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

Not yet recruiting

• Conditions: Immune Thrombotic Thrombocytopenia
• Interventions: Drug: Caplacizumab

• Registration Number: NCT06358703
• Lead Sponsor: US Thrombotic Microangiopathy Alliance

Brief 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

Detailed Description

Not available

Study Timeline

• Status Verified: 2024-04
• Study First Submitted: 2024-04-04
• Study First Submitted QC: 2024-04-04
• Study First Posted: 2024-04-10
• Last Update Submitted: 2024-11-10
• Last Update Posted: 2024-11-13
• Study Start: 2024-12
• Primary Completion: 2027-05
• Study Completion: 2028-05

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 116

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

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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Cases	Caplacizumab	-

Primary Outcome Measures

Name	Time	Method
Cognitive impairment	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
Silent cerebral infarction	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.

Secondary Outcome Measures

Name	Time	Method
Depression scores on BDI-II	12 months
SF-36 scores for quality of life	12 months
Patient reported cognitive performance	12 months	(PROMIS Cognitive Function - Short form 8a)

Trial Locations

Locations (1)

USTMA; 🇺🇸 Groveport, Ohio, United States