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]



Status:Recruiting
Conditions:Hematology, Hematology
Therapuetic Areas:Hematology
Healthy:No
Age Range:12 - Any
Updated:1/31/2019
Start Date:July 12, 2018
End Date:April 28, 2019
Contact:Shire Contact
Email:ClinicalTransparency@shire.com
Phone:+1 866 842 5335

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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.


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.
We found this trial at
3
sites
Atlanta, Georgia 30308
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Atlanta, GA
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Cambridge,
Principal Investigator: Will Thomas
Phone: (+44) (0) 122 3217 717
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Cambridge,
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Houston, Texas 77030
Principal Investigator: Lawrence Rice
Phone: 713-441-5418
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Houston, TX
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