Effect of Cannabinoids on Pain in Fabry Disease Patients; a Prospective, Randomized, Double-blind, Placebo-controlled, Crossover, Multicenter Study

Cannabinoids in Fabry pain

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.

2022-07-07

2023-12-29

2023-12-29

Recruiting

22

Inclusion Criteria: * • 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 Exclusion Criteria: * • Known hypersensitivity or allergy to cannabinoids. * Women who are pregnant or breast feeding; intention to become pregnant during the course of the study, lack of safe contraception, defined as: female subjects of childbearing potential, not using and not willing to continue using a medically reliable method of contraception for the entire study duration, such as oral, injectable, or implantable contraceptives, or intrauterine contraceptive devices, or who are not willing or able to use any other second (additional) considered sufficiently reliable by the investigator in individual cases. Female subjects who are surgically sterilized/hysterectomized or post-menopausal for longer than 2 years are not considered as being of child bearing potential. * Dementia * Other pain not of neuropathic origin thought by the investigator to be of nature or severity to interfere with the patient's assessment of neuropathic pain due to Fabry disease. * Patients with known or suspected non-compliance, drug or alcohol abuse, including Marijuana cigarettes. * Patients with known schizophrenia, other psychotic disorders, personality disorders or another severe psychiatric disorder or positive family history with these disorders, except depression. * Patients with another clinically significant disease (e.g. renal failure, hepatic dysfunction, severe cardiovascular or convulsive diseases). * Participation in another study with investigational drugs within the 30 days preceding and during the present study. * Previous enrolment into the current study * Enrolment of the investigator, his/her family members, employees and other dependent persons.

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2023-05-12