Rapid DFLC Response Predict CHR in AL Amyloidosis

NCT ID: NCT06627309

Last Updated: 2024-10-30

Basic Information

• Recruitment Status: RECRUITING
• Total Enrollment: 50 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2024-10-26
• Study Completion Date: 2026-07-01

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.

Conditions

Systemic AL Amyloidosis

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

Group 1

All patients in this cohort receive daratumumab, bortezomib and dexamethasone as treatment.

All patients receive additional sFLC examination in C1D7, C1D14.

Dara IV

Intervention Type: DRUG

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.

Bortezomib (drug)

Intervention Type: 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

Dexamethasone

Intervention Type: DRUG

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.

Cyclophosphamide (CTX)

Intervention Type: DRUG

Some patients may receive cyclophosphamide orally or intravenously at a dosage of 300 mg/m2 weekly.

Dara SC

Intervention Type: DRUG

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.

Interventions

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.

Intervention Type: 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

Intervention Type: 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.

Intervention Type: DRUG

Cyclophosphamide (CTX)

Some patients may receive cyclophosphamide orally or intravenously at a dosage of 300 mg/m2 weekly.

Intervention Type: 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.

Intervention Type: DRUG

Eligibility Criteria

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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 99 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Peking University People's Hospital

OTHER

Sponsor Role: lead

Responsible Party

Jin Lu, MD

Principal of Investigator

Responsibility Role: PRINCIPAL_INVESTIGATOR

Locations

Peking University People's Hospital

Beijing, Beijing Municipality, China

Site Status: RECRUITING

Fuxing Hospital affiliated to Capital Medical University

Beijing, Beijing Municipality, China

Site Status: RECRUITING

Countries

China

Central Contacts

Yang Liu, M.D.

Role: CONTACT

pkuphliuyang@bjmu.edu.cn

+86-13716926210

Facility Contacts

Liru Wang

Role: primary

wanglirumail@126.com

86-18618238625

Other Identifiers

2023PHB319-001-02

Identifier Type: -

Identifier Source: org_study_id