- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04350164
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.
Study Overview
Detailed Description
Thrombocytopenia is a life-threatening symptom in WAS patients. Subjects with WAS are at increased risk of debilitating and\ or life-threatening bleedings due to low platelet numbers. Hematopoietic stem cell transplantation is an effective treatment of WAS and all its symptoms yet requires time for donor search and is not widely utilized in cases with mild WAS with isolated thrombocytopenia. TPO-RAs have been used in individual WAS patients, wherein publications describing large WAS cohorts treated with TPO-RAs are lacking.
Based on the previous reports, WAS patients in our Center have been receiving treatment with TPO-RA romiplostim since 2012.
The aim of the study is to retrospective analyze patients' data in order to asses treatment efficacy and safety of romiplostim in WAS thrombocytopenia.
The study will collect and analyze information that is already in the patients' medical records. Information about clinical data (assessment of bleeding tendency with a modified World Health Organization (WHO) Bleeding Scale), laboratory values (such as clinical and biochemical analysis of blood) will be included.
Evaluation of the efficacy therapy was based on the results of physical examination, including bleeding events at the time of diagnosis and after 6-month TPO-RA was initiated and platelet response. A complete response was defined as a platelet count >100 x 109/L in the absence of bleeding symptoms, partial - 30 x 109/L higher than the patient's pretreatment baseline count to 100 x 109/L. Non-response was defined as not achieving a platelet count of > 30 x 109/L from the baseline count.
Study Type
Enrollment (Actual)
Contacts and Locations
Study Locations
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Moscow, Russian Federation, 117997
- Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Sampling Method
Study Population
Description
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.
Study Plan
How is the study designed?
Design Details
- Observational Models: Cohort
- Time Perspectives: Retrospective
Cohorts and Interventions
Group / Cohort |
Intervention / Treatment |
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treatment
romiplostim once weekly subcutaneously at an initial dose of 8-9 µg/kg per week for at least 1 month to 1 year.
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romiplostim once weekly subcutaneously at an initial dose of 8-9 µg/kg per week for at least 1 month to 1 year.
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
The percentage of participants with overall platelet response (complete response + partial response)
Time Frame: 1 month (30 day +/- 14 days)
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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.
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1 month (30 day +/- 14 days)
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Percentage of patients with a platelet response
Time Frame: until discontinuation, from at least one month to one year
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until discontinuation, from at least one month to one year
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Number of participants with bleeding events and severity of bleeding
Time Frame: until discontinuation, from at least one month to one year
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The incidence and severity of bleeding events 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
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Number of participants with adverse events
Time Frame: until discontinuation, from at least one month to one year
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until discontinuation, from at least one month to one year
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Collaborators and Investigators
Investigators
- Principal Investigator: Anna Shcherbina, MD, PhD, National Research Center for Pediatric Hematology , Moscow, Russian Federation
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Pathologic Processes
- Immunologic Deficiency Syndromes
- Immune System Diseases
- Disease
- Hematologic Diseases
- Blood Coagulation Disorders, Inherited
- Hemorrhagic Disorders
- Genetic Diseases, Inborn
- Genetic Diseases, X-Linked
- Blood Coagulation Disorders
- Blood Platelet Disorders
- Leukopenia
- Leukocyte Disorders
- Primary Immunodeficiency Diseases
- Lymphopenia
- Syndrome
- Thrombocytopenia
- Wiskott-Aldrich Syndrome
Other Study ID Numbers
- NCPHOI-2020-02
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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