Retrospective Study on Caplacizumab-treated Pediatric Patients With Immune-mediated Thrombocytopenic Purpura (iTTP)

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

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.

Study Overview

• Status: Completed
• Conditions: Immune-mediated Thrombocytopenic Purpura
• Intervention / Treatment: Drug: Caplacizumab

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 .

• Study Type: Observational
• Enrollment (Actual): 4

Contacts and Locations

Study Locations

• France
  • Chilly-Mazarin, France, 91380
    • Sanofi-Aventis

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 18 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Pediatric patients with Immune-mediated Thrombotic Thrombocytopenic Purpura treated with Caplacizumab

Description

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

Study Plan

How is the study designed?

Design Details

• Observational Models: Case-Control
• Time Perspectives: Retrospective

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Platelet count response	defined as time from caplacizumab initiation to initial platelet count ≥ 150×109/L with subsequent stop of daily plasma exchange (PE) within 5 days	From index date up to 12 weeks after last dose of caplacizumab
Proportion of subjects with refractory iTTP	defined as lack of doubling of platelet count after four days of caplacizumab treatment and a lactate dehydrogenase (LDH) level that remained above the upper limit of normal (ULN) range	From index date up to 12 weeks after last dose of caplacizumab
Proportion of subjects with recurrent disease	Proportion of subjects with iTTP exacerbation (defined as recurrence within 30 days after last PE) and Proportion of subjects with iTTP relapse (defined as recurrence more than 30 days after last PE)	From index date up to 12 weeks after last dose of caplacizumab
Time to normalization of organ damage marker levels	Defined asLDH ≤ 2 x ULN, Serum creatinine ≤ 1 x ULN, Cardiac troponin I ≤ 1 x ULN	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	defined as a normal platelet countand LDH < 2 ULN for at least 48 hours following initial normalization or response of platelet count	From index date up to 12 weeks after last dose of caplacizumab
Time to ADAMTS13 activity ≥ 20%	where available and feasible	From index date up to 12 weeks after last dose of caplacizumab
Number of participants with Adverse event	including serious adverse events	From index date up to 12 weeks after last dose of caplacizumab

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Treatment pattern of caplacizumab therapy	Dosing and duration	From index date up to 12 weeks after last dose of caplacizumab
Types and duration of concomitant medications		From index date up to 12 weeks after last dose of caplacizumab

Collaborators and Investigators

• Sponsor: Sanofi

Study record dates

Study Major Dates

• Study Start (Actual): June 8, 2022
• Primary Completion (Actual): October 28, 2022
• Study Completion (Actual): October 28, 2022

Study Registration Dates

• First Submitted: February 22, 2022
• First Submitted That Met QC Criteria: February 22, 2022
• First Posted (Actual): March 2, 2022

Study Record Updates

• Last Update Posted (Actual): December 14, 2022
• Last Update Submitted That Met QC Criteria: December 13, 2022
• Last Verified: December 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Immune System Diseases; Autoimmune Diseases; Hematologic Diseases; Hemorrhage; Hemorrhagic Disorders; Blood Coagulation Disorders; Skin Manifestations; Thrombocytopenia; Blood Platelet Disorders; Thrombotic Microangiopathies; Purpura; Purpura, Thrombocytopenic; Purpura, Thrombocytopenic, Idiopathic
• Other Study ID Numbers: OBS17325

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Yes
• IPD Plan Description: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No