Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients (PLEO-CMT) (PLEO-CMT)

February 13, 2020 updated by: Pharnext SA

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

The purpose of this study is to determine whether PXT3003 is effective and safe in the treatment of Charcot-Marie-Tooth disease - Type 1 A (CMT1A). This double-blind study will assess in parallel groups 2 doses of PXT3003 compared to Placebo in CMT1A patients treated for 15 months.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

PXT3003 is a fixed dose combination of (RS)-baclofen, naltrexone hydrochloride and D-sorbitol selected via a Systems Biology approach and developed by Pharnext, with the aim to limit the production of PMP22 and protect/improve axonal function in patients with CMT1A. On September 18th 2017, PXT3003 dose 2 was prematurely discontinued, due to an unexpected investigational medicinal product quality event (failed month 18 stability testing). This resulted in a large proportion of missing data that led us to reconsider the efficacy analysis that was initially planned in the protocol.The independent data safety monitoring committee did not identify any safety concern on September 5th 2017. All patients randomised to dose 2 were requested to undergo the end of study visit, and were offered to enter the extension study (CLN-PXT3003-03).

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
323

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Belgium
      • Leuven, Belgium
        • Departement of Neurology, UZ Leuven
  • Canada
      • Quebec, Canada, G1J 1Z4
        • University Hospital of Quebec
  • France
      • Lille, France
        • Centre de Référence des Maladies Neuromusculaires, Hôpital Swynghedauwl, CHU de Lille
      • Limoges, France
        • Centre de Référence des Neuropathies Périphériques Rares, Hôpital Dupuytren, CHU Limoges
      • Lyon, France
        • Service de Neurologie et du Sommeil, CHU Lyon Sud
      • Marseille, France
        • Centre de Référence des Maladies Neuromusculaires, Pôle des Neurosciences Clinique, CHU la Timone
      • Nantes, France
        • Centre de Référence des Maladies Neuromusculaires; Hôtel Dieu, CHU de Nantes
      • Paris, France
        • Service de Neurologie, Hopital Kremlin Bicetre
  • Germany
      • Aachen, Germany
        • Department of Neurology and Institute for Neuropathology, University Hospital RWTH Aachen
      • Göttingen, Germany
        • Department of Clinical Neurophysiology, University Medical Center Göttingen
      • Munich, Germany
        • Department of Neurology, Ludwig-Maximillian University, Munich
      • Münster, Germany
        • Department for Sleep Medicine and Neuromuscular, University Hospital Münster
  • Netherlands
      • Amsterdam, Netherlands
        • Departement of Neurology, Academic Medical Center
  • Spain
      • Barcelona, Spain
        • Department of neurology, Hospital Univesitario de Bellvitge
      • Madrid, Spain
        • Servicio de Neurología, Hospital Universitario La Paz
      • Sevilla, Spain
        • Centro de Diagnostico y Tratamiento, Hospital Universitario Virgen del Rocio
      • Valencia, Spain
        • Servicio de Neurologia, Hospital Univesitari i Politécnic La Fe
  • United Kingdom
    • Manchester
      • Salford, Manchester, United Kingdom, M6 8HD
        • Department of Neurology, Salford Royal NHS Foundation Trust
    • Scotland
      • Dundee, Scotland, United Kingdom, DD1 9SY
        • Ninewells Hospital and Medical School
  • United States
    • California
      • Los Angeles, California, United States, 90048
        • Department of Neurology, Cedars-Sinai Medical Center
    • Connecticut
      • New Britain, Connecticut, United States, 06053
        • Hospital for Special Care, New Britain
    • Florida
      • Gainesville, Florida, United States, 32610
        • Department of Neurology, McKnight Brain Institute
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • University of Kansas Medical Center
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Brigham and Women's Hospital
    • Michigan
      • Ann Arbor, Michigan, United States, 48109-5322
        • University of Michigan Health System
    • Minnesota
      • Minneapolis, Minnesota, United States, 55455
        • Department of Neurology, University of Minnesota
    • Missouri
      • Saint Louis, Missouri, United States, 63104-1027
        • Department of Neurology and Psichiatry, Saint Louis University
    • New York
      • New York, New York, United States, 10032
        • Peripheral Neuropathy Center, Neurological Institue Building, Columbia University Medical Center
    • Ohio
      • Columbus, Ohio, United States, 43210
        • Ohio State University
    • Washington
      • Spokane, Washington, United States, 99202-1330
        • Saint Luke's Rehabilitation Institute

