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]

NCT03519672 | Completed

• 1 other identifier: 281701
• Study Type: observational
• Target: 41
• Locations: 2 countries
• Sites: 4

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.

Conditions

Congenital Thrombotic Thrombocytopenic Purpura

Outcome Measures

Primary Outcomes (11)

• Change from baseline of Quality of life (QoL) outcomes: cTTP-specific PRO instrument

  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.

  Day 1, and Day 14

• Change from baseline of Quality of life (QoL) outcomes: PROMIS®-29

  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).

  Day 1, and Day 14

• Change from baseline of Quality of life (QoL) outcomes: Pediatric PROMIS® Subscales for adolescents

  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).

  Day 1, and Day 14

• Change from baseline of Quality of life (QoL) outcomes: HIT-6

  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.

  Day 1, and Day 14

• Change from baseline of Quality of life (QoL) outcomes: Condensed MCMDM-1VWD Bleeding Questionnaire: Bruising Subscale

  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).

  Day 1, and Day 14

• Change from baseline of Quality of life (QoL) outcomes: PDQ-5

  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.

  Day 1, and Day 14

• Change from baseline of Quality of life (QoL) outcomes: NEI-VFQ-25

  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.

  Day 1, and Day 14

• Change from baseline of Quality of life (QoL) outcomes: WPAI-GH

  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.

  Day 1, and Day 14

• Change from baseline of Quality of life (QoL) outcomes: WPAI-GH plus Classroom Impairment questions for adolescents

  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.

  Day 1, and Day 14

• Change from baseline of Quality of life (QoL) outcomes: PGI-S

  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).

  Day 1, and Day 14

• Change from baseline of Quality of life (QoL) outcomes: PGI-Change

  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).

  Day 1, and Day 14

Study Arms (2)

Participants with cTTP - adolescents

Adolescents aged 12 to 17 years

Other: No intervention

Participants with cTTP - adults

Adults aged ≥18 years

Other: No intervention

Interventions

No intervention OTHER

This is a non-interventional study.

Participants with cTTP - adolescents; Participants with cTTP - adults

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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.

You may not qualify if:

• The participant cannot read nor write;
• The participant is non-English speaking;
• The participant is currently participating in a clinical trial.

Sponsors & Collaborators

• Baxalta now part of Shire: lead

Study Sites (4)

AutoCruitment

Atlanta, Georgia, 30308, United States

Location

Houston Methodist Research Institute

Houston, Texas, 77030, United States

Location

Cambridge University Hospital

Cambridge, CB2 0QQ, United Kingdom

Location

University College London Hospital

London, WC1E 6HX, United Kingdom

Location

Related Links

• To obtain more information on the study, click here/on this link.

MeSH Terms

Conditions

Purpura, Thrombotic Thrombocytopenic

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Thrombophilia; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 23, 2018
• First Posted: May 9, 2018
• Study Start: July 12, 2018
• Primary Completion: April 8, 2019
• Study Completion: April 8, 2019
• Last Updated: March 17, 2021

Record last verified: 2021-03

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.

Locations

GB (2); US (2)