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Ulipristal Acetate In Disease Charcot-Marie-Tooth Type of 1A

NCT02600286 · Status: TERMINATED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 23

Last updated 2022-07-25

No results posted yet for this study

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.

Conditions

  • CMT1A

Interventions

DRUG

EllaOne

* 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.

DRUG

EllaOne placebo

* 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.

Sponsors & Collaborators

  • University Hospital, Strasbourg, France

    lead OTHER

Principal Investigators

  • Andoni Echaniz-Laguna, MD · University Hospital, Strasbourg, France

  • Nicolas Collongues, MD · University Hospital, Strasbourg, France

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Max Age
70 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2015-10-23
Primary Completion
2017-11-30
Completion
2017-11-02

Countries

  • France

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02600286 on ClinicalTrials.gov