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Charcot-Marie-Tooth Disease clinical trials

View clinical trials related to Charcot-Marie-Tooth Disease.

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NCT ID: NCT06121466 Active, not recruiting - Clinical trials for Anterior Cutaneous Nerve Entrapment Syndrome

Effect of Abdominal Wall Injections on Abdominal Pain

Start date: January 1, 2023
Phase: Phase 4
Study type: Interventional

This is a prospective cohort study of outpatient adults with chronic abdominal wall pain receiving abdominal wall injections, as part of their usual care, with lidocaine. Subjects will be recruited at the outpatient gastroenterology clinic at OHSU.

NCT ID: NCT05947578 Active, not recruiting - Clinical trials for Charcot Marie Tooth Disease, Type 1

The Safety and Tolerability of CLZ-2002 in Patients With Charcot-Marie Tooth Disease.

Start date: June 19, 2023
Phase: Phase 1
Study type: Interventional

A Phase 1, Open-Label, Prospective, Dose-finding Clinical Trial for Evaluation of Safety and Tolerability of Intramuscular Injections of CLZ-2002 for the Treatment of Subjects with Charcot-Marie-Tooth type 1(CMT 1)

NCT ID: NCT05142059 Active, not recruiting - Clinical trials for Charcot-Marie-Tooth Type 1A Neuropathy

Fall Risk Assessment in a Population of Charcot-Marie-Tooth Disease Type 1A (CMT 1A) by Timed Up and Go Test

DeteCTCMT
Start date: September 3, 2020
Phase: N/A
Study type: Interventional

The main objective of this study is to explore the relationship between the onset of fall and the time taken to complete the Timed Up and Go test (TUG) in this CMT1A patient population. The investigators hypothesize that patients with balance disorders and therefore a risk of major fall will require a longer time to perform the Timed Up and Go test. In addition, it seems important to confirm that the severity of the disease has a negative impact on the frequency of balance disorders.

NCT ID: NCT04762758 Active, not recruiting - Clinical trials for Charcot-Marie-Tooth Disease

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

PREMIER
Start date: March 30, 2021
Phase: Phase 3
Study type: Interventional

The study will consist of 2 periods: Double-blind Treatment and Open-Label Extension(OLE) Period. -Double-blind Treatment Period - This will be randomized, double-blind, placebo-controlled part of the study which will be conducted in parallel groups, ie,1 group receiving the active treatment (PXT3003) and the other group receiving placebo. Primary endpoint of the study will be assessed at Month 15. -Open-label Extension (OLE) Period - All subjects completing Double-blind Treatment Period will be given an opportunity to enter the OLE Period of the study and receive the active treatment (PXT3003). The duration of the OLE Period will be based on Sponsor discretion, ie, Sponsor intends to keep the study open until the study drug PXT3003 is commercially available. During this period, the long-term safety and efficacy of PXT3003 will be assessed as an exploratory objective. Double-blind Treatment Period Objectives: Primary: To evaluate the efficacy of treatment with PXT3003 (a fixed-dose combination of [RS]-baclofen, naltrexone hydrochloride [HCl], and D-sorbitol) compared to placebo in subjects with Charcot-Marie-Tooth disease type 1A (CMT1A). Secondary: To evaluate the safety and tolerability of PXT3003 treatment in subjects with CMT1A. Exploratory: To characterize the relationship between plasma biomarkers and response to PXT3003 treatment. OLE Period Objective: Exploratory: To evaluate the long-term safety and efficacy of PXT3003.

NCT ID: NCT03023540 Active, not recruiting - Clinical trials for Charcot-Marie-Tooth Disease, Type IA

Assessing Long Term Safety and Tolerability of PXT3003 in Patients With Charcot-Marie-Tooth Disease Type 1A

PLEO-CMT-FU
Start date: March 7, 2017
Phase: Phase 3
Study type: Interventional

All randomised patients with Charcot-Marie-Tooth Type 1A (CMT1A) who completed the primary study CLN-PXT3003-02, i.e. treatment with PXT3003 or placebo, are eligible to continue in the extension study CLN-PXT3003-03. Period 1: Patients randomised to PXT3003 dose 1 or placebo in the primary study (CLN-PXT3003-02) continued in the extension study on PXT3003 dose 1 (5 mL). Patients randomised to PXT3003 dose 2 (5 mL) in the primary study (CLN-PXT3003-02) continued in the extension study on PXT3003 dose 2 or PXT3003 twice dose 1 (2x5 mL). Period 2: All patients continue on twice dose 1 (2X5mL).

NCT ID: NCT02699190 Active, not recruiting - Clinical trials for Adrenoleukodystrophy

LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies

Start date: January 6, 2017
Phase:
Study type: Observational

Leukodystrophies, and other heritable disorders of the white matter of the brain, were previously resistant to genetic characterization, largely due to the extreme genetic heterogeneity of molecular causes. While recent work has demonstrated that whole genome sequencing (WGS), has the potential to dramatically increase diagnostic efficiency, significant questions remain around the impact on downstream clinical management approaches versus standard diagnostic approaches.