Phase II, Randomized, Placebo-controlled Trial in Patients With Charcot-marie-tooth Disease Type 1A
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ClinicalTrials.gov Identifier: NCT01401257 |
Recruitment Status :
Completed
First Posted : July 25, 2011
Results First Posted : February 16, 2017
Last Update Posted : November 22, 2017
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Charcot-Marie-Tooth Disease Hereditary Neuropathy With Liability to Pressure Palsies Genetic Disorders | Drug: PXT3003 Low dose Drug: PXT3003 Intermediate Dose Drug: PXT3003 High Dose Other: Placebo | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 80 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
Primary Purpose: | Treatment |
Official 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. |
Study Start Date : | December 2010 |
Actual Primary Completion Date : | October 2011 |
Actual Study Completion Date : | December 2012 |

Arm | Intervention/treatment |
---|---|
Experimental: PXT3003 Low dose
Oral Liquid formulation, 1/100, bid, 12 months
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Drug: PXT3003 Low dose
Liquid,5 ml, twice a day, 12-month treatment
Other Name: Pleocompound PXT3003 |
Experimental: PXT3003 Intermediate dose
Oral Liquid formulation, 1/50, bid, 12 months
|
Drug: PXT3003 Intermediate Dose
Liquid,5 ml, twice a day, 12-month treatment
Other Name: Pleocompound PXT3003 |
Experimental: PXT3003 High dose
Oral Liquid formulation, 1/10, bid, 12 months
|
Drug: PXT3003 High Dose
Liquid,5 ml, twice a day, 12-month treatment
Other Name: Pleocompound PXT3003 |
Placebo Comparator: Placebo
Oral Liquid formulation, bid, 12 months
|
Other: Placebo
Liquid,5 ml, twice a day, 12-month treatment
Other Name: Pleocompound PXT3003 |
- Safety and Tolerability of PXT3003 [ Time Frame: Screening, randomization, 1-, 3-, 6-, 9-, 12-month treatment and 1-month follow-up ]
The Primary Objective is to assess the clinical and laboratory safety and tolerability of three doses of PXT3003 administered orally for 12 months to CMT1A patients versus placebo.
Number of participants with adverse events in each arm.
- 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 ]
Efficacy scores and functional tests will be assessed CMTNS/CMTES:ONLS, VAS, fatigue, pain, six minute walk test (6MWT), nine-hole peg test, quantified muscular testing (QMT; hand grip and foot dorsiflexion), CGI.
For each test or score, change from baseline after 3-,6-, 9- and 12-months of 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 ]
A cutaneous biopsy (consisting in 2 small punch biopsies) will be performed to assess PMP22 mRNA expression and intra-epidermal axon density.
Change from baseline after 12-month of treatment.
- To Assess the Pharmacodynamic Effect of PXT3003 on Selected Neurophysiological Parameters [ Time Frame: Screening, randomization, 3-, 6-, 9- and 12-month treatment ]
Electrophysiological examination will be performed to assess sensory and motor responses of the median and ulnar nerves (non-dominant side) including: NCV, compound muscle action potential (CMAP) and SNAP.
Change from baseline after 3-,6-, 9- and 12-months of treatment.
- To Assess the Pharmacodynamic Effect of PXT3003 on a Series of Biochemical Biomarkers [ Time Frame: Randomization and 3-month treatment ]
Dosages of biochemical biomarkers in plasma.
Change from baseline after 3-month of treatment.
- To Assess the Plasma Concentrations of PXT3003 [ Time Frame: Randomization, 1-, 6- and 12-month treatment ]PXT3003 plasmatic concentrations after one administration (randomization) and after 1-,6-and 12-months of treatment.

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Ages Eligible for Study: | 18 Years to 65 Years (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01401257
France | |
Hôpital Roger Salengro | |
Lille, France, 59037 | |
CHU Dupuytren | |
Limoges, France, 87042 | |
CHU Lyon Sud | |
Lyon, France, 69495 | |
Hôpital La Timone | |
Marseille, France, 13385 | |
Hôtel Dieu | |
Nantes, France, 44093 | |
Groupe Hospitalier Pitié-Salpétrière | |
Paris, France, 75013 |
Principal Investigator: | Shahram ATTARIAN, MD | Hôpital La Timone | |
Study Director: | Viviane BERTRAND, PhD | Pharnext SA |
Responsible Party: | Pharnext SA |
ClinicalTrials.gov Identifier: | NCT01401257 |
Other Study ID Numbers: |
CLN-PXT3003-01 |
First Posted: | July 25, 2011 Key Record Dates |
Results First Posted: | February 16, 2017 |
Last Update Posted: | November 22, 2017 |
Last Verified: | October 2017 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
PXT3003 Charcot-Marie-Tooth Disease Hereditary Motor and Sensory Neuropathies |
Tooth Diseases Charcot-Marie-Tooth Disease Nerve Compression Syndromes Hereditary Sensory and Motor Neuropathy Genetic Diseases, Inborn Stomatognathic Diseases Nervous System Malformations |
Nervous System Diseases Heredodegenerative Disorders, Nervous System Neurodegenerative Diseases Polyneuropathies Peripheral Nervous System Diseases Neuromuscular Diseases Congenital Abnormalities |