Clinical trials: Charcot-Marie-Tooth Type 1A
Charcot-Marie-Tooth Disease, Type Ia
High Dose Ascorbic Acid Treatment of CMT1A
This study will look at the impact of ascorbic acid (Vitamin C) on the progression of disease in people with CMT1A as compared to volunteers receiving a placebo.
- Trial name
- NCT00484510
- Testing
- Ascorbic acid (Vitamin C)
- Where
- 3 sites in United States
- Volunteers
- 110 people
- Sponsor
- Wayne State University
Charcot-Marie-Tooth Type 1A
A Study to Assess the Efficacy and Safety of PXT3003 in Charcot-Marie-Tooth Type 1A
This is a randomized, double-blind, placebo-controlled and multicenter 3 phase trial evaluating the therapeutic effect and safety of CMT1A by PXT3003.
- Trial name
- NCT05092841
- Testing
- PXT3003
- Where
- 25 sites in China
- Volunteers
- 176 people
- Sponsor
- Tasly GeneNet Pharmaceuticals Co., Ltd
Charcot-Marie-Tooth Disease Type 1A
Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients (PLEO-CMT)
The purpose of this study is to determine whether PXT3003 is effective and safe in the treatment of Charcot-Marie-Tooth disease - Type 1 A (CMT1A).
- Trial name
- PLEO-CMT (NCT02579759)
- Testing
- PXT3003 dose 1, PXT3003 dose 2
- Where
- 30 sites in United States, Belgium and 6 more
- Volunteers
- 323 people
- Sponsor
- Pharnext S.C.A.
Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1
Baby Detect : Genomic Newborn Screening
Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions.
- Conditions
- Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1, Phosphoglucomutase 1 Deficiency, Maturity Onset Diabetes of the Young
- Trial name
- BabyDetect (NCT05687474)
- Where
- Belgium
- Volunteers
- 6,824 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Liege
Charcot-Marie-Tooth Disease Type 1A
Identification of Novel Biomarkers in Early Charcot-Marie-Tooth 1A Disease
This is a 2-year follow-up study of a cohort of 35 CMT1A patients and 20 healthy volunteers. The main objective is identifying prognostic markers for CMT1A using multi-omics analysis.
- Trial name
- CMT-MODS (NCT07049588)
- Testing
- Quantitative neuromuscular MRI, Skin biopsy, Clinical scores
- Where
- France
- Volunteers
- 55 people, healthy volunteers welcome
- Sponsor
- Assistance Publique Hopitaux De Marseille
Charcot-Marie-Tooth Disease, Hereditary Motor and Sensory Neuropathies
Ascorbic Acid Treatment in CMT1A Trial (AATIC)
Charcot-Marie-Tooth type IA (CMT1A) is the most prevalent hereditary peripheral neuropathy. Demyelination of peripheral nerves is the hallmark of CMT1A.
- Trial name
- AATIC (NCT00271635)
- Testing
- ascorbic acid
- Where
- Netherlands
- Volunteers
- 13 people
- Sponsor
- Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
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 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.