Clinical trial NCT05263193: Retrospective Study on Caplacizumab-treated Pediatric Patients With Immune-mediated Thrombocytopenic Purpura (iTTP)
Official title: Retrospective Data Collection of Pediatric Patients With Immune-mediated Thrombotic Thrombocytopenic Purpura (iTTP) Treated With Caplacizumab
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Immune-mediated Thrombocytopenic Purpura
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2022-03-02
Enrollment: 4
Sex: ALL
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: CASE_CONTROL
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Pediatric patients with Immune-mediated Thrombotic Thrombocytopenic Purpura treated with Caplacizumab

Brief summary: 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.

Detailed description: Pediatric patients who received caplacizumab will be identified for enrollment in the chart review. The eligibility period starts on August 30, 2018 in the United Kingdom (UK) and France and February 6, 2019 for the United States (US).

Data collection is fully retrospective and will be anchored to the patient's index event date. The index event date is defined as the date the patient initiated caplacizumab treatment. The study period begins at the index date and ends at the earliest date of chart abstraction initiation, 12 weeks after last dose of caplacizumab treatment, date of death, or loss to follow-upwhich ever comes first .

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

Primary outcomes:
measure: Platelet count response
timeFrame: From index date up to 12 weeks after last dose of caplacizumab
measure: Proportion of subjects with refractory iTTP
timeFrame: From index date up to 12 weeks after last dose of caplacizumab
measure: Proportion of subjects with recurrent disease
timeFrame: From index date up to 12 weeks after last dose of caplacizumab
measure: Time to normalization of organ damage marker levels
timeFrame: From index date up to 12 weeks after last dose of caplacizumab
measure: Total duration of hospitalization stays
timeFrame: From index date up to 12 weeks after last dose of caplacizumab
measure: Duration of intensive care unit (ICU) stay
timeFrame: From index date up to 12 weeks after last dose of caplacizumab
measure: Duration of therapeutic PE
timeFrame: From index date up to 12 weeks after last dose of caplacizumab
measure: Proportion of patients achieving clinical response
timeFrame: From index date up to 12 weeks after last dose of caplacizumab
measure: Time to ADAMTS13 activity ≥ 20%
timeFrame: From index date up to 12 weeks after last dose of caplacizumab
measure: Number of participants with Adverse event
timeFrame: From index date up to 12 weeks after last dose of caplacizumab
Secondary outcomes:
measure: Treatment pattern of caplacizumab therapy
timeFrame: From index date up to 12 weeks after last dose of caplacizumab
measure: Types and duration of concomitant medications
timeFrame: From index date up to 12 weeks after last dose of caplacizumab
Other outcomes:
Locations:
city: Chilly-Mazarin
country: France
facility: Sanofi-Aventis
Study officials:
affiliation: Sanofi
name: Clinical Sciences and Operations
role: STUDY_DIRECTOR
References: