Multiple Sclerosis Clinical Trial
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]
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.
Eligibility Criteria
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.
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There are 4 Locations for this study
Atlanta Georgia, 30308, United States
Houston Texas, 77030, United States
Cambridge , CB2 0, United Kingdom
London , WC1E , United Kingdom
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