Clinical trials: Charcot-Marie-Tooth Disease, Type 1A
Charcot-Marie-Tooth Type 1A
Pilot Study to Evaluate Fatigue and Fatigability Profiles and Their Impact on Walking and Balance in Patients With Charcot-Marie-Tooth Type 1A Disease Using a Global Approach in Comparison to Healthy Volunteers
Charcot-Marie-Tooth disease type 1A is the most common form of inherited neuropathy that causes peripheral nerve damage in all four limbs.
- Trial name
- FatMobCMT1A (NCT07066683)
- Testing
- CLINICAL ASSESSMENT OF BODY FUNCTION (PROM AND PHYSICAL EXAMINATION) and ACTIVITIES AND PARTICIPATION
- Where
- France
- Volunteers
- 44 people, healthy volunteers welcome
- Sponsor
- University Hospital, Strasbourg, France
Charcot-Marie-Tooth Disease, Type 1A
A First in Human Study to Assess the Safety, Tolerability, and Pharmacokinetics of EDK060 in Adults With CMT1A.
The purpose of this study is to characterize the safety, tolerability, and pharmacokinetics of EDK060 as compared to placebo in adult patients with CMT1A.
- Trial name
- NCT07140614
- Testing
- EDK060, dose A, EDK060, dose B, EDK060, dose C
- Where
- 4 sites in Canada
- Volunteers
- 28 people
- Sponsor
- Novartis Pharmaceuticals
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
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.
- Trial name
- DeteCTCMT (NCT05142059)
- Testing
- Exploratory physiopathology study, including non-invasive functional explorations carried out in patients with Charcot-Marie-Tooth disease type 1A
- Where
- France
- Volunteers
- 42 people
- Sponsor
- University Hospital, Clermont-Ferrand
Charcot-Marie-Tooth Neuropathy Type 1A
Phase I/IIa Trial of scAAV1.tMCK.NTF3 for Treatment of CMT1A
This clinical trial is an open-label one-time injection dose study in which scAAV1.tMCK.NTF3 will be administered by intramuscular injections into muscles in both legs in CMT1A subjects with PMP22 gene duplication.
- Trial name
- NCT03520751
- Testing
- scAAV1.tMCK.NTF3
- Where
- United States
- Sponsor
- Nationwide Children's Hospital
Charcot-Marie-Tooth Disease, Charcot-Marie-Tooth Disease Type 1A
Treadmill, Stretching and Proprioceptive Exercise (TreSPE) Rehabilitation Program for Charcot-Marie-Tooth Neuropathy Type 1A (CMT1A)
Charcot-Marie-Tooth neuropathy type 1A (CMT1A) is one of the most common inherited neurological disorders. The study will evaluate the efficacy and safety of an innovative rehabilitation protocol constituted by exercises at the…
- Trial name
- TreSPE (NCT01289704)
- Testing
- TreSPE, SPE
- Where
- 4 sites in Italy
- Volunteers
- 92 people, healthy volunteers welcome
- Sponsor
- University of Genova
CMT - Charcot-Marie-Tooth Disease, Peripheral Neuropathy
Effect of Orthoses and Underfoot Vibration on Balance in Neuropathy
A cross-sectional study which will look at the effects of AFO's and vibrating insoles on balance performance in people with CMT disease.
- Conditions
- CMT - Charcot-Marie-Tooth Disease, Peripheral Neuropathy, Hereditary Peripheral Neuropathy, CMT1A
- Trial name
- NCT03278093
- Where
- United Kingdom
- Volunteers
- 15 people
- Sponsor
- St George's, University of London
Charcot-Marie-Tooth Disease, Type IA
Biomarkers and Validation of Selected Outcome Measures (CMTBiomarker)
CMT is a rare disease for which novel treatments are being developed. Evaluation of intervention efficacy is hampered by slow progression and lack of sensitive outcome measures.
- Trial name
- NCT03386266
- Where
- Germany
- Volunteers
- 156 people
- Sponsor
- University Medical Center Goettingen
Charcot-Marie-Tooth Disease
Change in MUNIX in Patients With CMT1A Undergoing a Home Ankle Strengthening Program Versus Standard of Care
Here the investigators aim to show that a focused lower extremity resistance strength training program in patients with Charcot-Marie-Tooth disease (CMT) results in increased motor strength of ankle plantar- and dorsi-flexion.
