Ulipristal Acetate In Disease Charcot-Marie-Tooth Type of 1A (UPACOMT)
CMT1A
About this trial
This is an interventional treatment trial for CMT1A focused on measuring Physical medicine and rehabilitation, Neurology
Eligibility Criteria
Inclusion Criteria:
- Male 18-70 years
- CMT1A proven genetically (17p11.2 duplication)
- symptomatic CMT1A (MRC score <5 in at least one muscle group)
- Non severe axonal impairment (amplitude of the motor evoked potential on the median nerve and / or ulnar than 50% of normal)
- Subject contacted with a valid phone number
- Subject affiliated to a social security scheme
- Subject has been informed of the results of the medical examination prior
Exclusion Criteria:
- Another cause of neuropathy: Chronic alcohol intoxication, chemotherapy, diabetes, kidney failure, monoclonal gammopathy, cryoglobulin, B12 deficiency, hepatitis B / C, HIV, Lyme or poliomyelitis
- Liver failure
- Lapp lactase deficiency, malabsoprtion syndrome glucose / galactose
- Support long-term drug interacting with the CYP3A4
- Patients with indication against xylocaine adrenaline
- In the biopsy site: surgery, skin disease or local infection
- Immunosuppression innate or acquired
- Hypersensitivity to the active substance / excipient
- uncontrolled severe asthma
- Treatment with vitamin C or vitamin B3 in the four weeks preceding randomization
- Orthopaedic surgery of the lower limbs in the 6 months prior to randomization or planned
- Against indication xylocaine adrenaline
- Malfunction of the innate or acquired coagulation
Sites / Locations
- Service d'Explorations et pathologies neuro- musculaires
- Département de Neurologie
- Département de Neurologie
- Unité de pathologie neuro-musculaire
- Département de Neurologie Centre de Référence des Maladies Neuromusculaires Grand Est (CERNEST) Hôpital de Hautepierre
Arms of the Study
Arm 1
Arm 2
Arm 3
Experimental
Experimental
Experimental
1
2
3
Arm (1) will be randomised to receive either : 5 mg/per os of EllaOne every day through 12 months. 10 mg/per os of EllaOne every day through 12 months. EllaOne placebo/per os every day through 12 months.
Arm (2) will be randomised to receive either : 5 mg/per os of EllaOne every day through 12 months. 10 mg/per os of EllaOne every day through 12 months. EllaOne placebo/per os every day through 12 months.
Arm (3) will be randomised to receive either : 5 mg/per os of EllaOne every day through 12 months. 10 mg/per os of EllaOne every day through 12 months. EllaOne placebo/per os every day through 12 months.