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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]

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ClinicalTrials.gov Identifier: NCT03519672
Recruitment Status : Recruiting
First Posted : May 9, 2018
Last Update Posted : October 5, 2018
Sponsor:
Information provided by (Responsible Party):
Shire ( Baxalta now part of Shire )

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.

Condition or disease Intervention/treatment
Congenital Thrombotic Thrombocytopenic Purpura Other: No intervention

Study Type : Observational
Estimated Enrollment : 40 participants
Observational Model: Cohort
Time Perspective: Prospective
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]
Actual Study Start Date : July 12, 2018
Estimated Primary Completion Date : November 30, 2018
Estimated Study Completion Date : November 30, 2018


Group/Cohort Intervention/treatment
Participants with cTTP - adolescents
Adolescents aged 12 to 17 years
Other: No intervention
This is a non-interventional study.

Participants with cTTP - adults
Adults aged ≥18 years
Other: No intervention
This is a non-interventional study.




Primary Outcome Measures :
  1. Change from baseline of Quality of life (QoL) outcomes: cTTP-specific PRO instrument [ Time Frame: Day 1, and Day 14 ]
    The congenital thrombotic thrombocytopenic purpura (cTTP)-specific patient-reported outcomes (PRO) instrument consists of 26 questions designed to assess the patient's experience of fatigue, joint, muscle, abdominal and chest pain in the previous 24 hours, neurologic manifestations, bruising, feelings of depression and mood alterations, and activity limitation in the past 7 days, and patient's attitudes, experienced side effects, work/school absences and travel impact associated with treatment received for TTP during the previous 2 weeks.

  2. Change from baseline of Quality of life (QoL) outcomes: PROMIS®-29 [ Time Frame: Day 1, and Day 14 ]

    The Patient-Reported Outcomes Measurement Information System (PROMIS®)-29 Profile is a collection of 4-item short forms assessing anxiety, depression, fatigue, pain interference, physical function, sleep disturbance, and the ability to participate in social roles and activities, as well as a single pain intensity item.

    The individual scales for fatigue, pain interference, anxiety and depression, each being 5-point Likert scales, and the single pain intensity item, a 0 to 10 Numerical Rating Scale, will be used in this study. A higher score for all these scales indicate poorer HRQL (worse symptoms).


  3. Change from baseline of Quality of life (QoL) outcomes: Pediatric PROMIS® Subscales for adolescents [ Time Frame: Day 1, and Day 14 ]

    The Patient-Reported Outcomes Measurement Information System (PROMIS®)-29 Profile is a collection of 4-item short forms assessing anxiety, depression, fatigue, pain interference, physical function, sleep disturbance, and the ability to participate in social roles and activities, as well as a single pain intensity item. The Pediatric PROMIS® Subscales will be used for adolescents.

    The individual scales for fatigue, pain interference, anxiety and depression, each being 5-point Likert scales, and the single pain intensity item, a 0 to 10 Numerical Rating Scale, will be used in this study. A higher score for all these scales indicate poorer HRQL (worse symptoms).


  4. Change from baseline of Quality of life (QoL) outcomes: HIT-6 [ Time Frame: Day 1, and Day 14 ]
    The Headache Impact Test (HIT)-6 is a short-form scale, partially derived from the 54-item HIT, and used to measure the impact of headaches in the past 4 weeks on the ability to function at work, at school, at home and in social situations. Items are scored on a 5-point Likert scale ranging from 6=Never, 8=Rarely, 10=Sometimes, 11=Very often, 13=Always, with a global score ranging from 36 to 78, a higher score indicating a worse HRQL.

  5. Change from baseline of Quality of life (QoL) outcomes: Condensed MCMDM-1VWD Bleeding Questionnaire: Bruising Subscale [ Time Frame: Day 1, and Day 14 ]
    The Condensed MCMDM-1VWD Bleeding Questionnaire is a bleeding-specific questionnaire used to produce a bleeding score for von Willebrand disease, which was developed by eliminating all details not directly affecting the bleeding score from the validated and extensive MCMDM-1VWD questionnaire. This study will only use the 5 close-ended questions of the bruising scale. These 5 questions assess the presence of bruising (yes/no), location (exposed/unexposed sites) and average size (<1cm, 1 to 5cm and >5cm), presence of minimal or no trauma (yes/no), and whether medical attention was required (yes/no).

