search
Back to results

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

Primary Purpose

Light Chain (AL) Amyloidosis, CCND1 Translocation, Venetoclax

Status
Recruiting
Phase
Phase 2
Locations
China
Study Type
Interventional
Intervention
Venetoclax
Dexamethasone Oral
Sponsored by
Peking Union Medical College Hospital
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Light Chain (AL) Amyloidosis

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Peking Union Medical College HospitalRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Ven-D

Arm Description

Venetoclax combined with dexamethasone

Outcomes

Primary Outcome Measures

Complete response (CR)+very good partial response (VGPR) at 3 months after treatment initiation

Secondary Outcome Measures

Overall survival
Time to next treatment
CR+VGPR at 1 month after treatment initiation
CR+VGPR at 6 months after treatment initiation
CR+VGPR at 12 months after treatment initiation
Difference between involved and uninvolved free light chain (dFLC) < 10mg/L
Involved free light chain (iFLC) ≤ 20mg/L
Minimal residual disease (MRD) negativity
Time to hematologic response
Time to hematologic CR
Cardiac response
Renal response
Hepatic response
Time to cardiac response
Time to renal response
Time to hepatic response
Adverse events

Full Information

First Posted
August 9, 2023
Last Updated
September 7, 2023
Sponsor
Peking Union Medical College Hospital
search

1. Study Identification

Unique Protocol Identification Number
NCT05996406
Brief Title
Venetoclax and Dexamethasone for Newly Diagnosed Light-Chain Amyloidosis With Translocation (11;14)
Official Title
Venetoclax Combined With Dexamethasone for Newly Diagnosed Light-Chain Amyloidosis Patients With Translocation (11;14): A Multicenter Phase 2 Study
Study Type
Interventional

2. Study Status

Record Verification Date
September 2023
Overall Recruitment Status
Recruiting
Study Start Date
September 7, 2023 (Actual)
Primary Completion Date
December 2024 (Anticipated)
Study Completion Date
September 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Peking Union Medical College Hospital

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Light Chain (AL) Amyloidosis, CCND1 Translocation, Venetoclax

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
36 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Ven-D
Arm Type
Experimental
Arm Description
Venetoclax combined with dexamethasone
Intervention Type
Drug
Intervention Name(s)
Venetoclax
Intervention Description
Venetoclax 400mg po qd for 1 year
Intervention Type
Drug
Intervention Name(s)
Dexamethasone Oral
Intervention Description
Dexamethasone 40mg po qw for the first 6 months, then 10mg po qw for the next 6 months
Primary Outcome Measure Information:
Title
Complete response (CR)+very good partial response (VGPR) at 3 months after treatment initiation
Time Frame
3 months after treatment initiation
Secondary Outcome Measure Information:
Title
Overall survival
Time Frame
2 years
Title
Time to next treatment
Time Frame
2 years
Title
CR+VGPR at 1 month after treatment initiation
Time Frame
1 month after treatment initiation
Title
CR+VGPR at 6 months after treatment initiation
Time Frame
6 months after treatment initiation
Title
CR+VGPR at 12 months after treatment initiation
Time Frame
12 months after treatment initiation
Title
Difference between involved and uninvolved free light chain (dFLC) < 10mg/L
Time Frame
at 1, 3, 6 and 12 months after treatment initiation
Title
Involved free light chain (iFLC) ≤ 20mg/L
Time Frame
at 1, 3, 6 and 12 months after treatment initiation
Title
Minimal residual disease (MRD) negativity
Time Frame
12 and 24 months after treatment initiation
Title
Time to hematologic response
Time Frame
1 year
Title
Time to hematologic CR
Time Frame
1 year
Title
Cardiac response
Time Frame
at 3, 6, 12 and 24 months after treatment initiation
Title
Renal response
Time Frame
at 3, 6, 12 and 24 months after treatment initiation
Title
Hepatic response
Time Frame
at 3, 6, 12 and 24 months after treatment initiation
Title
Time to cardiac response
Time Frame
2 years
Title
Time to renal response
Time Frame
2 years
Title
Time to hepatic response
Time Frame
2 years
Title
Adverse events
Time Frame
treatment initiation to 30 days after last dose of treatment

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Kaini Shen, Dr.
Phone
+86 13693339884
Email
shenkaini3@sina.com
First Name & Middle Initial & Last Name or Official Title & Degree
Jian Li
Phone
+86 18610852525
Email
lijian@pumch.cn
Facility Information:
Facility Name
Peking Union Medical College Hospital
City
Beijing
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Kaini Shen
Email
shenkaini3@sina.com

12. IPD Sharing Statement

Learn more about this trial

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

We'll reach out to this number within 24 hrs