvement with evidence of active GI bleeding/risk of bleeding as determined by Investigator
• Prior therapies:

• CAR T cell therapy directed at any target
• Prior BCMA-targeting therapy
• Prior treatment with any FDA approved or investigational T cell engaging therapies (including T cell-directed bispecific or trispecific therapies) at any target within the last 6 months.
• Toxicity from previous anti-cancer or anti-PC-directed therapy did not resolve to baseline levels or to Grade 1 or less except for alopecia or peripheral neuropathy.
• Active plasma cell leukemia at the time of screening
• Symptomatic multiple myeloma (defined as clonal bone marrow plasma cells ≥10% plus at least one myeloma-defining event per IMWG 2014)

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

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

США · 12 центров

• Research Site — Phoenix
• Research Site — San Francisco
• Research Site — Tampa
• Research Site — Boston
• Research Site — Detroit
• Research Site — Rochester
• Research Site — St Louis
• Research Site — New York
• … и ещё 4 центра

Канада · 4 центра

• Research Site — Calgary
• Research Site — Calgary
• Research Site — Toronto
• Research Site — Toronto

Великобритания · 2 центра

• Research Site — London
• Research Site — London

Идентификаторы

NCT: NCT07081646 · D831AC00001 · AZD0120-AL-201

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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