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

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

Status

UNKNOWN

Phases

Phase 1

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT05546060

Enrollment

Registered

2022-09-19

Start date

2022-07-01

Completion date

2025-10-01

Last updated

2022-09-19

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hemophagocytic Lymphohistiocytosis

Keywords

Hemophagocytic Lymphohistiocytosis, Venetoclax

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.

Interventions

DRUGVenetoclax

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

DRUGDexamethasone

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

DRUGEtoposide

Etoposide 75mg/m2 qw d1-56

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

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.

Design outcomes

Primary

Measure	Time frame	Description
Overall response rate	1 year	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	1 years	The percentage of cases with adverse effects and its severity.

Secondary

Measure	Time frame	Description
Overall survival	1 years	OS is defined as the time from the initiaion of treatment to death from any cause or July 2025.

Countries

China

Contacts

Primary ContactZhao Wang, MD

wangzhao@ccmu.edu.cn63138303

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026