CASH (Cavernous Angiomas With Symptomatic Hemorrhage) Trial Readiness

The University of Chicago

Status: Completed

Conditions: Cerebral Cavernous Malformations 1; Cavernoma; Cerebral Cavernous Malformation; Cerebral Cavernous Malformations 2; Cavernous Angioma; CCM; Cerebral Cavernous Malformations 3

Study type: Observational

Funder types: Other; NIH

Identifiers

NCT03652181

U01NS104157 (U.S. NIH Grant/Contract)

Details and patient eligibility

About

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.

Full description

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 of 181 patients with an operational goal of demonstrating the feasibility of screening, enrollment rates, baseline disease categorization and follow-up of CASH using common data elements at multiple sites, and to assess the following endpoints for 123 participants enrolled at centers prespecified to do prospective follow-up: (1) the rates of recurrent hemorrhage; (2) the reliability of imaging biomarkers including quantitative susceptibility mapping and permeability measures which have been shown to correlate with lesion activity, and (3) change in functional status during prospective follow-up.

Enrollment: 123 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion Criteria

1. 18 years of age and older
2. Diagnosed with a brain CA (single or multiple)
3. Had a SH within the past year (with demonstrated new lesional bleeding or hemorrhagic growth on diagnostic studies AND attributable new symptoms)
4. Subject is able to provide informed consent

Exclusion Criteria

1. Spinal CA as source of SH
2. Prior brain irradiation
3. Cases where verification of SH with clinical and imaging review cannot be accomplished
4. 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:

1. Contraindication for administration of contrast agent or otherwise unwilling or unable to undergo research MRI studies
2. Pregnant or breastfeeding women
3. Homeless or incarcerated persons, or other reason a subject will be unable/unlikely to return for follow-up visits

Trial design

123 participants in 1 patient group

CASH (Cavernous Angiomas with Symptomatic Hemorrhage)

Description:

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.