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The ConNeCT Study: Neurological Complications of TTP

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04981028
Recruitment Status : Unknown
Verified July 2021 by Liverpool University Hospitals NHS Foundation Trust.
Recruitment status was:  Recruiting
First Posted : July 28, 2021
Last Update Posted : August 9, 2021
Sponsor:
Information provided by (Responsible Party):
Liverpool University Hospitals NHS Foundation Trust

Study Description
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Brief Summary:
Thrombotic thrombocytopenic purpura (TTP) is a rare condition, which has a very high risk of death if not recognised and given immediate treatment. TTP is caused by a very low level of an enzyme in the body, called ADAMTS13. A lack of ADAMTS13 causes multiple small clots to form around the body which can disrupt the blood flow to important organs. Although survival has improved significantly, it is now being recognised that patients with TTP may suffer with longer term complications as a result of their condition; literature from the USA reports higher rates of major depression and also poor memory and reduced concentration in patients with TTP. The investigators aim to improve the understanding of the long-term complications and review, for the first time, forward-looking data at multiple time points in patients with TTP in the UK. Both patients with a new diagnosis and patients with a known diagnosis of TTP identified in NHS hospitals will be included, over a minimum duration of 2 years. This will be a questionnaire based study with both doctor led and participant led questionnaires at pre-determined points in time. By improving the understanding and comparing symptoms to that of the general population, the investigators hope to improve the support and tailor the treatments which can be offered to patients with TTP.

Condition or disease Intervention/treatment
Thrombotic Thrombocytopenic Purpura Other: Questionnaires (including PHQ-9, TYM and SF-36)

Study Design
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Study Type : Observational
Estimated Enrollment : 250 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: The ConNeCT Study: Neurological Complications of Thrombotic Thrombocytopenic Purpura
Actual Study Start Date : June 25, 2020
Estimated Primary Completion Date : June 25, 2022
Estimated Study Completion Date : June 25, 2022

Resource links provided by the National Library of Medicine


Groups and Cohorts
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Group/Cohort Intervention/treatment
Patients with acute episode of thrombotic thrombocytopenic purpura (TTP)
Any adult patients with a suspected diagnosis of TTP (defined by low platelets and anaemia with evidence of red cell breakdown) and confirmed by a low ADAMTS13 enzyme level <10%
 Other: Questionnaires (including PHQ-9, TYM and SF-36)
For the acute group, a questionnaire is completed on neurological symptoms at presentation plus specific questions to include brain imaging performed, treatments received and whether they survived the hospital admission (AQ1). There will be a healthcare practitioner led questionnaire (AQ2) and 3 patient led questionnaires (PHQ-9, TYM and SF-36) plus a supplementary questionnaire (AQ3) completed at 1 week, 1 month, 3 months, 6 months and 12 months following diagnosis. For the chronic TTP group, participants will have a questionnaire completed (CQ1) and 3 patient-led questionnaires (PHQ-9, TYM and SF-36) plus a supplementary questionnaire (CQ2). Where neurological symptoms are present, questionnaires will be completed 6 monthly; where there are no neurological symptoms questionnaires are completed every 12 months. The healthy volunteers will complete participant led questionnaires (namely PHQ-9, TYM, SF-36) plus a supplementary baseline questionnaire at two time points 12 months apart.
Healthy volunteers
Non-blood relative / friend / carer
 Other: Questionnaires (including PHQ-9, TYM and SF-36)
For the acute group, a questionnaire is completed on neurological symptoms at presentation plus specific questions to include brain imaging performed, treatments received and whether they survived the hospital admission (AQ1). There will be a healthcare practitioner led questionnaire (AQ2) and 3 patient led questionnaires (PHQ-9, TYM and SF-36) plus a supplementary questionnaire (AQ3) completed at 1 week, 1 month, 3 months, 6 months and 12 months following diagnosis. For the chronic TTP group, participants will have a questionnaire completed (CQ1) and 3 patient-led questionnaires (PHQ-9, TYM and SF-36) plus a supplementary questionnaire (CQ2). Where neurological symptoms are present, questionnaires will be completed 6 monthly; where there are no neurological symptoms questionnaires are completed every 12 months. The healthy volunteers will complete participant led questionnaires (namely PHQ-9, TYM, SF-36) plus a supplementary baseline questionnaire at two time points 12 months apart.
Patients with known diagnosis of TTP
Any adult patients with a previously confirmed diagnosis of TTP (more than 12 months ago) based on an ADAMTS13 enzyme level <10% at initial diagnosis
 Other: Questionnaires (including PHQ-9, TYM and SF-36)
For the acute group, a questionnaire is completed on neurological symptoms at presentation plus specific questions to include brain imaging performed, treatments received and whether they survived the hospital admission (AQ1). There will be a healthcare practitioner led questionnaire (AQ2) and 3 patient led questionnaires (PHQ-9, TYM and SF-36) plus a supplementary questionnaire (AQ3) completed at 1 week, 1 month, 3 months, 6 months and 12 months following diagnosis. For the chronic TTP group, participants will have a questionnaire completed (CQ1) and 3 patient-led questionnaires (PHQ-9, TYM and SF-36) plus a supplementary questionnaire (CQ2). Where neurological symptoms are present, questionnaires will be completed 6 monthly; where there are no neurological symptoms questionnaires are completed every 12 months. The healthy volunteers will complete participant led questionnaires (namely PHQ-9, TYM, SF-36) plus a supplementary baseline questionnaire at two time points 12 months apart.


