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NCT Number: NCT06358703

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

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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Key information

Conditions

Immune Thrombotic Thrombocytopenia Cognition Disorders Cognitive Dysfunction Mental Disorders Neurocognitive Disorders

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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)

Exclusion criteria

  • 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

Treatment and study plan

Caplacizumab

Drug

Early Caplacizumab use

Primary outcomes

  1. 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.

  2. 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

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

    Time frame: 12 months

    (PROMIS Cognitive Function - Short form 8a)

Sponsors and collaborators

Lead sponsor

US Thrombotic Microangiopathy Alliance

Other

Registry information

Acronym: NeST

Important dates

Study start
2024
Primary completion
2027
Study completion
2028
First posted
Apr 10, 2024
Registry last updated
Nov 13, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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