CompletedNot Applicable

Romiplostim Treatment for Thrombocytopenia in Patients With Wiskott-Aldrich Syndrome.

Russia67 participantsStarted 2012-04-01

Plain-language 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).

Who can participate

Age range18 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * 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 Exclusion Criteria: • Patients, who do not meet the inclusion criteria.

What they're measuring

The percentage of participants with overall platelet response (complete response + partial response)

Timeframe: 1 month (30 day +/- 14 days)

Trial details

NCT IDNCT04350164

SponsorFederal Research Institute of Pediatric Hematology, Oncology and Immunology

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2019-12-27

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