Clinical trial NCT03519672: 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]
Official title: 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 type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Congenital Thrombotic Thrombocytopenic Purpura
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2018-05-09
Enrollment: 41
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Participants will be identified for inclusion by the Investigator (physician) and, if interested to participate, they will be asked to provide informed consent (and assent for adolescents 12 to 17 years). The legally authorized representative (LAR) of participating cTTP adolescents (ages 12 to 17 years) will provide informed consent on the adolescent's behalf. A qualified vendor will also be used to identify potentially eligible participants through various networks.

Brief 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:

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.

Primary outcomes:
measure: Change from baseline of Quality of life (QoL) outcomes: cTTP-specific PRO instrument
timeFrame: Day 1, and Day 14
measure: Change from baseline of Quality of life (QoL) outcomes: PROMIS®-29
timeFrame: Day 1, and Day 14
measure: Change from baseline of Quality of life (QoL) outcomes: Pediatric PROMIS® Subscales for adolescents
timeFrame: Day 1, and Day 14
measure: Change from baseline of Quality of life (QoL) outcomes: HIT-6
timeFrame: Day 1, and Day 14
measure: Change from baseline of Quality of life (QoL) outcomes: Condensed MCMDM-1VWD Bleeding Questionnaire: Bruising Subscale
timeFrame: Day 1, and Day 14
measure: Change from baseline of Quality of life (QoL) outcomes: PDQ-5
timeFrame: Day 1, and Day 14
measure: Change from baseline of Quality of life (QoL) outcomes: NEI-VFQ-25
timeFrame: Day 1, and Day 14
measure: Change from baseline of Quality of life (QoL) outcomes: WPAI-GH
timeFrame: Day 1, and Day 14
measure: Change from baseline of Quality of life (QoL) outcomes: WPAI-GH plus Classroom Impairment questions for adolescents
timeFrame: Day 1, and Day 14
measure: Change from baseline of Quality of life (QoL) outcomes: PGI-S
timeFrame: Day 1, and Day 14
measure: Change from baseline of Quality of life (QoL) outcomes: PGI-Change
timeFrame: Day 1, and Day 14
Secondary outcomes:
Other outcomes:
Locations:
city: Atlanta
country: United States
facility: AutoCruitment
state: Georgia
city: Houston
country: United States
facility: Houston Methodist Research Institute
state: Texas
city: Cambridge
country: United Kingdom
facility: Cambridge University Hospital
city: London
country: United Kingdom
facility: University College London Hospital
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References: