An International, multi-center, 9-month FOLLOW-UP extension study to study the long term safety of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A.

Phase 1

• Conditions: Charcot-Marie-Tooth Disease type 1A; MedDRA version: 19.1Level: LLTClassification code 10008414Term: Charcot-Marie-Tooth diseaseSystem Organ Class: 100000004850; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2015-002379-81-FR
• Lead Sponsor: Pharnext

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2017-01-12
• Last Update Posted: 3 April 2017

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 300

Inclusion Criteria

Male or female patients with all of the following inclusion criteria will be considered for randomization:
1.Patient previously randomized to the initial PLEO-CMT study (protocol CLN-PXT3003-02) and having completed 15 months of double-blind treatment in that study, including all procedures required at the Study Termination visit (V6)
2. Patient providing a signed written informed consent to participate in the extension study and willing and able to comply with all study procedures and scheduled visits.
3. Female patients must agree to continue using an approved method of birth control throughout the extension study
Are the trial subjects under 18? yes
Number of subjects for this age range: 30
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 270
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 0

Exclusion Criteria

Patients with any of the following criteria will be excluded from entry:
1.Presenting any clinically significant change in health status that, in the opinion of the Investigator, would prevent the subject from participating in this study or successfully completing this study.
2.Any unauthorized concomitant treatments, as study CLN-PXT3003-02.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified