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Clinical trial

Retrospective Data Collection of Pediatric Patients With Immune-mediated Thrombotic Thrombocytopenic Purpura (iTTP) Treated With Caplacizumab

ID
Name
OBS17325
Description
The purpose of this multi-country, retrospective data collection study (chart review) is to describe the effectiveness and safety of caplacizumab in pediatric patients with iTTP.
Trial arms
Trial start
2022-06-08
Estimated PCD
2022-10-28
Trial end
2022-10-28
Status
Completed
Treatment
Caplacizumab
as in real world practice
Other names:
Cablivi®
Size
4
Primary endpoint
Platelet count response
From index date up to 12 weeks after last dose of caplacizumab
Proportion of subjects with refractory iTTP
From index date up to 12 weeks after last dose of caplacizumab
Proportion of subjects with recurrent disease
From index date up to 12 weeks after last dose of caplacizumab
Time to normalization of organ damage marker levels
From index date up to 12 weeks after last dose of caplacizumab
Total duration of hospitalization stays
From index date up to 12 weeks after last dose of caplacizumab
Duration of intensive care unit (ICU) stay
From index date up to 12 weeks after last dose of caplacizumab
Duration of therapeutic PE
From index date up to 12 weeks after last dose of caplacizumab
Proportion of patients achieving clinical response
From index date up to 12 weeks after last dose of caplacizumab
Time to ADAMTS13 activity ≥ 20%
From index date up to 12 weeks after last dose of caplacizumab
Number of participants with Adverse event
From index date up to 12 weeks after last dose of caplacizumab
Eligibility criteria
Inclusion Criteria: * Patient's aged ≤18 years at start of caplacizumab treatment initiation * Patient has a diagnosis of iTTP documented in the medical records * Patient was treated with caplacizumab within the eligibility period Exclusion Criteria: * Patient declined use of data for study (where local regulations require patient notification of planned study) * Patient's medical chart is missing or not retrievable
Protocol
{'studyType': 'OBSERVATIONAL', 'patientRegistry': False, 'designInfo': {'observationalModel': 'CASE_CONTROL', 'timePerspective': 'RETROSPECTIVE'}, 'enrollmentInfo': {'count': 4, 'type': 'ACTUAL'}}
Updated at
2022-12-14

1 organization

1 product

1 indication

Organization
Sanofi
Product
Caplacizumab
Indication
Immune Thrombocytopenic Purpura