- Trial name
- NCT03715283
- Testing
- Home Ankle Strengthing Program, Standard of Care
- Where
- United States
- Volunteers
- 19 people, healthy volunteers welcome
- Sponsor
- Vanderbilt University Medical Center
Charcot-Marie-Tooth Disease, Type IA
Assessing Long Term Safety and Tolerability of PXT3003 in Patients With Charcot-Marie-Tooth Disease Type 1A
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.
- Trial name
- PLEO-CMT-FU (NCT03023540)
- Testing
- PXT3003
- Where
- 23 sites in United States, Belgium and 5 more
- Volunteers
- 187 people
- Sponsor
- Pharnext S.C.A.
Charcot-Marie-Tooth Type 1A Neuropathy
Correlation Between Clinical and Electrophysiological Phenotypes in a Population of Patients With Neuropathy Charcot-Marie-Tooth Disease Type 1A
This exploratory cross-sectional study proposes, firstly, to objectify in a population of Charcot-Marie-Tooth disease type 1A (CMT 1A)if there is a correlation between the recording of electrical parameters and upper limb muscle…
- Trial name
- CCA1 (NCT01750710)
- Where
- France
- Volunteers
- 37 people
- Sponsor
- University Hospital, Clermont-Ferrand
CMT 1A
Driving Ability in Patients With CMT 1A
Charcot Marie Tooth 1A (CMT 1A) is a common inherited disorder of the nerves in the body. The disease can result in difficulties with strength and balance as well as in foot deformities such as high arches, hammer toes, and tight…
- Trial name
- NCT02357355
- Testing
- UIHC Driving Simulator
- Where
- United States
- Volunteers
- 32 people
- Sponsor
- Jerath U Nivedita
Charcot-Marie-Tooth Disease
Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients
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…
- Trial name
- PREMIER (NCT04762758)
- Testing
- (RS)-baclofen, naltrexone hydrochloride and D-sorbitol
- Where
- 50 sites in United States, Belgium and 7 more
- Volunteers
- 350 people
- Sponsor
- Pharnext S.C.A.
Chronic Inflammatory Demyelinating Polyneuropathy, Peripheral Neuropathy
SERENDEM : MD1003 in Patients Suffering From Demyelinating Neuropathies, an Open Label Pilot Study
The single-center, open-label Phase II study has the objective of assess the effect of MD1003 on motor and sensory conduction in patients suffering from demyelinating polyneuropathies in 15 subjects.
- Conditions
- Chronic Inflammatory Demyelinating Polyneuropathy, Peripheral Neuropathy, Charcot-Marie-Tooth Disease, Charcot-Marie-Tooth Disease Type 1A
- Trial name
- NCT02967679
- Testing
- MD1003
- Where
- France
- Volunteers
- 15 people
- Sponsor
- MedDay Pharmaceuticals SA
Charcot-Marie-Tooth Disease, Peripheral Neuropathy
NT-3 Levels and Function in Individuals With CMT
This study will assess the serum NT-3 levels in individuals with the diagnosis of peripheral neuropathy or any type of Charcot-Marie-Tooth Neuropathy (CMT) and correlate this with function.
- Conditions
- Charcot-Marie-Tooth Disease, Peripheral Neuropathy, Chronic Inflammatory Demyelinating Polyneuropathy
- Trial name
- NCT05011006
- Testing
- NT3 blood draw
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Zarife Sahenk
Charcot-Marie-Tooth Disease, Type IA, Peripheral Neuropathy Hereditary
Physical Activity Level at Home in CMT1A Patients: Wearable Sensor Assessment
Charcot-Marie-Tooth disease type 1A (CMT1A) is the most common hereditary peripheral neuropathy, affecting approximately 26,000 patients in France.
- Conditions
- Charcot-Marie-Tooth Disease, Type IA, Peripheral Neuropathy Hereditary, Motor Activity, Walking, Difficulty
- Trial name
- CMT1A-HOME (NCT07591779)
- Where
- France
- Volunteers
- 60 people
- Sponsor
- University Hospital, Limoges