Charcot-Marie-Tooth disease type 1A MedDRA version: 18.0 Level: LLT Classification code 10008414 Term: Charcot-Marie-Tooth disease System Organ Class: 100000004850
Conditions
Interventions
Trade Name: elleOne
Product Name: ULIPRISTAL ACETATE
Pharmaceutical Form: Tablet
Pharmaceutical form of the placebo: Tablet
Route of adm
Sponsors
HÔPITAUX UNIVERSITAIRES DE STRASBOURG
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: Male from 18 to 70 years Signed informed consent CMT1A proven genetically (17p11.2 duplication) and symptomatic Non-severe axonal damage Belong to a social security scheme subject Having been informed about the results of the medical examination prior enrollment Subject contacted with a valid phone number agree to use with a double partner effective method of contraception at least one barrier for the duration of the study and during the 2 weeks following the end of treatment Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 45 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Major protected by law, under guardianship • Hypersensitivity to the active substance / excipient • Another cause of neuropathy: Chronic alcohol intoxication, chemotherapy, diabetes, kidney failure, monoclonal gammopathy, cryoglobulins, B12 deficiency, hepatitis B / C, HIV, Lyme or poliomyelitis • Hepatic and renal impairment • Lapp lactase deficiency, malabsoprtion syndrome glucose / galactose • Decision-going drug / plant interacting with CYP3A4 and / or inhibitor of proton pump • History of hypersensitivity to any product or device that can be used before, during and after the biopsy; • In the biopsy site: surgery, skin disease or local infection • Immunosuppression innate or acquired • Malfunction of the innate or acquired coagulation • a recognized addiction of alcoholism or drug addiction; • known healing disorders: keloids or delay healing. • biological control of active viral disease such as positive serology for HIV, hepatitis B or C ... (withdrawals made prior to inclusion);
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the biological effect of ulipristal acetate, a selective modulator of the progesterone receptor in CMT1A in humans, on the mRNA expression of the PMP22 in a skin biopsy. ;Primary end point(s): measure in skin biopsy the variation of mRNA expression of PMP22 after 1 year of treatment with ulipristal acetate.;Timepoint(s) of evaluation of this end point: one year after randomization ; Secondary Objective: - To evaluate the clinical efficacy of ulipristal acetate - To assess the biological effect of ulipristal acetate on serum markers of oxidative stress and in skin biopsies - Evaluate the clinical and biological tolerance - Assess the neurophysiological effects of ulipristal acetate - To evaluate the pharmacokinetics of ulipristal acetate | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. production of markers of oxidative stress in skin biopsy 2. clinical efficacy as measured by the QMT (Quantified Muscular Testing), the CMTNS2 (Charcot-Marie-Tooth Neuropathy Score Version 2), the T10MW (Ten-meter timed walking), the 9NHPT (Nine-hole peg test) the ONLS (Overall Neuropathy Limitations Scales), EVA (analogue rating Scale) pain / fatigue and quality of life (SF36). 3. the biological effectiveness measured by serum samples in search of markers of oxidative stress. 4. clinical and biological tolerance. ;Timepoint(s) of evaluation of this end point: Clinical evaluations will be conducted during the screening visit, randomization and repeated at 1, 3, 6, 9, 12 and 13 months after randomization. | — |
Countries
France
Contacts
Public ContactDRCI
Hôpitaux universitaires de Strasbourg
Outcome results
None listed