CASH (Cavernous Angiomas With Symptomatic Hemorrhage) Trial Readiness

Study Overview
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Brief Summary
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Brain Cavernous Angiomas with Symptomatic Hemorrhage (CASH) are rare, but they exact a heavy burden of neurologic disability from recurrent bleeding, for which there is no proven therapy. This trial readiness project aims to address current critical obstacles in identifying cases at multiple sites, characterizing their relevant features, and measuring their outcome. The timing cannot be more opportune, with therapeutic targets already identified, exceptional collaboration among researchers and with the patient community, and several drugs ready to benefit from a track to clinical testing in the next five years.

Detailed Description
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The Trial Readiness grant mechanism, funded by NINDS, proposes to address knowledge gaps and establish a research network as infrastructure for future research. This project includes an observational cohort study to assess (1) the feasibility of screening, enrollment rates, baseline disease categorization and follow-up of CASH using common data elements at multiple sites, (2) the reliability of imaging biomarkers including quantitative susceptibility mapping and permeability measures which have been shown to correlate with lesion activity, and (3) the rates of recurrent hemorrhage and change in functional status and biomarker measurements during prospective follow-up.

Official Title
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CASH (Cavernous Angiomas With Symptomatic Hemorrhage) Trial Readiness

Conditions
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CCM, Cavernoma, Cerebral Cavernous Malformation, Cerebral Cavernous Malformations 1, Cerebral Cavernous Malformations 2, Cerebral Cavernous Malformations 3, Cavernous Angioma

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria 18 years of age and older Diagnosed with a brain CA (single or multiple) Had a SH within the past year (with demonstrated new lesional bleeding or hemorrhagic growth on diagnostic studies AND attributable new symptoms) Subject is able to provide informed consent Exclusion Criteria Spinal CA as source of SH Prior brain irradiation Cases where verification of SH with clinical and imaging review cannot be accomplished Prior or planned treatment of the symptomatic lesion (after neurosurgical consultation) To be eligible for Aims 2 and 3, CASH cases enrolled in Aim 1 will be further excluded from follow-up and baseline validation (FUBV) for the following reasons: Contraindication for administration of contrast agent or otherwise unwilling or unable to undergo research MRI studies Pregnant or breastfeeding women Homeless or incarcerated persons, or other reason a subject will be unable/unlikely to return for follow-up visits

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| CASH (Cavernous Angiomas with Symptomatic Hemorrhage)<br>The adjudicated definition of CASH (Cavernous Angiomas with Symptomatic Hemorrhage) requires diagnostic evidence of new lesional bleeding or hemorrhagic growth, in association with directly attributable symptoms. | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Overall enrollment rate | Assessment of enrollment rates of CASH | Years 2-5 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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CCM (Cerebral Cavernous Malformations), Dynamic Contrast Enhanced Quantitative Perfusion (DCEQP), Quantitative Susceptibility Mapping (QSM), Cerebral Cavernous Malformation, Cavernoma, Cavernous Angioma