Daratumumab for Patients With Light Chain Amyloidosis

Официальное название

Daratumumab and Dexamethasone Combined With Pomalidomide (DPD) or ASCT in the Treatment of Newly Diagnosed Systemic Light Chain Amyloidosis (AL Amyloidosis): a Prospective, Single Center Clinical Trial

Обзор

This is a prospective, single-center study exploratory clinical trial, aim to exploring the efficacy and safety of daratumumab in patients with AL amyloidosis, patients were divided into three groups: one group received long-term treatment with daratumumab based regimen, and the other group received autologous stem cell transplantation after two standard treatment courses with daratumumab based regimen, and the third group consists of newly diagnosed stage IIIb AL amyloidosis patients who plan to receive DPD treatment. The purpose of this study is to observe the efficacy and safety of Daratumumab, in the treatment of newly diagnosed systemic AL amyloidosis.

Подробное описание

This is a prospective, single-center study exploratory clinical trial, aim to exploring the efficacy and safety of daratumumab in patients with AL amyloidosis, patients were divided into three groups: one group received long-term treatment with daratumumab based regimen (group A) and the other group received autologous stem cell transplantation after two standard treatment courses with daratumumab based regimen (group B) , and the third group consists of newly diagnosed stage IIIb AL amyloidosis patients who plan to receive DPD treatment (group C). Participants with AL Amyloidosis will receive the drug daratumumab by IV infusion once weekly for two months, then every 2 weeks for four months, then once each month. Study treatment may continue until disease progression, unacceptable toxicity, or decision to withdraw from the trial. Disease evaluations will be performed every three months until disease progression. Group C were given Daratumumab (16mg/kg), dexamethasone (40mg) and pomalidomide. The usage of Daratumumab and dexamethasone is the same as group A and B. Patients received oral pomalidomide on days 1-21 of a 28-days cycle from cycle 1 to 6. The dose of pomalidomide is adjusted based on the patient's renal function and ECOG score.

Вмешательства

• Препарат Dratumumab / Hyaluronidase Injection [Darzalex Faspro]
  Daratumumab will be administered at the FDA-approved dose of 16mg/kg as an intravenous infusion.
• Препарат Pomalidomide 4 MG
  Patients received oral pomalidomide on days 1-21 of a 28-days cycle from cycle 1 to 6.
• Процедура autologous stem cell transplantation (ASCT)
  The ASCT protocol included mobilisation with colony-stimulating factor alone and conditioning with high-dose melphalan140-200 mg/m2 .

Первичные конечные точки

• Hematological complete response rate [Срок оценки: 6 months]
• Organ response rate [Срок оценки: 1 year]

Вторичные конечные точки (3)

• Time to Next Treatment [Срок оценки: 3 years]
• Progression-free survival [Срок оценки: 3 years]
• Overall survival [Срок оценки: 5 years]

Критерии участия

Критерии включения

• Participants must be aged ≥18 and ≤75 years;
• Newly diagnosed AL amyloidosis ((confirmed by pathological examination with at least one major organ involvement (heart, kidney, or liver));
• In group A and B, according to the Mayo 2004 staging system, the disease is classified as stage Ⅰ-ⅢA; In group C, according to the Mayo 2004 staging system, the disease is classified as stage IIIB: NT⁃proBNP>8500ng/L and cTnT>0.035μg/L or cTnI>0.01g/L;
• Participants must personally sign an informed consent form approved by the Ethics Committee before the start of the study;
• Expected survival ≥ 12 weeks;
• ECOG performance status≤ 2;
• Female participants of childbearing potential must agree to use effective contraception from the day of signing the informed consent until 365 days after the infusion. Effective contraception is defined as abstinence or the use of a contraceptive method with a failure rate of <1% per year.

Критерии исключения

• eGFR< 30ml/min/1.73m2;
• Combined multiple myeloma;
• Acute or chronic infection requiring treatment within 30 days prior to baseline;
• Pregnant or breastfeeding women.
• Participants known to have life-threatening allergic reactions, hypersensitivity, or intolerance to Monoclonal antibodies or immune modulators.
• Other conditions deemed by the researcher as unsuitable for enrollment.

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

Открытое

Основная цель

Лечение

Центры проведения

Китай · 1 центр

• National Clinical Research Center for Kidney Diseases, Jinling Hospital — Нанкин

Идентификаторы

NCT: NCT06376214 · 2024-02

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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