Status:
COMPLETED
Phase II, Randomized, Placebo-controlled Trial in Patients With Charcot-marie-tooth Disease Type 1A
Lead Sponsor:
Pharnext S.C.A.
Conditions:
Charcot-Marie-Tooth Disease
Hereditary Neuropathy With Liability to Pressure Palsies
Eligibility:
All Genders
18-65 years
Phase:
PHASE2
Brief Summary
The present trial is a randomized, placebo-controlled study evaluating 3 different doses of PXT3003 in patients with CMT1A disease.
Detailed Description
In addition to the safety and tolerability of the treatment, clinical, electrophysiological and biological endpoints (PMP22 mRNA, skin biopsy histology and plasma biomarkers) will be assessed. Standar...
Eligibility Criteria
Inclusion
- DNA proven CMT1A
- Muscle weakness in at least foot dorsiflexion (clinical assessment)
- Age between 18 and 65 years
- Male or non pregnant, non breastfeeding female
- CMT neuropathy score at screening ≤ 20
- Agrees to perform electrorophysiological studies and two cutaneous biopsies for determination of PMP22 expression and histology
- Providing signed written informed consent to participate in the study and willing and able to comply with all study procedures and scheduled visits
Exclusion
- Patients with another neurological disease
- Patients using unauthorized concomitant treatments, ascorbic acid, opioids, levothyroxine and potentially neurotoxic drugs. Patients who can/agree to stop these medications 4 weeks before randomization can be included
- Patients who have participated in another trial of investigational drug within the past 30 days
- Concomitant major systemic disease
- Clinically significant history of unstable medical illness over the last 30 days (unstable angina…)
- History of significant hematologic, kidney, liver disease, or insulin-dependent diabetes
- Clinically significant abnormalities on the prestudy laboratory evaluation, physical evaluation, electrocardiogram (ECG)
- ASAT/ALAT levels above the upper limit of normal (ULN). However, patients with an isolated elevation of either ASAT or ALAT (\<1.5 ULN) can be included at investigators" discretion if the remaining liver function tests are normal and if ASAT or ALAT value is stable at 2 distinct evaluations in the month prior to inclusion
- Serum creatinine levels above the upper limit of normal
- Limited mental capacity or psychiatric disease rendering the subject unable to provide written informed consent or comply with evaluation procedures
- History of recent alcohol or drug abuse or non-adherence with treatment or other experimental protocols
- Female of childbearing potential (apart of patient using adequate contraceptive measures), pregnant or breast feeding
- Suspected inability to complete the study follow-up (foreign workers, transient visitors, tourists or any others for whom follow-up evaluation is not assured)
- Limb surgery in the six months before randomization or planned before completion of the trial
- Known hypersensitivity to any of the individual components of PXT3003
- Porphyria
Key Trial Info
Start Date :
December 1 2010
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 1 2012
Estimated Enrollment :
80 Patients enrolled
Trial Details
Trial ID
NCT01401257
Start Date
December 1 2010
End Date
December 1 2012
Last Update
November 22 2017
Active Locations (6)
Enter a location and click search to find clinical trials sorted by distance.
1
Hôpital Roger Salengro
Lille, France, 59037
2
CHU Dupuytren
Limoges, France, 87042
3
CHU Lyon Sud
Lyon, France, 69495
4
Hôpital La Timone
Marseille, France, 13385