Immune Thrombotic ThrombocytopeniaNeSTNCT06358703
Impact of Acute ITTP Therapies on Long Term Neurologic and Cognitive Outcomes in ITTP Survivors
An observational study of Immune Thrombotic Thrombocytopenia, run by US Thrombotic Microangiopathy Alliance at 1 site in United States.
Trial summary
1. We expect to find that the silent cerebral infarct (SCI) rate is two fold higher in patients treated without caplacizumab. We also expect to find that the rate of mild and major cognitive impairment in patients treated with caplacizumab within 3 days of starting plasma exchange will be lower than patients treated… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Immune Thrombotic Thrombocytopenia
- What is being tested?
- Caplacizumab
- How many people
- 116 participants
- Where
- 1 siteUnited States
- Study dates
- Dec 2024to May 2028
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Adults with iTTP
Who may be able to join
- Age >= 18 years
- Confirmed iTTP based on ADAMS13 activity < 10 % (or 10-20% with positive inhibitor or antibody) during an acute iTTP episode
- Only 1 episode of iTTP that was treated with plasma exchange and caplacizumab started within 3 days of diagnosis (or if more than 1 episode, then all episodes treated with plasma exchange and caplacizumab started within 3 days of diagnosis)
Who cannot join
- Any contraindication for MRI (metallic implants, shrapnel, MRI incompatible stents, etc)
- Unable to speak, read or understand instructions in English (for NIH ToolBox)
- Combination of iTTP episodes treated without caplacizumab or with caplacizumab.
- Caplacizumab started at >= 4 days from diagnosis or for refractory iTTP
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site
USTMA
Groveport, Ohio, 43125
Clare Martin, 6149994900, Clare@ustma.org
About this study
- We expect to find that the silent cerebral infarct (SCI) rate is two fold higher in patients treated without caplacizumab. We also expect to find that the rate of mild and major cognitive impairment in patients treated with caplacizumab within 3 days of starting plasma exchange will be lower than patients treated without caplacizumab.
- We expect that the differences in cognitive impairment in cases (caplacizumab) versus controls (no caplacizumab) will persist on serial evaluation 1 year later. We also expect that there will be differences in these groups even after adjusting for time since episode and severity of presentation.
- We expect to find that SCI and cognitive impairment is associated with worse scores on the health related quality of life instrument (SF-36)
- Based on studies in non-TTP populations, we expect to find that the rate of incident stroke over the period of follow up is at least 2 fold higher in patients that have SCI compared with patients who do not have SCI
- Conditions
- Immune Thrombotic Thrombocytopenia
- Being tested
- Caplacizumab
- Keywords
- TTP, iTTP, aTTP, Cognitive impairment
- Medical subject headings
- Cognitive Dysfunction
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CaplacizumabDrug
Early Caplacizumab use
| Study group | Role | What they receive |
|---|---|---|
| Cases | Not described Receives: Drug: Caplacizumab | |
| Controls | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
Silent cerebral infarction
Silent cerebral infarction (primary endpoint) - SCI is derived from quantitation (number and total volume) of ischemic
(infarct-like) lesions shown as foci of T2 and FLAIR hyperintensity. SCI is diagnosed if the participant has an infarct like
lesion, at least 3 mm, with normal neurologic examination or an abnormality on examination that is not explained by the
location of the brain lesion. We have assembled a panel of 3 neuroradiologists headed by Dr. Doris Lin. Each MRI will be
read by 2 radiologists. A third reviewer will serve as a tie breaker in case of disagreement.
