MedTrace Logo

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

NCT02579759 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 323

Last updated 2020-02-27

Study results available
· View outcomes & findings →

Summary

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.

Conditions

  • Charcot-Marie-Tooth Disease Type 1A

Interventions

DRUG

PXT3003 dose 1

Liquid oral solution, 5 ml twice a day, morning and evening with food

DRUG

PXT3003 dose 2

Liquid oral solution, 5 ml twice a day, morning and evening with food

DRUG

placebo

Liquid oral solution, 5 ml twice a day, morning and evening with food

Sponsors & Collaborators

  • Pharnext S.C.A.

    lead OTHER

Principal Investigators

  • Shahram Attarian, MD · CHU La Timone, Marseille, France

  • Peter Young, MD · University Hospital Munster, Germany

  • Teresa Sevilla, MD · Hospital Universitari i Politécnic La Fe, Valencia, Spain

  • Marianne De Visser, MD · Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)

  • Philip Van Damme, MD · UZ Leuven, Belgium

  • Mark Roberts, MD · Salford Royal NHS Foundation Trust, Manchester, UK

  • Florian Thomas, MD · Saint-Louis University, Saint-Louis, USA

  • Jack Puymirat, MD · University Hospital of Quebec

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Model
PARALLEL

Eligibility

Min Age
16 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2015-12-31
Primary Completion
2018-03-31
Completion
2018-08-31

Countries

  • United States
  • Belgium
  • Canada
  • France
  • Germany
  • Netherlands
  • Spain
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02579759 on ClinicalTrials.gov