Multi-center Phase 2 clinical trial for EB virus associated hemophagocytic lymphohistiocytosis of pediatric and adolescent and young adult

JRCT ID: jRCTs041190053

Registered date:10/07/2019

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: EB virus associated hemophagocytic lymphohistiocytosis
• Date of first enrollment: 22/07/2019
• Target sample size: 52
• Study type: Interventional
• Intervention(s): Based on steroid responsiveness, age and EBV DNA load 2 weeks after the start of the study treatment, the treatment was stratified into low risk (LR), intermediate risk (IR) and high risk (HR) groups, with LR groups Treatment with steroids only, treatment with etoposide (VP-16) reduced compared to conventional therapy in the IR and HR groups, and rituximab in the HR group.

Outcome(s)

• Primary Outcome: 1 year event free survival of patients treated with VP-16 (Intermediate risk or high risk patients)

Secondary Outcome

1. 1 year event free survival by risk group (low risk, intermediate risk, or high risk)
2. 1 year event overall survival by risk group
3. Complete remission rate by risk group
4. Incidence of adverse event (therapy-rerated severe toxicity) by risk group

Key inclusion & exclusion criteria

• Age minimum: >=
• Age maximum: < 18age old
• Gender: Both
• Include criteria: Newly diagnosed and untreated patients under 18 years old with EB virus associated hemophagocytic lymphohistiocytosis

Exclude criteria

1. < EBV-DNA 1000 copies/microgramDNA
2. Familial hemophagocytic lymphohistiocytosis or X linked lymphoproliferative syndrome
3. EBV HLH due to EB virus reactivation
4 .Coexistence of active cancer
5 .Uncontrolled organ dysfunction

Related Information

• Primary Sponsor: Sakamoto Kenichi
• Source(s) of Monetary Support: Japan Agency for Medical Research and Development,Friends of Leukemia Research Fund