Measured: 12 months
Cognitive impairment
Cognitive impairment (co-primary endpoint) - measured using NIH ToolBox Cognition Battery. Mild and major cognitive impairment are
defined as T scores that are 1-2 SD below mean and > 2SD below mean for any domain, respectively
Measured: 12 months
Secondary outcomes (3)
Depression scores on BDI-II
Measured: 12 months
SF-36 scores for quality of life
Measured: 12 months
Patient reported cognitive performance
(PROMIS Cognitive Function - Short form 8a)
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Apr 2024
- Study startDec 2024 (estimated)
- Primary completionMay 2027 (estimated)
- Study completionMay 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Immune Thrombotic ThrombocytopeniaStudies 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
- Case-control
- Time perspective
- Prospective
- Enrolment
- 116 participants (estimated)
Sponsor and investigators
- Lead sponsor
- US Thrombotic Microangiopathy Alliance
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06358703
- Sponsor's protocol ID
- P400
- Acronym
- NeST
- Status last verified
- Apr 2024
- First submitted
- 4 Apr 2024
- Last updated
- 13 Nov 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Impact of Acute ITTP Therapies on Long Term Neurologic and Cognitive Outcomes in ITTP Survivors
- ClinicalTrials.gov ID
- NCT06358703
- Acronym / sponsor ID
- NeST / P400
- Conditions
- Immune Thrombotic Thrombocytopenia
- Keywords
- TTP, iTTP, aTTP, Cognitive impairment
- Study type
- Observational
- Recruitment status
- Not yet recruiting
- Enrolment
- 116 participants (estimated)
- Lead sponsor
- US Thrombotic Microangiopathy AllianceUniversity, hospital or other
- Study start
- Dec 2024 (estimated)
- Primary completion
- May 2027 (estimated)
- Study completion
- May 2028 (estimated)
- First posted
- 10 Apr 2024 (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.
- We expect to find that the silent cerebral infarct (SCI) rate is two fold higher in patients treated without caplacizumab. We also expect to find that the rate of mild and major cognitive impairment in patients treated with caplacizumab within 3 days of starting plasma exchange will be lower than patients treated without caplacizumab.
- We expect that the differences in cognitive impairment in cases (caplacizumab) versus controls (no caplacizumab) will persist on serial evaluation 1 year later. We also expect that there will be differences in these groups even after adjusting for time since episode and severity of presentation.
- We expect to find that SCI and cognitive impairment is associated with worse scores on the health related quality of life instrument (SF-36)
- Based on studies in non-TTP populations, we expect to find that the rate of incident stroke over the period of follow up is at least 2 fold higher in patients that have SCI compared with patients who do not have SCI
Study design
- Observational model
- Case-control
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cases | – | Drug: Caplacizumab |
| Controls | – | – |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Adults with iTTP
Key inclusion criteria (3 listed)
- Age >= 18 years
- Confirmed iTTP based on ADAMS13 activity < 10 % (or 10-20% with positive inhibitor or antibody) during an acute iTTP episode
- Only 1 episode of iTTP that was treated with plasma exchange and caplacizumab started within 3 days of diagnosis (or if more than 1 episode, then all episodes treated with plasma exchange and caplacizumab started within 3 days of diagnosis)
Key exclusion criteria (4 listed)
- Any contraindication for MRI (metallic implants, shrapnel, MRI incompatible stents, etc)
- Unable to speak, read or understand instructions in English (for NIH ToolBox)
- Combination of iTTP episodes treated without caplacizumab or with caplacizumab.
- Caplacizumab started at >= 4 days from diagnosis or for refractory iTTP
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 |
|---|---|---|
| CaplacizumabEarly Caplacizumab use | Drug | Cases |
Primary outcome
Silent cerebral infarction
Time frame: 12 months
Silent cerebral infarction (primary endpoint) - SCI is derived from quantitation (number and total volume) of ischemic (infarct-like) lesions shown as foci of T2 and FLAIR hyperintensity. SCI is diagnosed if the participant has an infarct like lesion, at least 3 mm, with normal neurologic examination or an abnormality on examination that is not explained by the location of the brain lesion. We have assembled a panel of 3 neuroradiologists headed by Dr. Doris Lin. Each MRI will be read by 2 radiologists. A third reviewer will serve as a tie breaker in case of disagreement.
Cognitive impairment
Time frame: 12 months
Cognitive impairment (co-primary endpoint) - measured using NIH ToolBox Cognition Battery. Mild and major cognitive impairment are defined as T scores that are 1-2 SD below mean and > 2SD below mean for any domain, respectively
3 secondary outcomes
Depression scores on BDI-II
Time frame: 12 months
SF-36 scores for quality of life
Time frame: 12 months
Patient reported cognitive performance
Time frame: 12 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Nov 2024.
- Individual participant dataSharing planned: No
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Immune Thrombotic Thrombocytopenia, run by US Thrombotic Microangiopathy Alliance at 1 site in United States.
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 3 inclusion and 4 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: opening soon, as of the sponsor's last update on 13 Nov 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Silent cerebral infarction (time frame: 12 months). The study lists 2 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 13 Nov 2024.