Congenital Thrombotic Thrombocytopenic PurpuraNCT03519672
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]
An observational study of Congenital Thrombotic Thrombocytopenic Purpura, run by Baxalta now part of Shire at 4 sites in 2 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Congenital Thrombotic Thrombocytopenic Purpura
- How many people
- 41 participants
- Where
- 4 sitesin 2 countries
- Study dates
- 12 Jul 2018to 8 Apr 2019
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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.
Who may be able to join
- 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.
Who cannot join
- The participant cannot read nor write;
- The participant is non-English speaking;
- The participant is currently participating in a clinical trial.
Trial locations and contacts
4 sites in 2 countries.
4 sites in 2 countries
United States2 sites
AutoCruitment
Atlanta, Georgia, 30308
Houston Methodist Research Institute
Houston, Texas, 77030
United Kingdom2 sites
Cambridge University Hospital
Cambridge, CB2 0QQ
University College London Hospital
London, WC1E 6HX
About this study
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.
- Being tested
- No intervention
- Medical subject headings
- Purpura, Thrombotic Thrombocytopenic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
No interventionOther
This is a non-interventional study.
| Study group | Role | What they receive |
|---|---|---|
| Participants with cTTP - adolescents | Adolescents aged 12 to 17 years Receives: Other: No intervention | |
| Participants with cTTP - adults | Adults aged ≥18 years Receives: Other: No intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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).
Measured: 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).
Measured: 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.
Measured: 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).
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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).
Measured: 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).
Measured: Day 1, and Day 14
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 May 2018
- Study start12 Jul 2018
- Primary completion8 Apr 2019
- Study completion8 Apr 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Congenital Thrombotic Thrombocytopenic PurpuraStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 41 participants (actual)
Sponsor and investigators
- Lead sponsor
- Baxalta now part of Shire
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03519672
- Sponsor's protocol ID
- 281701
- Status last verified
- Mar 2021
- First submitted
- 23 Apr 2018
- Last updated
- 17 Mar 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
Study at a glance
From the registry record, last updated by the sponsor on 17 Mar 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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]
- ClinicalTrials.gov ID
- NCT03519672
- Acronym / sponsor ID
- 281701
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 41 participants (actual)
- Lead sponsor
- Baxalta now part of ShireIndustry (pharma, biotech, devices)
- Study start
- 12 Jul 2018 (actual)
- Primary completion
- 8 Apr 2019 (actual)
- Study completion
- 8 Apr 2019 (actual)
- First posted
- 9 May 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Participants with cTTP - adolescents | – | Other: No interventionAdolescents aged 12 to 17 years |
| Participants with cTTP - adults | – | Other: No interventionAdults aged ≥18 years |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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.
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (3 listed)
- The participant cannot read nor write;
- The participant is non-English speaking;
- The participant is currently participating in a clinical trial.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| No interventionThis is a non-interventional study. | Other | Participants with cTTP - adolescents, Participants with cTTP - adults |
Primary outcome
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.
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).
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).
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.
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).
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.
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.
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.
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.
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).
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).
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Mar 2021.
- To obtain more information on the study, click here/on this link.Link provided by the sponsor.
- Individual participant dataSharing planned: Yes. Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Study Director study director
Takeda
4 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Congenital Thrombotic Thrombocytopenic Purpura, run by Baxalta now part of Shire at 4 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 17 Mar 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from baseline of Quality of life (QoL) outcomes: cTTP-specific PRO instrument (time frame: Day 1, and Day 14). The study lists 11 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Mar 2021.