Clinical Trial: Ulipristal Acetate In Disease Charcot-Marie-Tooth Type of 1A
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: LONG-TERM EFFECTS TOLERANCE AND THE Ulipristal Acetate IN DISEASE Charcot-MARIE-TOOTH TYPE OF 1A
Brief Summary:
The disease Charcot-Marie-Tooth disease type 1A (CMT1A) is the most common inherited peripheral neuropathy, for which no treatment has proved its effectiveness. It is autosomal dominant, associated with a duplication of the chromosome 17p11.2 region which leads to overexpression of the gene and the protein-peripheral myelin protein-22 (PMP22), a major component of peripheral myelin.
In animals and humans, PMP22 mRNA level of glutathione S-transferase theta 2 and Cathepsin A (markers of oxidative stress), detected in a skin biopsy are markers that may play a role in the prognosis evolution of the disease. Furthermore, several studies have shown that the administration of progesterone increased the expression of PMP22 gene (measured in a skin biopsy) and worsening symptoms. In contrast, anti-progestins reduce the synthesis of PMP22 and improve symptoms in rat CMT1A.
The long-term safety of anti-progesterone was evaluated for mifepristone (RU486) ulipristal acetate and (EllaOne®). Few side effects have been reported including a few cases of endometrial hyperplasia reversible upon discontinuation of treatment. With the RU486, rare cases of adrenal androgen and failure have been observed. However, EllaOne® has low antagonistic action on the glucocorticoid receptor and no action on androgen receptors. The investigators therefore believe that it will be well tolerated in humans and will reduce the synthesis of PMP22 and the action of oxidative stress by improving disability of patients.
Detailed Summary:
Sponsor: University Hospital, Strasbourg, France
Current Primary Outcome: Analysis of the effectiveness of Ellaone. This will be assessed by the production of RNA PMP22 using skin biopsy. [ Time Frame: 12 months after treatment with EllaOne ]
Original Primary Outcome: Analysis of the effectiveness of Ellaone. This will be assessed by the production of RNA PMP22 using skin biopsy. [ Time Frame: At M12 after treatment for 12 months with EllaOne ]
Current Secondary Outcome:
Original Secondary Outcome:
Information By: University Hospital, Strasbourg, France
Dates:
Date Received: November 4, 2015
Date Started: October 23, 2015
Date Completion: October 23, 2018
Last Updated: May 15, 2017
Last Verified: May 2017
