Ascorbic acid treatment for childhood Charcot-Marie-Tooth disease 1A

JRCT ID: jRCTs031190109

Registered date:03/10/2019

Basic Information

• Recruitment status: Not Recruiting
• Health condition(s) or Problem(s) studied: Charcot-Marie-Tooth disease type 1A
• Date of first enrollment: 25/10/2019
• Target sample size: 12
• Study type: Interventional
• Intervention(s): ascorbic acid dayly intake

Outcome(s)

• Primary Outcome: CMT score
• Secondary Outcome: Electrophysiological examination, Six minutes walking distance

Key inclusion & exclusion criteria

• Age minimum: Not applicable
• Age maximum: < 18age old
• Gender: Both

Include criteria

1. Children under 18 years of age with confirmed PMP22 duplication in genetic testing.
2. Normal data in blood examination.
3. Normal data in urinary analysis.
4. Written informed consent from the patient, or parents in case of children.

Exclude criteria

1. The patient has already taken daily vitamin treatment.
2. When the investigator or the investigator considers it inappropriate to conduct this study safely.

Related Information

• Primary Sponsor: SHIOHAMA TADASHI