Wiskott-Aldrich Syndrome Clinical Trial
Official title:
Randomized, Two-arm Single-center Phase II Clinical Trial Comparing the Efficacy and Safety of Romiplostim Versus Eltrombopag in the Treatment of Thrombocytopenia in Patients With Wiskott-Aldrich Syndrome
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
Status | Recruiting |
Enrollment | 30 |
Est. completion date | July 30, 2023 |
Est. primary completion date | December 2022 |
Accepts healthy volunteers | No |
Gender | All |
Age group | N/A to 18 Years |
Eligibility |
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 |
Country | Name | City | State |
---|---|---|---|
Russian Federation | Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology | Moscow |
Lead Sponsor | Collaborator |
---|---|
Federal Research Institute of Pediatric Hematology, Oncology and Immunology |
Russian Federation,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Percentage of patients with overall platelet response (complete response + partial response) for romiplostim and eltrombopag group | A complete response will be defined as a platelet count >100 x 109/L, partial - 30 x 109/L higher than the patient's pretreatment baseline count to 100 x 109/L. | 1 month (30 day +/- 7 days) | |
Secondary | Number of participants with bleeding events and severity of bleeding in romiplostim and eltrombopag group | The incidence and severity of bleeding events is evaluated with a modified World Health Organization (WHO) Bleeding Scale. (G1=Petechiae, epistaxis <30 min, G2=Mild blood loss, hematomas, epistaxis >30 min, melanotic stool G3=Gross blood loss, requiring blood transfusions, G4=Fatal bleeding). |
until discontinuation, from at least one month to one year | |
Secondary | Number of participants with drug related adverse events in each treatment group | Adverse events are graded according to Common Terminology Criteria for Adverse Events (CTCAE). | until discontinuation, from at least one month to one year | |
Secondary | Percentage of patients with overall platelet response (complete response + partial response) after switching from one TPO-RA to another one. | until discontinuation, from at least one month to one year |
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