Combining venetoclax with dexamethasone and etoposide for treating HLH
Efficacy and Safety of Venetoclax Combined With Dexamethasone and Etoposide in the Treatment of Hemophagocytic Lymphohistiocytosis
This study is testing a new combination of medications for people with hemophagocytic lymphohistiocytosis (HLH) who haven't improved with standard treatments to see if it helps them feel better.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 20 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Beijing Friendship Hospital Academic / other |
| Locations | 1 site (Beijing, Beijing) |
| Trial ID | NCT05546060 on ClinicalTrials.gov |
What this trial studies
This study investigates the efficacy and safety of a combination therapy using venetoclax, dexamethasone, and etoposide for patients with hemophagocytic lymphohistiocytosis (HLH) who have not responded to standard treatments. The trial aims to enroll 20 patients who meet specific diagnostic criteria and will receive the treatment for 8 weeks, with safety and efficacy evaluations conducted throughout the study. The overall response rate will be assessed at the end of the treatment period, and patients will be followed up every three months thereafter.
Who should consider this trial
Good fit: Ideal candidates are adults aged 18 and older with refractory or recurrent HLH who meet specific diagnostic and health criteria.
Not a fit: Patients with unrelated malignancies, severe infections, or those who have previously been treated with Bcl-2 inhibitors may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could provide a new salvage therapy option for patients with refractory HLH.
How similar studies have performed: While there is limited evidence on this specific combination, other studies have explored similar approaches in treating HLH, indicating potential for success.
Eligibility criteria
Show full inclusion / exclusion 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.
Where this trial is running
Beijing, Beijing
- Beijing Friendship Hospital, Capital Medical University — Beijing, Beijing, China (Recruiting)
Study contacts
- Principal investigator: Zhao Wang, MD — Beijing Friendship Hospital
- Study coordinator: Zhao Wang, MD
- Email: wangzhao@ccmu.edu.cn
- Phone: 63138303
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.