Bortezomib, Pomalidomide, Dexamethasone for Systemic AL Amyloidosis

Официальное название

A Study of Bortezomib, Pomalidomide, Dexamethasone in Patients with Systemic AL Amyloidosis

Обзор

This is an open-label, multicenter, Phase 2 study in subjects with newly diagnosed or previous treated systemic light chain (AL) amyloidosis. Approximately 40 subjects will receive therapy with bortezomib, pomalidomide, and dexamethasone. The primary outcome is hematologic very good partial response and complete response rate at 6 months.

Подробное описание

Proteasome inhibitor and immunmodulators have synergistic effect for plasma cell dyscrasia. Due to the fact that more than 70-80% of patients with amyloidosis have renal involvement, the application of lenalidomide is limited. Thus, the investigators designed this open-label, multicenter, phase 2 study for newly diagnosed or previously treated systemic AL amyloidosis with bortezomib, pomalidomide and dexamethasone regimen.

Вмешательства

• Препарат Bortezomib
  Bortezomib 1.3mg/m2 sc weekly
• Препарат Pomalidomide
  4mg per day taken orally on 1-21 of repeated 28-day cycles
• Препарат Dexamethasone
  Dexamethasone 20mg-40mg weekly

Первичные конечные точки

• Hematologic VGPR + CR rate at 6 months [Срок оценки: 6 months]

Вторичные конечные точки (6)

• Overall Hematologic response rate at 6 months [Срок оценки: 6 months]
• At least one organ response at 6 months [Срок оценки: 6 months]
• TTNT at 2 years [Срок оценки: 2 years]
• Estimated PFS at 2 years [Срок оценки: 2 years]
• Estimated OS at 2 years [Срок оценки: 2 years]
• TRAE [Срок оценки: 6 months]

Критерии участия

Критерии включения

• Diagnosis of AL amyloidosis, confirmed by histology and typed with immunohistochemistry, immunoelectron microscopy or mass spectrometry.
• Newly diagnosed or previous treated AL amyloidosis
• Patients must be ≥ 18 years of age.
• ECOG performance status 0, 1 or 2.
• Measurable disease defined by at least one of the following:

① serum free light chain (FLC) ≥2.0 mg/dL (20 mg/L) with an abnormal kappa:lambda ratio or the difference between involved and uninvolved free light chains (dFLC) ≥2mg/dL (20 mg/L).

②. presence of a monoclonal spike that is ≥5 g/l.

• Symptomatic organ involvement (heart, kidney, liver/GI tract, peripheral nervous system).
• Absolute neutrophil count (ANC) ≥1.0 X 10\^9/L, Hemoglobin ≥70 g/L, Platelets ≥50 X 10\^9/L
• eGFR ≥20 mL/min/ 1.73 m\^2
• Written informed consent in accordance with local and institutional guidelines.
• Contraceptive use should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.

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

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Китай · 1 центр

• Peking University People's Hospital — Пекин

Идентификаторы

NCT: NCT06342466 · 2023-358-02

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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