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Not yet recruitingNCT06358703NeSTUpdated Nov 13, 2024

Impact of Acute ITTP Therapies on Long Term Neurologic and Cognitive Outcomes in ITTP Survivors

An observational study in Immune Thrombotic Thrombocytopenia, sponsored by US Thrombotic Microangiopathy Alliance. Not yet recruiting at 1 site in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2024-11-13.

Sponsored by US Thrombotic Microangiopathy Alliance · Observational

Study type
Observational
Model
Case-control
Time perspective
Prospective
Enrollment
116
Ages
18 Years and older
Sex
All
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Study 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 without caplacizumab.
  2. 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.
  3. We expect to find that SCI and cognitive impairment is associated with worse scores on the health related quality of life instrument (SF-36)
  4. 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
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Conditions studied

  • Immune Thrombotic Thrombocytopenia

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Keywords

  • TTP
  • iTTP
  • aTTP
  • Cognitive impairment
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In context

Thrombocytopenia

697 studies on the registry are indexed under Thrombocytopenia; 153 are open to participants now.

This study's planned enrollment of 116 is close to the median of 120 across 183 observational studies indexed under Thrombocytopenia.

Browse Thrombocytopenia studies →

Lead sponsor

This is the only study on the registry with US Thrombotic Microangiopathy Alliance as lead sponsor.

Counted across the registry records on this site, refreshed daily.

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Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No
Sampling method
Non-probability sample

Study population

Adults with iTTP

Inclusion criteria

  1. Age >= 18 years
  2. Confirmed iTTP based on ADAMS13 activity \< 10 % (or 10-20% with positive inhibitor or antibody) during an acute iTTP episode
  3. 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)

Exclusion criteria

Exclusion Criteria:

  1. Any contraindication for MRI (metallic implants, shrapnel, MRI incompatible stents, etc)
  2. Unable to speak, read or understand instructions in English (for NIH ToolBox)
  3. Combination of iTTP episodes treated without caplacizumab or with caplacizumab.
  4. Caplacizumab started at >= 4 days from diagnosis or for refractory iTTP
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Study design

Observational model
Case-control
Time perspective
Prospective
Enrollment
116 participants (estimated)
Patient registry
No
Biospecimen retention
Samples without dna

Groups and cohorts

  • Cases

    Drug: Caplacizumab

  • Controls

Interventions

  • DrugCaplacizumab

    Early Caplacizumab use

06

What researchers measure

Primary outcomes

  1. 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.

    Time frame: 12 months

  2. 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

    Time frame: 12 months

Secondary outcomes

  1. Depression scores on BDI-II

    Time frame: 12 months

  2. SF-36 scores for quality of life

    Time frame: 12 months

  3. Patient reported cognitive performance

    (PROMIS Cognitive Function - Short form 8a)

    Time frame: 12 months

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Study locations

1 site
  • USTMA
    Groveport, Ohio 43125, United States
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References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

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Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Nov 13, 2024, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
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Registry details

Key details

Study ID
NCT06358703
Lead sponsor
US Thrombotic Microangiopathy Alliance
Responsible party
Sponsor
First posted
Apr 10, 2024
Start date
Dec 2024 (estimated)
Primary completion
May 2027 (estimated)
Completion
May 2028 (estimated)
Last update
Nov 13, 2024

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is not yet recruiting, as verified in Apr 2024. You cannot join it, but the record below documents what was studied.

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Discussion

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