Charcot-Marie-Tooth disease 1A
Conditions
Interventions
Sponsors
Eligibility
Inclusion criteria
Inclusion criteria: 1) CMT1A patients with PMP duplications (triple gene doses) 2) Patients with normal function of main organs (heart, lung, liver and kidney) 3) Patients with no severe complications
Exclusion criteria
Exclusion criteria: 1) Patients with dementia 2) Patients with severe complications 3) Patients who are not appropriate for the trial by the decision of the physician in charge
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| An improvement in CMT neuropathy score at baseline, 4, 8 and 12 weeks thereafter. | — |
Secondary
| Measure | Time frame |
|---|---|
| An improvement in muscle strength and ulnar nerve conduction study at baseline, 4, 8 and 12 weeks thereafter. | — |
Countries
Japan
Contacts
Kyoto Prefectural University of Medicine, Graduate School of Medical Science Department of Neurology