Rapid dFLC Response Predict CHR in AL Amyloidosis

Официальное название

Rapid dFLC Response Predict Complete Hematologica Response in Systemic AL Amyloidosis Patients Treated With Daratumumab-based Regimen

Обзор

Light chain amyloidosis (AL amyloidosis) is a rare plasma cell dyscrasia characterized by the deposition of insoluble amyloid fibrils in multiple organ systems. The treatment of amyloidosis primarily relies on anti-plasma cell therapy and supportive care. The application of anti-plasma cell therapy has significantly improved outcomes in patients with AL amyloidosis. Standard first-line therapy typically includes daratumumab, bortezomib, cyclophosphamide, and dexamethasone (Dara-BCD), achieving a complete hematologic response in nearly 60% of patients.The depth and speed of the hematologic response are strongly correlated with organ response and overall survival. An early achievement of a complete hematologic response is particularly crucial in cases of AL amyloidosis characterized by significant organ involvement. The median time to a hematologic response for the daratumumab based treatment is only 7-9 days. The retrospective data showed that the hematologic response in Day 7 in Cycle 1 (C1D7) may predict the complete hematologic response rate. In order to validate the conclusion, the investigator design this prospective study.

Вмешательства

• Препарат Dara IV
  The treament follow the clinical routine practice, as daratumumab 16mg/kg iv qw OR dara SC 1800mg initially. The treatment schedule involve weekly administrations during cycles 1 to 2, following by biweekly administrations during cycles 3 to 6. Subsequently, monthly administrations are given as monotherapy.
• Препарат Bortezomib (drug)
  Bortezomib is administered subcutaneously. The dosage range from 0.7 to 1.3 mg/m2 on days 1, 8, 15, and 22 of each cycle, for a maximum of 6 cycles
• Препарат Dexamethasone
  Dexamethasone is administered intravenously at a dose of 40 mg weekly for each cycle, for a maximum of 6 cycles. For patients over 70 years of age or with severe edema, heart failure, or uncontrolled diabetes mellitus, dexamethasone can be administered at a dose of 10-20 mg per week.
• Препарат Cyclophosphamide (CTX)
  Some patients may receive cyclophosphamide orally or intravenously at a dosage of 300 mg/m2 weekly.
• Препарат Dara SC
  The treament follow the clinical routine practice, as daratumumab 16mg/kg iv qw OR dara SC 1800mg initially. The treatment schedule involve weekly administrations during cycles 1 to 2, following by biweekly administrations during cycles 3 to 6. Subsequently, monthly administrations are given as monotherapy.

Первичные конечные точки

• Complete hematologic response [Срок оценки: 6 months]

Вторичные конечные точки (6)

• Overall hematologic response [Срок оценки: 6 months]
• At least one organ response [Срок оценки: 12 months]
• Minimal residual disease [Срок оценки: 6 months, 12 months]
• TTNT [Срок оценки: 12 months]
• MOD-EFS [Срок оценки: 12 months]
• MOD-PFS [Срок оценки: 12 months]

Критерии участия

Критерии включения

• Diagnosis of systemic AL amyloidosis;
• Daratumumab, bortezomib, dexamethasone used in treatment;
• Informed consent explained to, understood by and signed by the patient;
• dFLC ≥ 50 mg/L;

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

Модель наблюдения

Когортное

Центры проведения

Китай · 8 центров

• Peking University People's Hospital — Пекин
• Fuxing Hospital affiliated to Capital Medical University — Пекин
• Beijing Anzhen Hospital — Пекин
• Peking University First Hospital — Пекин
• First Affiliated Hospital of Harbin Medical University — Харбин
• Chinese PLA Eastern Theater General Hospital — Нанкин
• Shengjing Hospital of China Medical University — Шэньян
• The First Affiliated Hospital of Xi'an Jiao Tong University — Сиань

Идентификаторы

NCT: NCT06627309 · 2023PHB319-001-02

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

Открыть это исследование на ClinicalTrials.gov ↗