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Active clinical trials for "Charcot-Marie-Tooth Disease"

Results 21-30 of 88

Genetics of Charcot Marie Tooth (CMT) - Modifiers of CMT1A, New Causes of CMT2

Charcot-Marie-Tooth DiseaseType Ia (Disorder)1 more

This project includes two projects. One is looking for new genes that cause Charcot Marie Tooth disease (CMT). The other is looking for genes that do not cause CMT, but may modify the symptoms a person has.

Recruiting19 enrollment criteria

Development of Charcot Marie Tooth Disease (CMT) Pediatric Scale for Children With CMT

Charcot Marie Tooth Disease

The primary goal of this project is to develop and test a Charcot Marie Tooth disease (CMT) Pediatric Scale for use in evaluation in natural history CMT study.

Recruiting5 enrollment criteria

A Registered Cohort Study on Charcot-Marie-Tooth Disease

Charcot-Marie-Tooth Disease

The aim of the study is to analyze the natural history data data from Charcot-Marie-Tooth disease and related disorders in China, to assess the clinical, genetic, epigenetic features of patients with Charcot-Marie-Tooth disease, and to optimize clinical management.

Recruiting5 enrollment criteria

Validation Study of Posturology Platforms for Evaluating Postural Control of Hemiparetic and Neuro-muscular...

Neuromuscular DiseasesHemiparesis1 more

The instrumental evaluation of standing postural control by posturographic analysis supplements the clinical evaluation and, as such, is recommended by the French National Authority for Health (HAS 2007). The quantitative data obtained after a standardized postural examination appear relevant for the longitudinal follow-up of neuromuscular patients and hemiparetic patients. Neuro muscular (NM) diseases are progressive, these instrumental evaluations can highlight a deterioration or stabilization of postural control possibly not observable with the scores on clinical scales. The K-Force Plates, recently developed appear to be an interesting alternative to the stabilometry platforms currently used. Static and dynamic conditions in upright position are tested. Moreover, posturologic, kinematics data and clinical scores are correlated to increase understanding of strategies during postural control tasks in patients with stroke and in patients with NM disorders.

Recruiting11 enrollment criteria

Natural History Study for Charcot Marie Tooth Disease

Charcot-Marie-Tooth DiseaseCharcot-Marie-Tooth51 more

The goal of this Natural History Study for Charcot-Marie-Tooth is to acquire, record, and analyze patient-reported data and associated genetic reports, Electronic Health Records (EHRs) and clinical notes to identify the burden, diagnostic journey, and prevalence of disease that will aid scientists in their work toward finding a cure. Participants will be asked to complete a Natural History Survey.

Recruiting5 enrollment criteria

A Study to Assess the Safety and Effectiveness of FLX-787 in Subjects With Charcot-Marie-Tooth Disease...

Charcot-Marie-Tooth Disease

The COMMIT Study will assess the safety and effectiveness of FLX-787 in men and women with Charcot-Marie-Tooth disease (CMT) experiencing muscle cramps. Participants will be asked to take two study products during the course of the study. One of these study products will be a placebo. Approximately 120 participants in 20 study centers across the United States are expected to take part. Participants will be in the study for approximately 3 months and visit the study clinic 3 times.

Terminated11 enrollment criteria

Study of ACE-083 in Patients With Charcot-Marie-Tooth Disease

Charcot-Marie-Tooth Disease

This is a multicenter, phase 2 study to evaluate the safety, tolerability, pharmacodynamics (PD), efficacy, and pharmacokinetics (PK) of ACE-083 in patients with Charcot-Marie-Tooth Disease Type 1 and Type X (CMT1 and CMTX), to be conducted in two parts. Part 1 is non-randomized, open-label, dose-escalation and Part 2 is randomized, double-blind, and placebo-controlled.

Terminated36 enrollment criteria

Erythropoietin Spinal Cord Compression Randomized Trial

Nerve Compression Syndromes

To determine whether erythropoietin, steroids and radiotherapy is safe and feasible to administer to patients with malignant spinal cord compression.

Terminated19 enrollment criteria

Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients With Facioscapulohumeral...

Facioscapulohumeral Muscular DystrophyCharcot-Marie-Tooth Disease

This is an open-label, multicenter, phase 2 extension study to evaluate the safety, tolerability, PK, PD, and efficacy of ACE-083 in subjects with FSHD previously enrolled in Study A083-02 and subjects with CMT1 and CMTX previously enrolled in Study A083-03. This study will be conducted in two Parts: Part 1, which is a loading phase of 6 months' duration, and Part 2, the maintenance phase, which will last up to 24 months.

Terminated24 enrollment criteria

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

CMT1A

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.

Terminated20 enrollment criteria
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