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

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional




Official Title: International, Multi-center, Double Blind 9-month Follow-up Extension Study Assessing the Long Term Safety and Tolerability of PXT3003 in Patients With Charcot-Marie-Tooth

Brief Summary:

All randomised patients with Charcot-Marie-Tooth Type 1A (CMT1A) who completed the primary study CLN-PXT3003-02, i.e. 15-month double-blind treatment with PXT3003 or placebo, will be eligible to continue in the extension study CLN-PXT3003-03.

Patients randomised to PXT3003 dose 1 or 2 in the primary study (CLN-PXT3003-02) will continue in the extension study at the same dose, while the patients who received placebo will be assigned to one of the two active doses by a second randomization at the entry in the extension study.