Rapid DFLC Response Predict CHR in AL Amyloidosis

Recruiting

• Conditions: Systemic AL Amyloidosis
• Interventions: Drug: Dara IV; Drug: Bortezomib (drug); Drug: Dexamethasone; Drug: Cyclophosphamide (CTX); Drug: Dara SC

• Registration Number: NCT06627309
• Lead Sponsor: Peking University People's Hospital

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2024-09-29
• Status Verified: 2024-10
• Study First Submitted QC: 2024-10-03
• Study First Posted: 2024-10-04
• Study Start: 2024-10-26
• Last Update Submitted: 2024-10-26
• Last Update Posted: 2024-10-30
• Primary Completion: 2025-12-31
• Study Completion: 2026-07-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 50

Inclusion Criteria

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

Exclusion Criteria

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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Group 1	Cyclophosphamide (CTX)	All patients in this cohort receive daratumumab, bortezomib and dexamethasone as treatment. All patients receive additional sFLC examination in C1D7, C1D14.
Group 1	Dara IV	All patients in this cohort receive daratumumab, bortezomib and dexamethasone as treatment. All patients receive additional sFLC examination in C1D7, C1D14.
Group 1	Bortezomib (drug)	All patients in this cohort receive daratumumab, bortezomib and dexamethasone as treatment. All patients receive additional sFLC examination in C1D7, C1D14.
Group 1	Dara SC	All patients in this cohort receive daratumumab, bortezomib and dexamethasone as treatment. All patients receive additional sFLC examination in C1D7, C1D14.
Group 1	Dexamethasone	All patients in this cohort receive daratumumab, bortezomib and dexamethasone as treatment. All patients receive additional sFLC examination in C1D7, C1D14.

Primary Outcome Measures

Name	Time	Method
Complete hematologic response	6 months

Secondary Outcome Measures

Name	Time	Method
Overall hematologic response	6 months
At least one organ response	12 months
Minimal residual disease	6 months, 12 months	Bone marrow miminal residual disease
TTNT	12 months	Time to next treatment
MOD-EFS	12 months	Major Organ Deterioration Event-Free Survival (MOD-EFS) is defined as the time from the beginning of treatment to hematologic progression, clinical manifestation of end-stage cardiac or renal disease, initiation of subsequent other anti-plasma cell therapy, or death, whichever occur first
MOD-PFS	12 months	Major organ deterioration progression-free survival (MOD-PFS) is defined as the time from the beginning of treatment to death, clinical manifestation of end-stage cardiac or renal failure, or hematologic progression, whichever occur first

Trial Locations

Locations (2)

Fuxing Hospital affiliated to Capital Medical University; 🇨🇳 Beijing, Beijing, China

Peking University People's Hospital; 🇨🇳 Beijing, Beijing, China