International, multi-center, randomized, double-blind, placebo-controlled phase III study assessing in parallel groups the efficacy and safety of 2 doses of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A treated 15 months

Recruitment & Eligibility

Status: Completed

Sex: Not specified

Target Recruitment: 20

Inclusion Criteria

1. Male or female, aged from 16 to 65 years;
2. Patient with a proven genetic diagnosis of CMT1A;
3. Mild-to-moderate severity assessed by CMTNS-v2 with a score >2 and *18;
4. Muscle weakness in at least foot dorsiflexion (clinical assessment);
5. Motor nerve conduction of the ulnar nerve of at least 15m/sec;
6. Providing signed written informed consent to participate in the study and willing and
able to comply with all study procedures and scheduled visits.

Exclusion Criteria

1. Any other associated cause of peripheral neuropathy such as diabetes;
2. Patients with another significant neurological disease or a concomitant major systemic
disease;
3. Clinically significant history of unstable medical illness since the last 30 days (unstable
angina, cancer*) that may jeopardize the participation in the study;
4. Significant hematologic disease, hepatitis or liver failure, renal failure;
5. Limb surgery within six months before randomization or planned before trial
completion;
6. Clinically significant abnormalities on the pre-study laboratory evaluation, physical
evaluation, electrocardiogram (ECG);
7. Elevated ASAT/ALAT (> 3 x ULN) and elevated serum creatinine levels (> 1.25 x ULN);
8. History of recent alcohol or drug abuse or non-adherence with treatment or other
experimental protocols;
9. Patients using unauthorized concomitant treatments including but not limited to baclofen, naltrexone, sorbitol (pharmaceutical form), opioids, levothyroxin and
potentially neurotoxic drugs such as amiodarone, chloroquine, cancer drugs susceptible
to induce a peripheral neuropathy; (list provided in appendix 1). Patients who
can/agree to stop these medications 4 weeks before randomization and during the
whole study duration can be included;
10. Female of childbearing potential (apart of patients using adequate contraceptive
measures), pregnant or breast feeding;
11. Known hypersensitivity to any of the individual components of PXT3003;
12. Porphyria as it is a contra indication to baclofen, and it may also induce neuropathy;
13. Suspected inability to complete the study follow-up (foreign workers, transient visitors,
tourists or any others for whom follow-up evaluation is not assured);
14. Limited mental capacity or psychiatric disease rendering the subject unable to provide
written informed consent or comply with evaluation procedures;
15. Patients who have participated in another trial of investigational drug(s) within the past
30 days;
16. If a patient from the same family, living in the same household, has already been
included in this study, it will not be possible to include another patient from the same
family to avoid mixing of therapeutic units; therefore there would be a risk of inversion
of the blind treatments which could jeopardize the interpretation of study results.

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Improvement of disability measured by the Overall Neuropathy Limitation Scale<br /><br>(ONLS)<br /><br>score.</p><br>

Secondary Outcome Measures

Name	Time	Method

Related Trials