PXT3003 for Charcot-Marie-Tooth Disease

(PLEO-CMT-FU Trial)

Recruiting at 22 trial locations

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Pharnext SA

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

187 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial involves PXT3003, a mix of three low-dose drugs, aimed at patients with CMT1A. The drug combination aims to improve nerve function and reduce disability. Earlier research has shown preliminary evidence of efficacy for PXT3003 in treating CMT1A.

Research Team

Teresa Sevilla, MD

Principal Investigator

Hospital Universitario i Politécnico La F, Valencia, Spain

Florian Thomas, MD PhD

Principal Investigator

Seton Hall-Hackensack-Meridian School of Medicine, Hackensack, USA

Marianne de Visser, MD

Principal Investigator

Academic Medical Center, Amsterdam, Netherlands

Mark Roberts, MD

Principal Investigator

Selor Royal NHS Foundation Trust, Manchester, UK

Shahram Attarian, MD

Principal Investigator

CHU la Timone, Marseille, France

Eligibility Criteria

This trial is for patients with Charcot-Marie-Tooth Disease Type 1A who completed the primary study CLN-PXT3003-02. Participants must agree to birth control use, have finished 15 months of prior treatment, and sign a new consent form. Children aged 16-18 need parental consent. Those with significant health changes or using unauthorized treatments can't join.

Inclusion Criteria

I was in the CLN-PXT3003-02 study, took placebo or dose 1, and completed 15 months including the final visit.

I have signed, or if under 18, my parents and I have both signed the consent form for this study.

Patients whose V6 was performed within 4 weeks before entering the extension study or if not done must have a new baseline visit (VB)

Exclusion Criteria

Any clinically significant change in health status that, in the opinion of the Investigator, would prevent the subject from participating in this study or successfully completing this study

I am not taking any medications that are not allowed in the study.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Period 1

Patients continue on PXT3003 dose 1 or dose 2 for 9 months

9 months

Regular visits as per study protocol

Period 2

All patients continue on twice dose 1 (2X5mL)

15 months

Regular visits as per study protocol

Follow-up

Participants are monitored for safety and effectiveness after treatment

9 months

Treatment Details

Interventions

• PXT3003

Trial Overview The trial tests PXT3003's long-term safety and tolerability in CMT1A patients. It continues from a previous study where participants either received PXT3003 or placebo. Now all receive PXT3003 at varying doses over two periods to assess ongoing effects.

Participant Groups

Group I: PXT3003 dose 1 Active Control 1 Intervention

Period 1, PXT3003 : Liquid oral solution (0.6 mg/mL baclofen, 0.07 mg/mL naltrexone HCl and 210 mg/mL D-sorbitol), 5 mL bid (taken morning and evening with food) for 9 consecutive months

Group II: PXT3003 dose 2 Active Control 1 Intervention

Period 1, PXT3003: Liquid oral solution (1.2 mg/mL baclofen, 0.14 mg/mL naltrexone HCl and 420 mg/mL D-sorbitol), 5 mL bid (taken morning and evening with food) for 9 consecutive months Period 2, PXT3003: Liquid oral solution (0.6 mg/mL baclofen, 0.07 mg/mL naltrexone HCl and 210 mg/mL D-sorbitol), 10 mL bid (taken morning and evening with food)

Who Is Running the Clinical Trial?

Pharnext SA

Lead Sponsor

Trials

7

Recruited

4,400+

Pharnext S.C.A.

Lead Sponsor

Trials

7

Recruited

4,400+

SynteractHCR

Industry Sponsor

Trials

10

Recruited

1,200+

Syneos Health

Collaborator

Trials

181

Recruited

69,600+

Amarex

Collaborator

Trials

1

Recruited

190+

Theradis

Collaborator

Trials

1

Recruited

190+

Premier Research Group plc

Industry Sponsor

Trials

65

Recruited

74,200+

John Ratliff

Premier Research Group plc

Chief Executive Officer since 2024

MBA

Dr. Milena Kanova-Petrova

Premier Research Group plc

Chief Medical Officer since 2024

MD

Synteract HCR (Syneos Health)

Collaborator

Trials

1

Recruited

190+

Eurofins Optimed

Industry Sponsor

Trials

17

Recruited

1,900+

Greenphire

Collaborator

Trials

1

Recruited

190+