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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

TERMINATED

Clinical Phase

PHASE2

Total Enrollment

23 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-10-23

Study Completion Date

2017-11-02

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

Phase II, Randomized, Placebo-controlled Trial in Patients With Charcot-marie-tooth Disease Type 1A

NCT01401257

Charcot-Marie-Tooth Disease Hereditary Neuropathy With Liability to Pressure Palsies Genetic Disorders

COMPLETED PHASE2

Placebo Controlled Study of ONO2506PO in the Presence of Riluzole in Patients With Amyotrophic Lateral Sclerosis (ALS)

NCT00403104

Amyotrophic Lateral Sclerosis (ALS)

COMPLETED PHASE2

Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients

NCT04762758

Charcot-Marie-Tooth Disease

ACTIVE_NOT_RECRUITING PHASE3

Riluzole in the Treatment of Spasticity in the Traumatic Chronic Spinal Cord Injury Condition

NCT02859792

Spinal Cord Injury

UNKNOWN PHASE2

Retrospective Non-interventional Study of Stiripentol Use in Dravet Patients in the USA

NCT05544058

Dravet Syndrome

COMPLETED

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

CMT1A

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

1

Arm (1) will be randomised to receive either :

* 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.

Group Type EXPERIMENTAL