View clinical trials related to Cerebral Cavernous Malformation.
Filter by:The goal is to characterize vascular reactivity and cardiac pulsatility of normal appearing white matter in patients with CCM during hypercarbia/hypocarbia using quantitative real-time high-speed multi-echo fMRI to develop prognostic biomarkers for CCM formation
Cerebral cavernous malformations (CCMs), one of the most common microvascular malformations in the capillary beds of the brain, are susceptible to hemorrhagic stroke. As an autosomal dominant disorder with incomplete penetrance, the majority of CCM gene mutation carriers are largely asymptomatic but when symptoms occur, the disease has typically reached the stage of focal hemorrhage with irreversible brain damage. Currently, the invasive neurosurgery removal of CCM lesions is the only treatment option, despite the recurrence of the symptoms after surgery. Therefore, there is a grave need for prognostic/monitoring biomarkers as risk predictors for stroke prevention. The objective of the proposal is to develop a set of blood prognostic/monitoring biomarkers as precise risk indicators for stroke prevention. In this project, the plan is to validate the novel serum biomarkers identified in Ccms animal models and human CCMs patients, and utilize these biomarkers with statistical algorithms for risk prediction of hemorrhagic CCMs. This proposal has been formulated based on recent findings of five serum etiological biomarkers associated with disruption of the Blood-Brain Barrier (BBB), the first step for hemorrhagic CCMs in Ccm mice models. This work will lay the groundwork for larger human trials for final validation and revolutionary potential clinical applications.
This is a two-part, multi-center, randomized, double-blind, placebo-controlled study to investigate the safety, efficacy and pharmacokinetics of REC-994 (200 mg and 400 mg) compared to placebo in subjects with symptomatic cerebral cavernous malformation (CCM).
The project aims to develop prognostic and diagnostic blood tests for symptomatic brain hemorrhage in patients diagnosed with cavernous angiomas, a critical clinical challenge in a disease affecting more than a million Americans. We further examine whether blood biomarkers can replace or enhance the accuracy of advanced imaging in association with lesional bleeding. The project tests a novel integrational approach of biomarker development in a mechanistically defined cerebrovascular disease, with a clinically relevant context of use.
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.
Cerebral Cavernous Malformation (CCM) is a cerebrovascular disease which can be either congenital in origin or sporadic and is characterized by the presence of isolated or multiple CCM lesions, causing recurrent headache, seizures, focal neurological deficits and hemorrhages. Inasmuch, to date, the only curative treatment available is limited to surgical lesion eradication or stereotactic radiosurgery. It is therefore necessary to find an effective medical treatment that may limit disease progression and decrease the burden of adverse clinical events. The non-selective betablocker propranolol has been found to be effective in the treatment of infantile cutaneous hemangioma, and anecdotal reports have been published on its efficacy in CCM. The safety profile of propranolol has been documented in millions of patients of all ages. The primary objective of this exploratory trial is to test whether a chronic treatment with propranolol will reduce the burden of cerebrovascular lesions, of clinical events and symptoms in patients with familial CCM.
The aim of this prospective study is to reveal the natural history of symptomatic hemorrhage in adult patients with cerebral cavernous malformation with the goal of informing the treatment plan.
This phase I/II randomized, placebo-controlled, double-blinded, single-site clinical trial is designed to investigate the effect of a prolonged course of atorvastatin versus placebo on CCM lesional iron deposition assessed by validated quantitative susceptibility mapping (QSM) MRI studies in patients who suffered a symptomatic bleed within the preceding one year.