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Efficacy and Safety of Venetoclax Combined With Dexamethasone and Etoposide in HLH

Primary Purpose

Hemophagocytic Lymphohistiocytosis

Status

Recruiting

Phase

Phase 1

Locations

China

Study Type

Interventional

Intervention

Venetoclax

Dexamethasone

Etoposide

About this trial

This is an interventional treatment trial for Hemophagocytic Lymphohistiocytosis focused on measuring Hemophagocytic Lymphohistiocytosis, Venetoclax

Eligibility Criteria

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

Inclusion Criteria:

age ≥18 years old, expected survival time more than 3 months;
met HLH-2004 diagnostic criteria；
ECOG score 0-2；
ECG QTcF interval: male ≤450ms, female ≤470ms;
AST and ALT ≤3.0 ULN, TB ≤1.5×ULN；serum creatinine≤1.5×ULN，or CrCL ≥ 50mL/min；INR、APTT、PT ≤1.5×ULN；
without pregnancy or lactation, and agree to contraception during and for at least 6 months after the study；
signed informed consent.

Exclusion Criteria:

patients with malignancies unrelated to HLH, except for fully recovered non-melanoma skin cancer and carcinoma in situ;
patients participated in other clinical trials within 4 weeks；
previously treated with Bcl-2 inhibitors；
unable to take oral medication；
history of substance abuse or patients with mental illness；
severe infection；
cardiovascular disease，NYHA II-IV；
allergic to venetoclax or etoposide.

Sites / Locations

Beijing Friendship Hospital, Capital Medical UniversityRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Venetoclax Combined With Dexamethasone and Etoposide

Arm Description

Patients who met the inclusion criteria will receive venetoclax combined with dexamethasone and etoposide for 8 weeks.

Outcomes

Primary Outcome Measures

Overall response rate

The percentage of cases with complete response (CR) and partial response (PR) after treatment in the total evaluable cases

Incidence and severity of adverse effects

The percentage of cases with adverse effects and its severity.

Secondary Outcome Measures

Overall survival

OS is defined as the time from the initiaion of treatment to death from any cause or July 2025.

Full Information

NCT ID

NCT05546060

First Posted

September 15, 2022

Last Updated

September 15, 2022

Sponsor

Beijing Friendship Hospital

1. Study Identification

Unique Protocol Identification Number

NCT05546060

Brief Title

Efficacy and Safety of Venetoclax Combined With Dexamethasone and Etoposide in HLH

Official Title

Efficacy and Safety of Venetoclax Combined With Dexamethasone and Etoposide in the Treatment of Hemophagocytic Lymphohistiocytosis

Study Type

Interventional

2. Study Status

Record Verification Date

September 2022

Overall Recruitment Status

Recruiting

Study Start Date

July 1, 2022 (Actual)

Primary Completion Date

July 1, 2025 (Anticipated)

Study Completion Date

October 1, 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Beijing Friendship Hospital

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

Brief Summary

This study aimed to investigate the efficacy and safety of venetoclax combined with dexamethasone and etoposide as a salvage therapy for hemophagocytic lymphohistiocytosis.

Detailed Description

Hemophagocytic lymphohistiocytosis (HLH) is a rare and aggressive disease with high mortality and poor prognosis. The HLH-94 and HLH-04 regimens are most commonly used, but approximately 30% of the patients remain unresponsive. Currently, there is no unified evidence-based salvage treatment. There is no unified salvage regimen. This study plans to enroll 20 patients with refractory and recurrent hemophagocytic lymphohistiocytosis. Subjects will receive venetoclax combined with dexamethasone and etoposide for 8 weeks.The safety was evaluated once a week, and the efficacy was evaluated once every 2 weeks. Overall response rate will be evaluated at 8 weeks. Patients were followed up every 3 months thereafter.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Hemophagocytic Lymphohistiocytosis

Keywords

Hemophagocytic Lymphohistiocytosis, Venetoclax

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

20 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Venetoclax Combined With Dexamethasone and Etoposide

Arm Type

Experimental

Arm Description

Patients who met the inclusion criteria will receive venetoclax combined with dexamethasone and etoposide for 8 weeks.

Intervention Type

Drug

Intervention Name(s)

Venetoclax

Intervention Description

Venetoclax 100mg qd d1，200mg qd d2，400mg qd d3-56

Intervention Type

Drug

Intervention Name(s)

Dexamethasone

Intervention Description

Dexamethasone 10mg/m2 qd d1-14, 5mg/m2 qd d15-28, 2.5 mg/m2 qd d29-42, and 1.25mg/m2 qd43-56

Intervention Type

Drug

Intervention Name(s)

Etoposide

Intervention Description

Etoposide 75mg/m2 qw d1-56

Primary Outcome Measure Information:

Title

Overall response rate

Description

The percentage of cases with complete response (CR) and partial response (PR) after treatment in the total evaluable cases

Time Frame

1 year

Title

Incidence and severity of adverse effects

Description

The percentage of cases with adverse effects and its severity.

Time Frame

1 years

Secondary Outcome Measure Information:

Title

Overall survival

Description

OS is defined as the time from the initiaion of treatment to death from any cause or July 2025.

Time Frame

1 years

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: age ≥18 years old, expected survival time more than 3 months; met HLH-2004 diagnostic criteria； ECOG score 0-2； ECG QTcF interval: male ≤450ms, female ≤470ms; AST and ALT ≤3.0 ULN, TB ≤1.5×ULN；serum creatinine≤1.5×ULN，or CrCL ≥ 50mL/min；INR、APTT、PT ≤1.5×ULN； without pregnancy or lactation, and agree to contraception during and for at least 6 months after the study； signed informed consent. Exclusion Criteria: patients with malignancies unrelated to HLH, except for fully recovered non-melanoma skin cancer and carcinoma in situ; patients participated in other clinical trials within 4 weeks； previously treated with Bcl-2 inhibitors； unable to take oral medication； history of substance abuse or patients with mental illness； severe infection； cardiovascular disease，NYHA II-IV； allergic to venetoclax or etoposide.

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Zhao Wang, MD

Phone

63138303

wangzhao@ccmu.edu.cn

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Zhao Wang, MD

Organizational Affiliation

Beijing Friendship Hospital

Official's Role

Principal Investigator

Facility Information:

Facility Name

Beijing Friendship Hospital, Capital Medical University

City

Beijing

State/Province

Beijing

ZIP/Postal Code

100050

Country

China

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Zhao Wang, MD

Phone

63138303

wangzhao@ccmu.edu.cn

12. IPD Sharing Statement

Plan to Share IPD

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Efficacy and Safety of Venetoclax Combined With Dexamethasone and Etoposide in HLH

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