Efficacy and Safety of Romiplostim Versus Eltrombopag in the Treatment of Thrombocytopenia in Patients With Wiskott-Aldrich Syndrome

• Status: Recruiting
• Trial end date: 2023-07-30
• Target enrollment: 0
• Participant gender: All

Summary

This is a prospective, open-label, randomized, two-arm clinical trial conducted to evaluate the safety and efficacy of romiplostim in comparison with eltrombopag in the treatment of thrombocytopenia in patients with Wiskott-Aldrich syndrome

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Federal Research Institute of Pediatric Hematology, Oncology and Immunology

Criteria

Inclusion Criteria:

• Genetically verified Wiskott-Aldrich syndrome

• Thrombocytopenia (platelet count < 70 x 109/L)

• Age: under 18 years

• Subject/legal representative has signed written informed consent.

Exclusion Criteria:

• Patients, who do not meet the inclusion criteria.

• Any prior history of arterial or venous thrombosis within the past year.

• Arm II (eltrombopag):

1. abnormal hepatic function -elevated AST/ALT > 1.5 times upper limit of normal within 4 weeks prior to enrollment

2. Active colitis