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

16 years to 65 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Triple

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Active Comparator: PXT3003 dose 1
Oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months
Drug: PXT3003 dose 1
Liquid oral solution, 5 ml twice a day, morning and evening with food
Other Names:
  • DOSE 1
Active Comparator: PXT3003 dose 2
Oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months
Drug: PXT3003 dose 2
Liquid oral solution, 5 ml twice a day, morning and evening with food
Other Names:
  • DOSE 2
Placebo Comparator: placebo
Oral solution, 5 ml b.i.d. (taken morning and evening with food) during 15 months
Drug: placebo
Liquid oral solution, 5 ml twice a day, morning and evening with food

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Overall Neuropathy Limitation Scale (ONLS) Total Score
Time Frame: From Baseline to Month 15

The primary efficacy variable used in the main analysis is the mean of the available ONLS values at month 12 and month 15.

The ONLS is a disability scale that was derived and improved from the Overall Disability Sum Score (ODSS) to measure limitations in the everyday activities of the upper limbs (rated on 5 points) and the lower limbs (rated on 7 points). The total score is a 12-point scale: 0 (no disability) to 12 (maximum disability). Lower values in the ONLS indicate a better clinical condition.

Reported values are the values at Baseline (Base) and the average of the available values at Month 12 and Month 15 (Fin).
From Baseline to Month 15

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Mean of Ten Meter Walking Test (10MWT)
Time Frame: From Baseline to Month 15

This outcome measure is the mean of the available 10MWT values at month 12 and month 15.

The 10MWT is a simple to administer, standardized, reliable and valid evaluation of functional exercise capacity and gait that has been used to evaluate neurologic disorders and CMT patients.

Lower Time to Walk 10 Meters values indicate a better clinical condition.

Reported values are the values at Baseline (Base) and the average of the available values at Month 12 and Month 15 (Fin).
From Baseline to Month 15
Mean of the CMTNS-v2 Sensory Score
Time Frame: From Baseline to Month 15

This outcome measure is the mean of the available CMTNS-v2 Sensory Score values at month 12 and month 15.

The CMTNS-v2 is a specific scale designed to assess severity of impairment in CMT disease. It is a 36-point scale based on nine items to quantify impairment (sensory symptoms, pin sensibility, vibration and arm and leg strength), activity limitations (motor symptoms arms and legs) and electrophysiological function (amplitudes of ulnar CMAP and SNAP). The CMTNS-v2 goes from 0 (no impairment) to 36 (maximum impairment) whom each sub-items goes from 0 to 4.

The CMTNS-v2 Sensory score is summed of items 1+4+5 of CMTNS-v2 (Sensory symptoms, Pinprick sensibility and Vibration). It is a 12-point score: 0 (no impairment) to 12 (maximum impairment).

Lower CMTNS-v2 Sensory Score values indicate a better clinical condition.

Reported values are the values at Baseline (Base) and the average of the available values at Month 12 and Month 15 (Fin).
From Baseline to Month 15
Mean of the CMTNS-v2 Examination Score (CMTES-v2)
Time Frame: From Baseline to Month 15

This outcome measure is the mean of the available CMTNS-v2 Examination Score values at month 12 and month 15.

The CMTNS-v2 is a specific scale designed to assess severity of impairment in CMT disease. It is a 36-point scale based on nine items to quantify impairment (sensory symptoms, pin sensibility, vibration and arm and leg strength), activity limitations (motor symptoms arms and legs) and electrophysiological function (amplitudes of ulnar CMAP and SNAP). The CMTNS-v2 goes from 0 (no impairment) to 36 (maximum impairment) whom each sub-items goes from 0 to 4.

The CMTES-v2 is summed of item 1 to 7 of the CMTNS-v2 (limited to impairment items and excluding electrophysiological items). It is a 28-point score: 0 (no impairment) to 28 (maximum impairment).

Lower CMTES-v2 values indicate a better clinical condition.

Reported values are the values at Baseline (Base) and the average of the available values at Month 12 and Month 15 (Fin).
From Baseline to Month 15
Mean of the Results at the Nine-Hole Peg Test (9-HPT)
Time Frame: From Baseline to Month 15

This outcome measure is the mean of the available 9-HPT values at month 12 and month 15.

The Nine-Hole Peg Test (9HPT) is a simple timed test of fine motor coordination of extremitied in the upper limbs. It measures the time needed by the patient to insert 9 pegs in nine holes and to remove them (normal required time 18 seconds).

Lower 9HPT values indicate a better clinical condition.

Reported values are the values at Baseline (Base) and the average of the available values at Month 12 and Month 15 (Fin).
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)

Safety selection was to include all randomized patients that have received at least one dose of study treatment.

