The ConNeCT Study: Neurological Complications of TTP
1 other identifier
observational
250
1 country
1
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for all trials
Started Jun 2020
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
June 25, 2020
CompletedFirst Submitted
Initial submission to the registry
July 21, 2021
CompletedFirst Posted
Study publicly available on registry
July 28, 2021
CompletedPrimary Completion
Last participant's last visit for primary outcome
June 25, 2022
CompletedStudy Completion
Last participant's last visit for all outcomes
June 25, 2022
CompletedAugust 9, 2021
July 1, 2021
2 years
July 21, 2021
August 2, 2021
Conditions
Outcome Measures
Primary Outcomes (2)
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
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 Outcomes (3)
The percentage of 'follow-up' patients with TTP with a depressive disorder, based on PHQ-9 scoring system, compared to the general UK population.
6 month, 12 months, 18 months, 2 years
The percentage of 'follow-up' patients with TTP with neurocognitive deficit, based on TYM scoring system, compared to the general UK population.
6 month, 12 months, 18 months, 2 years
The percentage of 'follow-up' patients with TTP with reduced quality of life, based on SF-36 score, compared to the general UK population.
6 month, 12 months, 18 months, 2 years
Study Arms (3)
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%
Healthy volunteers
Non-blood relative / friend / carer
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
Interventions
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.
Eligibility Criteria
Adult patients diagnosed with thrombotic thrombocytopenic purpura in the United Kingdom
You may qualify if:
- 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.
You may not qualify if:
- 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
- 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
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
Royal Liverpool University Hospital
Liverpool, United Kingdom
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Central Study Contacts
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- OTHER GOV
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
July 21, 2021
First Posted
July 28, 2021
Study Start
June 25, 2020
Primary Completion
June 25, 2022
Study Completion
June 25, 2022
Last Updated
August 9, 2021
Record last verified: 2021-07