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

NCT05263193 | Completed FDA Drug

• 1 other identifier: OBS17325
• Study Type: observational
• Target: 4
• Locations: 1 country
• Sites: 1

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.

Conditions

Immune-mediated Thrombocytopenic Purpura

Outcome Measures

Primary Outcomes (10)

• 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 Outcomes (2)

• Treatment pattern of caplacizumab therapy

  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

Interventions

Caplacizumab DRUG

as in real world practice

Also known as: Cablivi®

Eligibility Criteria

• Age: Up to 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

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

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Sanofi: lead

Study Sites (1)

Sanofi-Aventis

Chilly-Mazarin, 91380, France

Location

MeSH Terms

Interventions

caplacizumab

Study Officials

• Clinical Sciences and Operations

  Sanofi

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: CASE CONTROL
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 22, 2022
• First Posted: March 2, 2022
• Study Start: June 8, 2022
• Primary Completion: October 28, 2022
• Study Completion: October 28, 2022
• Last Updated: December 14, 2022

Record last verified: 2022-12

Data Sharing

• IPD Sharing: Will share

Locations

FR (1)