Prospective Psychometric Evaluation Study of a Patient-reported Outcomes (PRO) Instrument for Congenital Thrombotic Thrombocytopenic Purpura (cTTP, Upshaw-Schulman Syndrome [USS], Hereditary Thrombotic Thrombocytopenic Purpura [hTTP]

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

41 participants

Study Classification

OBSERVATIONAL

Study Start Date

2018-07-12

Study Completion Date

2019-04-08

Brief Summary

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The purpose of this study is to assess the psychometric properties of a recently developed congenital thrombotic thrombocytopenic purpura (cTTP)-specific patient-reported outcomes (PRO) instrument.

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Detailed Description

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Conditions

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Congenital Thrombotic Thrombocytopenic Purpura

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Participants with cTTP - adolescents

Adolescents aged 12 to 17 years

No intervention

Intervention Type OTHER

This is a non-interventional study.

Participants with cTTP - adults

Adults aged ≥18 years

No intervention

Intervention Type OTHER

This is a non-interventional study.

Interventions

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No intervention

This is a non-interventional study.

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

1. The participant is an adolescent aged 12 to 17 years or an adult aged ≥18 years;
2. The participant has been diagnosed with congenital thrombotic thrombocytopenic purpura (cTTP) and is currently receiving prophylactic or on-demand treatment with fresh frozen plasma (FFP), or solvent detergent (S/D) plasma, or Von Willebrand Factor/Factor VIII (VWF/FVIII) concentrates;
3. In the instance that the participant is identified and recruited remotely (Direct-to-Patient (DtP) recruitment), access to the internet and possession of an internet-connecting device is required;
4. The participant has provided informed consent, and in the instance that the participant is an adolescent, a legal guardian has provided informed consent and the adolescent has provided assent.

Exclusion Criteria

1. The participant cannot read nor write;
2. The participant is non-English speaking;
3. The participant is currently participating in a clinical trial.

Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No