Safety and tolerability of PXT3003 were compared to placebo on the incidence of treatment-emergent adverse events (TEAEs); they were evaluated by type/nature, severity/intensity, seriousness, and relationship to study drug.
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 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)
Safety and tolerability of PXT3003 were compared to placebo on the incidence of TEAEs leading to withdrawal of study drug.
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).
Safety and tolerability of PXT3003 were compared to placebo on the incidence of serious adverse events (SAEs).
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).

Other Outcome Measures

Other Outcome Measures

For clinical trials, a planned measurement described in the protocol that is used to determine the effect of an intervention/treatment on participants. For observational studies, a measurement or observation that is used to describe patterns of diseases or traits, or associations with exposures, risk factors, or treatment. Types of outcome measures include primary outcome measure and secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Mean of the CMTNS-v2 Sensory Symptoms
Time Frame: From Baseline to Month 15

This outcome measure is the mean of the available CMTNS-v2 Sensory Symptoms values at month 12 and month 15.

The CMTNS-v2 is a specific scale designed to assess severity of impairment in CMT disease. It is a 36-point scale based on nine items to quantify impairment (sensory symptoms, pin sensibility, vibration and arm and leg strength), activity limitations (motor symptoms arms and legs) and electrophysiological function (amplitudes of ulnar CMAP and SNAP). The CMTNS-v2 goes from 0 (no impairment) to 36 (maximum impairment) whom each sub-items goes from 0 to 4.

The CMTNS-v2 Sensory Symptoms is the first item of the CMTNS-v2. It is a 4-point score: 0 (no impairment) to 4 (maximum impairment).

Lower CMTNS-v2 Sensory Symptoms values indicate a better clinical condition.

Reported values are the values at Baseline (Base) and the average of the available values at Month 12 and Month 15 (Fin).
From Baseline to Month 15
Plasma Concentrations of Baclofen at Trough and at 90 Min After Drug Intake
Time Frame: At Month 12 and Month 15

Plasma concentration of PXT3003 components were measured at trough (prior to dose) and 90 minutes after drug intake.

The mean plasma values of the baseline correspond to half of the administered dose.
At Month 12 and Month 15
Plasma Concentrations of Naltrexone at Trough and at 90 Min After Drug Intake
Time Frame: At Month 12 and month 15

Plasma concentration of PXT3003 components were measured at trough (prior to dose) and 90 minutes after drug intake.

The mean plasma values of the baseline correspond to half of the administered dose.
At Month 12 and month 15
Plasma Concentrations of 6β-naltrexol at Trough and at 90 Min After Drug Intake
Time Frame: At Month 12 and Month 15

Plasma concentration of PXT3003 components were measured at trough (prior to dose) and peak (90 minutes post dose).

The mean plasma values of the baseline correspond to half of the administered dose.
At Month 12 and Month 15
Number of Participants With ONLS Therapy Response 1
Time Frame: From Baseline to Month 15
ONLS Therapy Response 1 was defined as the number of participants (responders) with an improvement on final ONLS Total Score of at least one point. A higher response rate indicate a better clinical condition.
From Baseline to Month 15
Number of Participants With ONLS Therapy Response 2
Time Frame: From Baseline to Month 15

ONLS Therapy Response 2 was defined as the number of participants with no deterioration (responders) on final ONLS Total Score.

A higher response rate indicates a better clinical condition.
From Baseline to Month 15

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Pharnext SA

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Principal Investigator: Shahram Attarian, MD, CHU la Timone, Marseille, France
  • Principal Investigator: Peter Young, MD, University Hospital Münster, Germany
  • Principal Investigator: Teresa Sevilla, MD, Hospital Universitari i Politècnic La Fe, Valencia, Spain
  • Principal Investigator: Marianne De Visser, MD, Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
  • Principal Investigator: Philip Van Damme, MD, UZ Leuven, Belgium
  • Principal Investigator: Mark Roberts, MD, Salford Royal NHS Foundation Trust, Manchester, UK
  • Principal Investigator: Florian Thomas, MD, Saint-Louis University, Saint-Louis, USA
  • Principal Investigator: Jack Puymirat, MD, University Hospital of Quebec

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
December 1, 2015

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
March 1, 2018

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
August 1, 2018

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
September 28, 2015

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
October 16, 2015

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
October 20, 2015

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
February 27, 2020

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
February 13, 2020

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
February 1, 2020

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • CLN-PXT3003-02
  • 2015-002378-19 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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