a Phase 1 clinical trials evaluate the safety of a treatment. Phase 2 evaluates both safety and efficacy of the treatment. Phase 3 is a larger trial needed to more firmly establish the effects observed in the Phase 2 trail.
b Oral capsules containing 285 mg of mixed tocotrienols and tocopherol, both compounds in the vitamin E family that are antioxidants.
c A natural history study is a prospective observational study that follows a group of individuals over time without experimental intervention. It collects health information necessary to understand how a medical condition develops and how it might be treated.
d Participants include 20 with CADASIL, 40 with non-CADASIL sporadic white matter disease, 20 with lobar cerebral microbleeds, and 20 age-matched healthy controls.
e Cilostazol is a platelet aggregation inhibitor approved to treat intermittent claudication, a condition causing pain in the legs during exertion due to inadequate blood supply.
f Adrenomedullin is a hormone found in all tissues and in blood. Its significance is unknown, but when administered it causes vasodilatation, or relaxation of arteries, and may improve blood flow and lower blood pressure.
g Cerebrolysin is a mixture of peptides (short chains of amino acids) believed to improve cognitive function in a number of conditions including vascular dementia. But evidence that benefits outweigh risks is weak (PMID: 31710397).
Abbreviations:
Cog – cognitive tests
NA – not available
Nat C & CV Cntr – National Cerebral and Cardiovascular Center
NCT – National Clinical Trial
NHLBI – National Heart Lung Blood Institute
NIH – National Institutes of Health
PRC – People’s Republic of China
RCT – randomized controlled trial
SVD – Small Vessel Disease, including but not exclusively CADASIL
UNSW – University of New South Wales, Australia