Romiplostim Treatment for Thrombocytopenia in Patients With Wiskott-Aldrich Syndrome.
Retrospective Chart Review of Children With Wiskott-Aldrich Syndrome Who Received Romiplostim in Treatment of Thrombocytopenia.
1 other identifier
observational
67
1 country
1
Brief Summary
The rationale for this retrospective study is to evaluate the efficacy and safety of thrombopoietin-receptor agonist (TPO-RA) romiplostim for reducing thrombocytopenia and bleeding tendency in pediatric participants with genetically confirmed Wiskott-Aldrich syndrome (WAS).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for all trials
Started Apr 2012
Longer than P75 for all trials
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
April 1, 2012
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 27, 2019
CompletedFirst Submitted
Initial submission to the registry
April 10, 2020
CompletedFirst Posted
Study publicly available on registry
April 16, 2020
CompletedStudy Completion
Last participant's last visit for all outcomes
June 1, 2020
CompletedDecember 28, 2020
December 1, 2020
7.7 years
April 10, 2020
December 24, 2020
Conditions
Outcome Measures
Primary Outcomes (1)
The percentage of participants with overall platelet response (complete response + partial response)
A complete response 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 +/- 14 days)
Secondary Outcomes (3)
Percentage of patients with a platelet response
until discontinuation, from at least one month to one year
Number of participants with bleeding events and severity of bleeding
until discontinuation, from at least one month to one year
Number of participants with adverse events
until discontinuation, from at least one month to one year
Study Arms (1)
treatment
romiplostim once weekly subcutaneously at an initial dose of 8-9 µg/kg per week for at least 1 month to 1 year.
Interventions
romiplostim once weekly subcutaneously at an initial dose of 8-9 µg/kg per week for at least 1 month to 1 year.
Eligibility Criteria
patients of the immunology department
You may qualify if:
- Age under \< 18 years
- Subject/legal representative has signed written informed consent. ?
- Subjects diagnosed with WAS based on genetic findings.
- Subjects with thrombocytopenia (platelet count of less than 70 x 109/L).
- Subjects with a history of bleeding.
- Subjects received treatment with romiplostim 8-9 µg /kg for at least 30 days
- Available records of the points of analysis
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology
Moscow, 117997, Russia
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Anna Shcherbina, MD, PhD
National Research Center for Pediatric Hematology , Moscow, Russian Federation
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- RETROSPECTIVE
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
April 10, 2020
First Posted
April 16, 2020
Study Start
April 1, 2012
Primary Completion
December 27, 2019
Study Completion
June 1, 2020
Last Updated
December 28, 2020
Record last verified: 2020-12