Rapid dFLC Response Predict CHR in AL Amyloidosis
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Dara IV, Bortezomib (drug), Dexamethasone, Cyclophosphamide (CTX).
- Who it may be relevant to
- Registry conditions: Systemic AL Amyloidosis. Basic parameters: 18 years — 99 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- China
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
Rapid dFLC Response Predict Complete Hematologica Response in Systemic AL Amyloidosis Patients Treated With Daratumumab-based Regimen
Overview
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.
Interventions
- Drug 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. - Drug 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 - Drug 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. - Drug Cyclophosphamide (CTX)
Some patients may receive cyclophosphamide orally or intravenously at a dosage of 300 mg/m2 weekly. - Drug 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.
Primary outcome measures
- Complete hematologic response [Time frame: 6 months]
Secondary outcome measures (6)
- Overall hematologic response [Time frame: 6 months]
- At least one organ response [Time frame: 12 months]
- Minimal residual disease [Time frame: 6 months, 12 months]
- TTNT [Time frame: 12 months]
- MOD-EFS [Time frame: 12 months]
- MOD-PFS [Time frame: 12 months]
Eligibility criteria
Inclusion criteria
- 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;
Exclusion criteria
- 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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
China · 8 centers
- Peking University People's Hospital — Beijing
- Fuxing Hospital affiliated to Capital Medical University — Beijing
- Beijing Anzhen Hospital — Beijing
- Peking University First Hospital — Beijing
- First Affiliated Hospital of Harbin Medical University — Harbin
- Chinese PLA Eastern Theater General Hospital — Nanjing
- Shengjing Hospital of China Medical University — Shenyang
- The First Affiliated Hospital of Xi'an Jiao Tong University — Xi'an
Identifiers
NCT: NCT06627309 · 2023PHB319-001-02