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

NCT06358703 | Not Yet Recruiting

• 1 other identifier: P400
• Study Type: observational
• Target: 116
• Locations: 1 country
• Sites: 1

Brief Summary

1. 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. 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. 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. 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

Conditions

Immune Thrombotic Thrombocytopenia

Keywords

TTP; iTTP; aTTP; Cognitive impairment

Outcome Measures

Primary Outcomes (2)

• 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.

  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

  12 months

Secondary Outcomes (3)

• Depression scores on BDI-II

  12 months

• SF-36 scores for quality of life

  12 months

• Patient reported cognitive performance

  12 months

Study Arms (2)

Cases

Drug: Caplacizumab

Controls

Interventions

Caplacizumab DRUG

Early Caplacizumab use

Cases

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Adults with iTTP

You may qualify if:

• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• US Thrombotic Microangiopathy Alliance: lead

Study Sites (1)

USTMA

Groveport, Ohio, 43125, United States

Location

Biospecimen

Retention: SAMPLES WITHOUT DNA

Plasma will be held in a biobank

MeSH Terms

Conditions

Cognitive Dysfunction

Interventions

caplacizumab

Condition Hierarchy (Ancestors)

Cognition Disorders; Neurocognitive Disorders; Mental Disorders

Study Design

• Study Type: observational
• Observational Model: CASE CONTROL
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 4, 2024
• First Posted: April 10, 2024
• Study Start: December 1, 2024
• Primary Completion (Estimated): May 1, 2027
• Study Completion (Estimated): May 1, 2028
• Last Updated: November 13, 2024

Record last verified: 2024-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)