LONG TERM EFFECT AND TOLERANCE OF ULIPRISTAL ACETATE IN Charcot-Marie-TOOTH DISEASE TYPE 1A (UPACOMT)

• Register: EUCTR
• Last refreshed on: 10 December 2019
• Main ID: EUCTR2015-001716-36-FR
• Date of registration: 25/06/2015
• Prospective Registration: Yes
• Primary sponsor: HÔPITAUX UNIVERSITAIRES DE STRASBOURG
• Public title: LONG TERM EFFECT AND TOLERANCE OF ULIPRISTAL ACETATE IN Charcot-Marie-TOOTH DISEASE TYPE 1A (UPACOMT)
• Scientific title: LONG TERM EFFECT AND TOLERANCE OF ULIPRISTAL ACETATE IN Charcot-Marie-TOOTH DISEASE TYPE 1A (UPACOMT) - UPACOMT
• Date of first enrolment: 03/08/2015
• Target sample size: 45
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2015-001716-36
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes; Randomised: yes; Open: no; Single blind: no; Double blind: yes; Parallel group: yes; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: yes; Other: no; Number of treatment arms in the trial: 3
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

France

Contacts

• Name: DRCI
• Affiliation: Hôpitaux universitaires de Strasbourg

• Name: DRCI
• Affiliation: Hôpitaux universitaires de Strasbourg

Key inclusion & exclusion 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:
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);


Age minimum:
Age maximum:
Gender:
Female: no
Male: yes

Health Condition(s) or Problem(s) studied

Charcot-Marie-Tooth disease type 1A
MedDRA version: 18.0 Level: LLT Classification code 10008414 Term: Charcot-Marie-Tooth disease System Organ Class: 100000004850

Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Intervention(s)

Trade Name: elleOne
Product Name: ULIPRISTAL ACETATE
Pharmaceutical Form: Tablet
Pharmaceutical form of the placebo: Tablet
Route of administration of the placebo: Oral use

Primary Outcome(s)

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

Timepoint(s) of evaluation of this end point: one year after randomization

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.

Secondary Outcome(s)

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.

Secondary ID(s)

6100

Source(s) of Monetary Support

Hôpitaux universitaires de Strasbourg

Ethics review

Status: Approved
Approval date:
Contact: