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.
- Conditions
- Charcot-Marie-Tooth Disease type 1AMedDRA version: 19.1Level: LLTClassification code 10008414Term: Charcot-Marie-Tooth diseaseSystem Organ Class: 100000004850Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
- Registration Number
- EUCTR2015-002379-81-ES
- Lead Sponsor
- Pharnext
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 300
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
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
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method