Assessing Long Term Safety and Tolerability of PXT3003 in Patients With Charcot-Marie-Tooth Disease Type 1A

Summary

All randomised patients with Charcot-Marie-Tooth Type 1A (CMT1A) who completed the primary study CLN-PXT3003-02, i.e. treatment with PXT3003 or placebo, are eligible to continue in the extension study CLN-PXT3003-03.

Period 1: Patients randomised to PXT3003 dose 1 or placebo in the primary study (CLN-PXT3003-02) continued in the extension study on PXT3003 dose 1 (5 mL). Patients randomised to PXT3003 dose 2 (5 mL) in the primary study (CLN-PXT3003-02) continued in the extension study on PXT3003 dose 2 or PXT3003 twice dose 1 (2x5 mL).

Period 2: All patients continue on twice dose 1 (2X5mL).

Conditions

• Charcot-Marie-Tooth Disease, Type IA

Interventions

DRUG

PXT3003

Liquid oral solution, twice 5 mL (Dose 1) bid

Sponsors & Collaborators

• Synteract HCR (Syneos Health)

  collaborator UNKNOWN

• Premier Research Group plc

  collaborator UNKNOWN

• Greenphire

  collaborator UNKNOWN

• Theradis

  collaborator UNKNOWN

• Amarex

  collaborator UNKNOWN

• Eurofins Optimed

  collaborator INDUSTRY

• Pharnext S.C.A.

  lead OTHER

Principal Investigators

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

• Teresa Sevilla, MD · Hospital Universitario i Politécnico La F, Valencia, Spain

• Marianne de Visser, MD · Academic Medical Center, Amsterdam, Netherlands

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

• Florian Thomas, MD PhD · Seton Hall-Hackensack-Meridian School of Medicine, Hackensack, USA

Study Design

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Model

PARALLEL

Eligibility

Min Age

17 Years

Max Age

67 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2017-03-07

Primary Completion

2024-12-31

Completion

2024-12-31

FDA Drug

Yes

Countries

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

Study Locations