Optimize First-line Treatment for AL Amyloidosis With t (11; 14)

Официальное название

Optimize First-line Treatment for Systemic Light Chain Amyloidosis With t (11; 14)

Обзор

Achievement of complete hematologic response (CHR) is vital for systemic AL amyloidosis. Currently, the CHR rate of daratumumab, bortezomib, and dexamethasone (DBD) is close to 60%. Considering that Bcl-2 inhibitor is effective for AL amyloidosis with t(11; 14) and the median hematologic onset time of DBD is 7 days. We design a a prospective study on AL amyloidosis with t(11; 14). All patients receive DBD at the beginning. Patient will receive DBD for at least 6 cycles if achieve rapid hematologic response at day 7, while other patients will receive daratumumab, venetoclax and dexamethasone.

Подробное описание

The goal of this clinical trial is to optimize the first line treatment for systemic AL amyloidosis with t(11;14). The aim of this study is to pursue early complete hematologic response. The primary endpoint is overall complete hematologic response (CHR) rate at 6 months. Participants will be treated according to the hematologic response after 7 days. If the patient get rapid response after 7 days, he/she will receive daratumumab, venetoclax and dexamethasone (DBD) for at least 6 cycles. If the patient do not get rapid response, he/she will receive daratumumab, venetoclax and dexamethasone.

Вмешательства

• Препарат Daratumumab
  Daratumumab 16 mg/kg was administered intravenously weekly in cycles one and two, every two weeks for cycles three to six, for at least 6 cycles. Daratumumab and hyaluronidase-fihj 1800mg is allowed according to the patients' choice.
• Препарат Bortezomib
  All patients received 1.0-1.3 mg/m2 subcutaneous bortezomib once weekly of 28 days each for at 6 cycles.
• Препарат Dexamethasone
  All patients received 20-40 mg oral or intravenous dexamethasone
• Препарат Venetoclax
  All patients received venetoclax 400mg daily.

Первичные конечные точки

• Overall CHR rate at 6 months [Срок оценки: Overall CHR rate at 6 months]

Вторичные конечные точки (7)

• Cardiac response at 6 months [Срок оценки: Cardiac response at 6 months]
• Renal response at 6 months [Срок оценки: Renal response at 6 months]
• Hepatic response at 6 months [Срок оценки: Hepatic response at 6 months]
• Estimated 2-year PFS [Срок оценки: Estimated 2-year PFS]
• Estimated 2-year OS [Срок оценки: Estimated 2-year overall survival]
• MRD status at 6 months [Срок оценки: MRD status at 6 months]
• TRAEs [Срок оценки: TRAEs]

Критерии участия

Критерии включения

• Diagnosis of systemic AL amyloidosis;
• Daratumumab, bortezomib, dexamethasone used in 1st line treatment;
• Life expectancy greater than 12 weeks;
• HGB ≥70g/L;
• Blood oxygen saturation >90%;
• Total bilirubin (TBil) ≤3×upper limit of normal (ULN); aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤3.0×ULN;
• Informed consent explained to, understood by and signed by the patient.

Критерии исключения

• Fulfill with the criteria of active multiple myeloma or active lymphoplasmacytic lymphoma.
• Presence of other tumors which is/are in advanced malignant stage and has/have systemic metastasis;
• Severe or persistent infection that cannot be effectively controlled;
• Presence of severe autoimmune diseases or immunodeficiency disease;
• Patients with active hepatitis B or hepatitis C (\[HBVDNA+\] or \[HCVRNA+\]);
• Patients with HIV infection or syphilis infection;
• Any situations that the researchers believe will increase the risks for the subject or affect the results of the study.

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

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Нерандомизированное

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Китай · 7 центров

• Peking University People's Hospital — Пекин
• Capital Medical University Affiliated Fuxing Hospital — Пекин
• Beijing Anzhen Hospital — Пекин
• Chinese PLA Eastern Theater General Hospital — Нанкин
• Qilu Hospital of Shandong University (QingDao) — Qingdao
• Qingdao Municipal Hospital — Qingdao
• The First Affiliated Hospital of Xi'an Jiao Tong University — Xi’an

Идентификаторы

NCT: NCT06192979 · 2023PHB319-001

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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