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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.
Register: ClinicalTrials.gov
Last refreshed on: 12 December 2020
Main ID:  NCT02579759
Date of registration: 28/09/2015
Prospective Registration: Yes
Primary sponsor: Pharnext SA
Public title: Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients (PLEO-CMT) PLEO-CMT
Scientific title: International, Multi-center, Randomized, Double-blind, Placebo-controlled Phase III Study Assessing in Parallel Groups the Efficacy and Safety of 2 Doses of PXT3003 in Patients With Charcot-Marie-Tooth Disease Type 1A Treated 15 Months
Date of first enrolment: December 2015
Target sample size: 323
Recruitment status: Completed
URL:  https://clinicaltrials.gov/show/NCT02579759
Study type:  Interventional
Study design:  Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Triple (Participant, Investigator, Outcomes Assessor).  
Phase:  Phase 3
Countries of recruitment
Belgium Canada France Germany Netherlands Spain United Kingdom United States
Contacts
Name:     Marianne De Visser, MD
Address: 
Telephone:
Email:
Affiliation:  Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Name:     Shahram Attarian, MD
Address: 
Telephone:
Email:
Affiliation:  CHU La Timone, Marseille, France
Name:     Teresa Sevilla, MD
Address: 
Telephone:
Email:
Affiliation:  Hospital Universitari i Politécnic La Fe, Valencia, Spain
Name:     Florian Thomas, MD
Address: 
Telephone:
Email:
Affiliation:  Saint-Louis University, Saint-Louis, USA
Name:     Mark Roberts, MD
Address: 
Telephone:
Email:
Affiliation:  Salford Royal NHS Foundation Trust, Manchester, UK
Name:     Peter Young, MD
Address: 
Telephone:
Email:
Affiliation:  University Hospital Munster, Germany
Name:     Jack Puymirat, MD
Address: 
Telephone:
Email:
Affiliation:  University Hospital of Quebec
Name:     Philip Van Damme, MD
Address: 
Telephone:
Email:
Affiliation:  UZ Leuven, Belgium
Key inclusion & exclusion criteria

Inclusion Criteria:

- Male or female, aged from 16 to 65 years;

- Patient with a proven genetic diagnosis of CMT1A;

- Mild-to-moderate severity assessed by Charcot-Marie-Tooth Neuropathy Score (version 2)
with a score >2 and =18;

- Muscle weakness in at least foot dorsiflexion;

- Motor nerve conduction of the ulnar nerve of at least 15 m/sec;

- 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:

- Any other associated cause of peripheral neuropathy such as diabetes;

- Patient with another significant neurological disease or a concomitant major systemic
disease;

- Clinically significant history of unstable medical illness since the last 30 days
(unstable angina, cancer…) that may jeopardize the participation in the study;

- Significant hematologic disease, hepatitis or liver failure, renal failure;

- Limb surgery within six months before randomization or planned before trial
completion;

- Clinically significant abnormalities on the pre-study laboratory evaluation, physical
evaluation, electrocardiogram (ECG);

- Elevated ASAT/ALAT (> 3 x ULN) and elevated serum creatinine levels (> 1.25 x ULN);

- History of recent alcohol or drug abuse or non-adherence with treatment or other
experimental protocols;

- Patient using unauthorized concomitant treatments including but not limited to
baclofen, naltrexone, sorbitol (pharmaceutical form), opioids, levothyroxin and
potentially neurotoxic drugs such as amiodarone, chloroquine, cancer drugs susceptible
to induce a peripheral neuropathy. Patient who can/agrees to stop these medications 4
weeks before randomization and during the whole study duration can be included;

- Female of childbearing potential (apart of patient using adequate contraceptive
measures), pregnant or breast feeding;

- Known hypersensitivity to any of the individual components of PXT3003;

- Porphyria as it is a contra indication to baclofen, and it may also induce neuropathy;

- Suspected inability to complete the study follow-up (foreign workers, transient
visitors, tourists or any others for whom follow-up evaluation is not assured);

- Limited mental capacity or psychiatric disease rendering the subject unable to provide
written informed consent or comply with evaluation procedures;

- Patient who has participated in another trial of investigational drug(s) within the
past 30 days;

- If a patient from the same family, living in the same household, has already been
included in this study, it will not be possible to include another patient from the
same family to avoid mixing of therapeutic units; therefore there would be a risk of
inversion of the blind treatments which could jeopardize the interpretation of study
results.



Age minimum: 16 Years
Age maximum: 65 Years
Gender: All
Health Condition(s) or Problem(s) studied
Charcot-Marie-Tooth Disease Type 1A
Intervention(s)
Drug: placebo
Drug: PXT3003 dose 1
Drug: PXT3003 dose 2
Primary Outcome(s)
Overall Neuropathy Limitation Scale (ONLS) Total Score [Time Frame: From Baseline to Month 15]
Secondary Outcome(s)
Incidence of AE Leading to Withdrawal of Study Drug [Time Frame: The period between the patient signing the informed consent and 30 days after the end of study (i.e. completion/early discontinuation/last contact as recorded on the 'Study Completion on Early Termination' form up to 15 months)]
Incidence of SAEs [Time Frame: The period between the patient signing the informed consent and 30 days after the end of study (i.e. completion/early discontinuation/last contact as recorded on the 'Study Completion on Early Termination' form up to 15 months).]
Mean of Ten Meter Walking Test (10MWT) [Time Frame: From Baseline to Month 15]
Mean of the CMTNS-v2 Examination Score (CMTES-v2) [Time Frame: From Baseline to Month 15]
Mean of the CMTNS-v2 Sensory Score [Time Frame: From Baseline to Month 15]
Mean of the Results at the Nine-Hole Peg Test (9-HPT) [Time Frame: From Baseline to Month 15]
Number of Subjects With at Least One TEAE [Time Frame: The period between the patient signing the informed consent and 30 days after the end of study (i.e. completion/early discontinuation/last contact as recorded on the 'Study Completion on Early Termination' form up to 15 months)]
Secondary ID(s)
2015-002378-19
CLN-PXT3003-02
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Ethics review
Results
Results available: Yes
Date Posted: 27/02/2020
Date Completed:
URL: https://clinicaltrials.gov/ct2/show/results/NCT02579759
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