Phase 2
Completed N=80
Phase II, Randomized, Placebo-controlled Trial in Patients With Charcot-marie-tooth Disease Type 1A
Charcot-Marie-Tooth Disease · Hereditary Neuropathy With Liability to Pressure Palsies · genetic disorders
Source: ClinicalTrials.gov NCT01401257 ↗
Enrolled (actual)
80
Serious AEs
2.5%
Results posted
Feb 2017
Primary outcomePrimary: Safety and Tolerability of PXT3003 — 21; 21; 19; 19 Participants
Summary
The present trial is a randomized, placebo-controlled study evaluating 3 different doses of PXT3003 in patients with CMT1A disease.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Safety and Tolerability of PXT3003 |
21; 21; 19; 19 | — |
| SECONDARY To Obtain Preliminary Data on the Efficacy of PXT3003 on Clinical Scores and Functional Tests |
— | — |
| SECONDARY To Assess the Pharmacodynamic Effect of PXT3003 on PMP22 mRNA Levels and Intra-epidermal Axon Density in Cutaneous Biopsy |
— | — |
| SECONDARY To Assess the Pharmacodynamic Effect of PXT3003 on Selected Neurophysiological Parameters |
— | — |
| SECONDARY To Assess the Pharmacodynamic Effect of PXT3003 on a Series of Biochemical Biomarkers |
— | — |
| SECONDARY To Assess the Plasma Concentrations of PXT3003 |
— | — |
Eligibility Criteria
Inclusion Criteria
- 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 Criteria
- 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
Data sourced from ClinicalTrials.gov (NCT01401257). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.