Effect of Cannabinoids on Pain in Fabry Disease Patients; a Prospective, Randomized, Double-blind, Placebo-controlled, Crossover, Multicenter Study
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.
• • Age: 18-70 years
‣ Patients with genetically confirmed Fabry disease
⁃ On treatment with Enzyme Replacement Therapy (ERT)
⁃ Sufficient command of German language
⁃ Pain duration of more than 3 months
⁃ Stable analgesic regimen for at least 2 weeks preceding the study entry day
⁃ Baseline worst last week pain intensity ≥4 on numerical rating scale (NRS)
⁃ Signed and dated informed consent
⁃ ERT or chaperone therapy at a stable dose for at least 3 last months