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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Last refreshed on: 16 December 2017
Main ID:  NCT01401257
Date of registration: 20/07/2011
Prospective Registration: No
Primary sponsor: Pharnext SA
Public title: Phase II, Randomized, Placebo-controlled Trial in Patients With Charcot-marie-tooth Disease Type 1A
Scientific title: A Phase II, Randomized, Placebo-controlled Trial of the Safety, Efficacy, Pharmacodynamics and Pharmacokinetics of PXT3003 in Patients With Charcot-Marie-Tooth Disease Type 1A.
Date of first enrolment: December 2010
Target sample size: 80
Recruitment status: Completed
Study type:  Interventional
Study design:  Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).  
Phase:  Phase 2
Countries of recruitment
Name:     Shahram ATTARIAN, MD
Affiliation:  Hôpital La Timone
Name:     Viviane BERTRAND, PhD
Affiliation:  Pharnext SA
Key inclusion & exclusion 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

- 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

Age minimum: 18 Years
Age maximum: 65 Years
Gender: All
Health Condition(s) or Problem(s) studied
Charcot-Marie-Tooth Disease
Genetic Disorders
Hereditary Neuropathy With Liability to Pressure Palsies
Drug: PXT3003 High Dose
Drug: PXT3003 Intermediate Dose
Drug: PXT3003 Low dose
Other: Placebo
Primary Outcome(s)
Safety and Tolerability of PXT3003 [Time Frame: Screening, randomization, 1-, 3-, 6-, 9-, 12-month treatment and 1-month follow-up]
Secondary Outcome(s)
To Assess the Pharmacodynamic Effect of PXT3003 on a Series of Biochemical Biomarkers [Time Frame: Randomization and 3-month treatment]
To Assess the Pharmacodynamic Effect of PXT3003 on PMP22 mRNA Levels and Intra-epidermal Axon Density in Cutaneous Biopsy [Time Frame: Randomization and 12-month treatment]
To Assess the Pharmacodynamic Effect of PXT3003 on Selected Neurophysiological Parameters [Time Frame: Screening, randomization, 3-, 6-, 9- and 12-month treatment]
To Assess the Plasma Concentrations of PXT3003 [Time Frame: Randomization, 1-, 6- and 12-month treatment]
To Obtain Preliminary Data on the Efficacy of PXT3003 on Clinical Scores and Functional Tests [Time Frame: Screening, randomization, 3-, 6-, 9- and 12-months treatment]
Secondary ID(s)
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Ethics review
Results available: Yes
Date Posted: 16/02/2017
Date Completed:
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