Outcome Measures
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Primary Outcome Measures :
  1. The percentage of patients with TTP with neurological complications at acute presentation [ Time Frame: 1 week, 1 month, 3 months, 6 months, 12 months ]
    The primary outcome is to estimate the proportion of both new acute and remission TTP patients developing neurological conditions. These will be reported as counts and percentages with 95% confidence intervals. If we recruit 100 patients in both groups, acute and remission, then we can estimate prevalence rates of 10% with an accuracy of +/- 6%, a 20% prevalence with an accuracy of +/-8% and a 40% prevalence with an accuracy of +/-10%.

  2. The percentage of patients with TTP in remission with long-term neurological complications [ Time Frame: 6 months, 12 months, 18 months, 2 years ]
    The primary outcome is to estimate the proportion of both new acute and remission TTP patients developing neurological conditions. These will be reported as counts and percentages with 95% confidence intervals. If we recruit 100 patients in both groups, acute and remission, then we can estimate prevalence rates of 10% with an accuracy of +/- 6%, a 20% prevalence with an accuracy of +/-8% and a 40% prevalence with an accuracy of +/-10%.


Secondary Outcome Measures :
  1. The percentage of 'follow-up' patients with TTP with a depressive disorder, based on PHQ-9 scoring system, compared to the general UK population. [ Time Frame: 6 month, 12 months, 18 months, 2 years ]
    Secondary outcomes include proportion of TTP patients developing depressive or neurological symptoms or having reduced quality of life. These will be reported using counts and percentages, along with 95% confidence intervals. Also, these outcomes will be compared with the corresponding outcomes from the general population (control group). However, as the study is not powered to detect between group differences no formal hypothesis tests will be undertaken and the data will be presented using summary statistics, counts, percentages and 95% confidence intervals.

  2. The percentage of 'follow-up' patients with TTP with neurocognitive deficit, based on TYM scoring system, compared to the general UK population. [ Time Frame: 6 month, 12 months, 18 months, 2 years ]
    Secondary outcomes include proportion of TTP patients developing depressive or neurological symptoms or having reduced quality of life. These will be reported using counts and percentages, along with 95% confidence intervals. Also, these outcomes will be compared with the corresponding outcomes from the general population (control group). However, as the study is not powered to detect between group differences no formal hypothesis tests will be undertaken and the data will be presented using summary statistics, counts, percentages and 95% confidence intervals.

  3. The percentage of 'follow-up' patients with TTP with reduced quality of life, based on SF-36 score, compared to the general UK population. [ Time Frame: 6 month, 12 months, 18 months, 2 years ]
    Secondary outcomes include proportion of TTP patients developing depressive or neurological symptoms or having reduced quality of life. These will be reported using counts and percentages, along with 95% confidence intervals. Also, these outcomes will be compared with the corresponding outcomes from the general population (control group). However, as the study is not powered to detect between group differences no formal hypothesis tests will be undertaken and the data will be presented using summary statistics, counts, percentages and 95% confidence intervals.


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Sampling Method:   Non-Probability Sample
Study Population
Adult patients diagnosed with thrombotic thrombocytopenic purpura in the United Kingdom
Criteria

Inclusion Criteria:

1.Acute episode TTP:

  • Adult male or female patient ≥18 years of age at the time of signing the consent form, with a confirmed diagnosis of TTP (initial or relapse) based on ADAMTS13 <10% 2. Known diagnosis of TTP:
  • Adult male or female patient ≥ 18 years of age at time of signing the consent form, with a historical confirmed diagnosis of TTP (based on ADAMTS13 at initial presentation <10%) 3. Healthy control:
  • Non-blood relative / friend / carer of patients under the care of Haematology clinics at the Royal Liverpool University hospital or other participating centres.

Exclusion Criteria:

  1. Acute episode of TTP:

    • Participants less than 18 years old at the time of signing the consent form
    • Patient with ADAMTS13 greater than 10%
    • Patient with cancer or transplant associated MAHA will not be included
    • Patient (or NOK, where patient does not have capacity) not wishing to consent to trial

  2. Known diagnosis of TTP:

    • Participants less than 18 years old at the time of signing the consent form
    • Patient with ADAMTS13 greater than 10%
    • Patient with cancer or transplant associated MAHA will not be included
    • Patient (or NOK, where patient does not have capacity) not wishing to consent to trial

  3. For healthy control:

    • Participants less than 18 years old at the time of signing the consent form
    • Participant not wishing to consent to trial
    • Any personal or family history of thrombotic microangiopathy
Contacts and Locations
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Top of Page Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information

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): NCT04981028


Contacts
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Contact: Tina Dutt 01512826724 tina.dutt@liverpoolft.nhs.uk
Contact: Rebecca J Shaw R.J.Shaw@liverpool.ac.uk

Locations
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United Kingdom
Royal Liverpool University Hospital Recruiting
Liverpool, United Kingdom
Contact: Rebecca J Shaw         
Sponsors and Collaborators
Liverpool University Hospitals NHS Foundation Trust
More Information
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Responsible Party: Liverpool University Hospitals NHS Foundation Trust
ClinicalTrials.gov Identifier: NCT04981028    
Other Study ID Numbers: 5988
First Posted: July 28, 2021    Key Record Dates
Last Update Posted: August 9, 2021
Last Verified: July 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Purpura
Purpura, Thrombocytopenic
Purpura, Thrombotic Thrombocytopenic
Blood Coagulation Disorders
Hematologic Diseases
Hemorrhage
Pathologic Processes
 Skin Manifestations
Thrombotic Microangiopathies
Thrombocytopenia
Blood Platelet Disorders
Cytopenia
Immune System Diseases
Thrombophilia