ONG TERM EFFECT AND TOLERANCE OF ULIPRISTAL ACETATE IN Charcot-Marie-TOOTH DISEASE TYPE 1A (UPACOMT)
- Conditions
- Charcot-Marie-Tooth disease type 1AMedDRA version: 18.0 Level: LLT Classification code 10008414 Term: Charcot-Marie-Tooth disease System Organ Class: 100000004850Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
- Registration Number
- EUCTR2015-001716-36-FR
- Lead Sponsor
- HÔPITAUX UNIVERSITAIRES DE STRASBOURG
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Not specified
- Target Recruitment
- 45
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
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);
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <br> 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.<br> ;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 ;<br> Secondary Objective: - To evaluate the clinical efficacy of ulipristal acetate<br> - To assess the biological effect of ulipristal acetate on serum markers of oxidative stress and in skin biopsies<br> - Evaluate the clinical and biological tolerance<br> - Assess the neurophysiological effects of ulipristal acetate<br> - To evaluate the pharmacokinetics of ulipristal acetate<br>
- Secondary Outcome Measures
Name Time Method <br> Secondary end point(s): 1. production of markers of oxidative stress in skin biopsy<br> 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).<br> 3. the biological effectiveness measured by serum samples in search of markers of oxidative stress.<br> 4. clinical and biological tolerance.<br> ;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.