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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT04981028
Other study ID # 5988
Secondary ID
Status Recruiting
Phase
First received
Last updated
Start date June 25, 2020
Est. completion date June 25, 2022

Study information

Verified date July 2021
Source Liverpool University Hospitals NHS Foundation Trust
Contact Tina Dutt
Phone 01512826724
Email tina.dutt@liverpoolft.nhs.uk
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial 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.


Recruitment information / eligibility

Status Recruiting
Enrollment 250
Est. completion date June 25, 2022
Est. primary completion date June 25, 2022
Accepts healthy volunteers Accepts Healthy Volunteers
Gender All
Age group 18 Years and older
Eligibility 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

Study Design


Related Conditions & MeSH terms


Intervention

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.

Locations

Country Name City State
United Kingdom Royal Liverpool University Hospital Liverpool

Sponsors (1)

Lead Sponsor Collaborator
Liverpool University Hospitals NHS Foundation Trust

Country where clinical trial is conducted

United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary The percentage of patients with TTP with neurological complications at acute presentation 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%. 1 week, 1 month, 3 months, 6 months, 12 months
Primary The percentage of patients with TTP in remission with long-term neurological complications 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%. 6 months, 12 months, 18 months, 2 years
Secondary The percentage of 'follow-up' patients with TTP with a depressive disorder, based on PHQ-9 scoring system, compared to the general UK population. 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. 6 month, 12 months, 18 months, 2 years
Secondary The percentage of 'follow-up' patients with TTP with neurocognitive deficit, based on TYM scoring system, compared to the general UK population. 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. 6 month, 12 months, 18 months, 2 years
Secondary The percentage of 'follow-up' patients with TTP with reduced quality of life, based on SF-36 score, compared to the general UK population. 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. 6 month, 12 months, 18 months, 2 years
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