Phase II, Randomized, Placebo-controlled Trial in Patients With Charcot-marie-tooth Disease Type 1A

• Register: ClinicalTrials.gov
• Last refreshed on: 17 October 2012
• Main ID: NCT01401257
• Date of registration: 20/07/2011
• Primary sponsor: Pharnext, SAS
• 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: Active, not recruiting
• URL: http://clinicaltrials.gov/show/NCT01401257
• Study type: Interventional
• Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Countries of recruitment

France

Contacts

• Name: Shahram ATTARIAN, MD
• Affiliation: Hôpital La Timone

• Name: Viviane BERTRAND, PhD
• Affiliation: Pharnext, SAS

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
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



Age minimum: 18 Years
Age maximum: 65 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Charcot-Marie-Tooth Disease

Genetic Disorders

Hereditary Neuropathy With Liability to Pressure Palsies

Tooth Disorders

Intervention(s)

Drug: PXT3003

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)

CLN-PXT3003-01

Source(s) of Monetary Support

Please refer to primary and secondary sponsors