Effect of Cannabinoids on Pain in Fabry Disease Patients
Status:
Not yet recruiting
Trial end date:
2022-07-01
Target enrollment:
Participant gender:
Summary
Fabry Disease (FD) is a rare lysosomal storage disorder due to the absence or deficiency of
hydrolase α-galactosidase A (α-Gal A) activity in lysosomes. This dysfunction results in
progressive accumulation of glycosphingolipids in a wide variety of cells, resulting in major
organ system damage.
Patients with Fabry disease can suffer from neuropathic pain, since lysosomal accumulation
affects small unmyelinated nerve fibers. Neuropathic pain is one of the prominent and
debilitating symptoms significantly interfering with life quality in FD patients. Current
treatment of Fabry patients with neuropathic pain is deficient, as they respond poorly to a
conventional pain therapy, often require a high-dose opioids treatment and presentation to
the Emergency Department.
Sativex® has been shown to be a successful treatment option in neuropathic pain of different
origin with minimal neuropsychological influence: in multiple sclerosis (MS),
chemotherapy-induced neuropathic pain and other. It contains Δ-9-Tetrahydrocannabinol (THC)
and Cannabidiol (CBD) and has recently been licensed in Switzerland for treatment of
neuropathic chronic pain in MS. Sativex® is an oral spray.