Prospective Psychometric Evaluation Study of a Patient-reported Outcomes (PRO) Instrument for Con… (NCT03519672) | Clinical Trial Compass
CompletedNot Applicable
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]
United States, United Kingdom41 participantsStarted 2018-07-12
Plain-language summary
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.
Who can participate
Age range12 Years
SexALL
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Inclusion criteria
✓. The participant is an adolescent aged 12 to 17 years or an adult aged ≥18 years;
✓. 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;
✓. 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;
✓. 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
✕. The participant cannot read nor write;
✕. The participant is non-English speaking;
✕. The participant is currently participating in a clinical trial.
What they're measuring
1
Change from baseline of Quality of life (QoL) outcomes: cTTP-specific PRO instrument
Timeframe: Day 1, and Day 14
2
Change from baseline of Quality of life (QoL) outcomes: PROMIS®-29
Timeframe: Day 1, and Day 14
3
Change from baseline of Quality of life (QoL) outcomes: Pediatric PROMIS® Subscales for adolescents
Timeframe: Day 1, and Day 14
4
Change from baseline of Quality of life (QoL) outcomes: HIT-6
Timeframe: Day 1, and Day 14
5
Change from baseline of Quality of life (QoL) outcomes: Condensed MCMDM-1VWD Bleeding Questionnaire: Bruising Subscale
Timeframe: Day 1, and Day 14
6
Change from baseline of Quality of life (QoL) outcomes: PDQ-5
Timeframe: Day 1, and Day 14
7
Change from baseline of Quality of life (QoL) outcomes: NEI-VFQ-25
Timeframe: Day 1, and Day 14
8
Change from baseline of Quality of life (QoL) outcomes: WPAI-GH