NCT03652181

CASH (Cavernous Angiomas With Symptomatic Hemorrhage) Trial Readiness

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 123 (actual)
• Sponsor: University of Chicago · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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

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.

Conditions

• CCM
• Cavernoma
• Cerebral Cavernous Malformation
• Cerebral Cavernous Malformations 1
• Cerebral Cavernous Malformations 2
• Cerebral Cavernous Malformations 3
• Cavernous Angioma

Timeline

• Start date: 2018-08-20
• Primary completion: 2022-11-03
• Completion: 2022-11-03
• First posted: 2018-08-29
• Last updated: 2024-06-27
• Results posted: 2024-06-27

Locations

1 site across 1 country: United States