  6. Change from baseline of Quality of life (QoL) outcomes: PDQ-5 [ Time Frame: Day 1, and Day 14 ]
    The Perceived Deficits Questionnaire 5-Item (PDQ-5) is a subscale of the full-length Perceived Deficits Questionnaire (PDQ), developed specifically for participants with Multiple Sclerosis to assess perceived cognitive deficits from the subject's perspective. The PDQ has 4 5-item subscales: Attention/Concentration, Retrospective Memory, Prospective Memory, and Planning/Organization. The PDQ-5 consists of the 5 PDQ items correlating most strongly with the total PDQ score; items from all 4 subscales are represented.

  7. Change from baseline of Quality of life (QoL) outcomes: NEI-VFQ-25 [ Time Frame: Day 1, and Day 14 ]
    The National Eye Institute Visual Function Questionnaire (NEI-VFQ or VFQ)-25 is a valid and reliable 25-item version of the 51-item NEI-VFQ. The questionnaire measures the impact of visual impairment on Health Related Quality of Life (HRQL). The general vision (1 item), near vision (3 items), and distant vision activities (3 items) subscales will be used in this study. Each item is associated with a 6-point Likert scale ranging from 1 (no difficulty at all) to 6 (stopped doing this for other reason or not interested in doing this), and global scores range from 0 to 100, a higher score indicating better HRQL.

  8. Change from baseline of Quality of life (QoL) outcomes: WPAI-GH [ Time Frame: Day 1, and Day 14 ]
    The self-administered Work Productivity and Activity Impairment-General Health (WPAI-GH) is a 6-item scale measuring work (5 items) and daily activities (1 item). Scores express the impairment percentage so a lower score represents more productivity.

  9. Change from baseline of Quality of life (QoL) outcomes: WPAI-GH plus Classroom Impairment questions for adolescents [ Time Frame: Day 1, and Day 14 ]
    The Work Productivity and Activity Impairment (WPAI) plus Classroom Impairment Questionnaire will be used for adolescents. The WPAI plus Classroom Impairment Questionnaire measures school attendance; productivity levels at school; and ability to perform regular daily activities. The WPAI plus Classroom Impairment Questionnaire is a 9-item scale measuring school/work (8 items) and daily activities (1 item). Scores express the impairment percentage so a lower score represents more productivity. While the instructions from the instrument will not be changed, adolescent participants will be instructed to consider their school work and activities when completing the questionnaire.

  10. Change from baseline of Quality of life (QoL) outcomes: PGI-S [ Time Frame: Day 1, and Day 14 ]
    The Patient Global Impression of Severity (PGI-S) is a 1-item questionnaire designed to assess participant's impression of disease severity that was adapted to congenital thrombotic thrombocytopenic purpura (cTTP). The PGI-S item asks the respondent to best describe how their cTTP symptoms severity is now Scores are on a 4-point scale scored as: "normal" (1), "mild" (2), "moderate" (3), or "severe" (4).

  11. Change from baseline of Quality of life (QoL) outcomes: PGI-Change [ Time Frame: Day 1, and Day 14 ]
    The Patient Global Impression of Change (PGI-C) is a single 1 item questionnaire that compares the participant's current health state to when treatment was started. Response options range from 1 (Very Much Improved) through 4 (No Change) to 7 (Very Much Worse).



Information from the National Library of Medicine

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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03519672


Contacts
Contact: Abiola Oladapo, PhD +1 617 588 8254 Abiola.oladapo@shire.com
Contact: Shire Contact +1 866 842 5335 ClinicalTransparency@shire.com

Locations
United States, Georgia
AutoCruitment Recruiting
Atlanta, Georgia, United States, 30308
Sponsors and Collaborators
Baxalta now part of Shire
Investigators
Study Director: Study Director Shire

Responsible Party: Baxalta now part of Shire
ClinicalTrials.gov Identifier: NCT03519672     History of Changes
Other Study ID Numbers: 281701
First Posted: May 9, 2018    Key Record Dates
Last Update Posted: October 5, 2018
Last Verified: October 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Blood Coagulation Disorders
Hematologic Diseases
Hemorrhage
Blood Platelet Disorders
Immune System Diseases
Purpura
Purpura, Thrombocytopenic
Purpura, Thrombotic Thrombocytopenic
Pathologic Processes
Skin Manifestations
Signs and Symptoms
Thrombotic Microangiopathies
Thrombocytopenia
Thrombophilia