Venetoclax and Dexamethasone for Newly Diagnosed Light-Chain Amyloidosis with Translocation (11;14)

NCT ID: NCT05996406

Last Updated: 2025-01-22

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 36 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-09-07
• Study Completion Date: 2025-09-30

Brief Summary

Venetoclax is considered as a promising agent for light-chain (AL) amyloidosis due to the high percentage of t(11;14). Several retrospective studies showed venetoclax-based therapy could induce rapid and profound hematologic response in AL patients with favorable safety profile. As an oral agent with encouraging data, it is worth to prospectively evaluate the efficacy and safety of venetoclax in untreated AL amyloidosis patients.

Conditions

Light Chain (AL) Amyloidosis; CCND1 Translocation; Venetoclax

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Ven-D

Venetoclax combined with dexamethasone

Group Type: EXPERIMENTAL

Venetoclax

Intervention Type: DRUG

Venetoclax 400mg po qd for 1 year

Dexamethasone Oral

Intervention Type: DRUG

Dexamethasone 40mg po qw for the first 6 months, then 10mg po qw for the next 6 months

Interventions

Venetoclax

Venetoclax 400mg po qd for 1 year

Intervention Type: DRUG

Dexamethasone Oral

Dexamethasone 40mg po qw for the first 6 months, then 10mg po qw for the next 6 months

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• Biopsy proved treatment-naïve AL amyloidosis
• Fluorescence in situ hybridization (FISH) t(11;14) ≥ 10%
• dFLC > 50mg/L

Exclusion Criteria

• Co-morbidity of uncontrolled infection
• Co-morbidity of other active malignancy
• Co-diagnosis of multiple myeloma or waldenstrom macroglobulinemia
• Co-morbidity of grade 2 Mobitz II or grade 3 atrioventricular block (expect for those with implanted pacemaker)
• Co-morbidity of sustained or recurrent nonsustained ventricular tachycardia
• Seropositive for human immunodeficiency virus
• Hepatitis B virus (HBV)-DNA > 1000 copies/mL
• Seropositive for hepatitis C (except in the setting of a sustained virologic response)
• Systemic treatment with moderate or strong cytochrome P450 3A （CYP3A） inducers, moderate or strong CYP3A inhibitors within 7 days prior to the first dose of study drug
• Neutrophil <1×10E9/L，hemoglobin < 8g/dL，or platelet < 100×10E9/L.
• Severely compromised hepatic or renal function: alanine transaminase (ALT) or aspertate aminotransferase (AST) > 2.5 × upper limit of normal (ULN), total bilirubin > 3 × ULN，eGFR < 15 mL/min, or receiving renal replacement therapy

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

Sponsors

Peking Union Medical College Hospital

OTHER

Sponsor Role: lead

Responsible Party

Jian Li

Professor

Responsibility Role: PRINCIPAL_INVESTIGATOR

Locations

Peking Union Medical College Hospital

Beijing, , China

Countries

China

Other Identifiers

Ven-D001

Identifier Type: -

Identifier Source: org_study_id