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PXT3003 for Charcot-Marie-Tooth Disease (PLEO-CMT-FU Trial)

Phase 3
Waitlist Available
Led By Florian Thomas, MD PhD
Research Sponsored by Pharnext SA
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Patients previously randomized to study CLN-PXT3003-02 under placebo and dose 1 and having completed 15 months of double-blind treatment in that study, including all procedures required at the Study Termination visit (V6) or
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 9 or 24 months
Awards & highlights
No Placebo-Only Group
Pivotal Trial

Summary

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.

Who is the study for?
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.
What is being tested?
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.
What are the potential side effects?
While specific side effects are not listed here, they may include any unexpected symptoms or medical conditions that arise during the course of taking PXT3003 as observed by the investigators.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I was in the CLN-PXT3003-02 study, took placebo or dose 1, and completed 15 months including the final visit.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~9 or 24 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and 9 or 24 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Incidence of treatment-emergent adverse events (TEAEs) related to PXT3003 during the follow-up in patients with CMT1A
Secondary study objectives
Tooth structure
Compound Muscle Action Potential (CMAP) on ulnar nerve
Incidence of adverse events leading to withdrawal of study drug
+9 more
Other study objectives
Peak plasma concentration of PXT3003
Through plasma concentration of PXT3003

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups
Active Control
Group I: PXT3003 dose 1Active Control1 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 2Active Control1 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)

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
Charcot-Marie-Tooth Disease (CMT) is a hereditary neuropathy characterized by progressive muscle weakness and atrophy. The combination treatment PXT3003, which includes baclofen, naltrexone, and sorbitol, aims to reduce the overexpression of the PMP22 gene, a common cause of CMT1A. Baclofen acts as a muscle relaxant by activating GABA-B receptors, which can help alleviate muscle spasms. Naltrexone, an opioid receptor antagonist, may modulate neuroinflammation and pain. Sorbitol, a sugar alcohol, is used to enhance the bioavailability of the other components. By targeting the overexpression of PMP22, this combination therapy aims to address the underlying genetic cause of CMT1A, potentially slowing disease progression and improving patient outcomes.

Find a Location

Who is running the clinical trial?

Pharnext SALead Sponsor
6 Previous Clinical Trials
3,345 Total Patients Enrolled
4 Trials studying Charcot-Marie-Tooth Disease
3,253 Patients Enrolled for Charcot-Marie-Tooth Disease
SynteractHCRIndustry Sponsor
9 Previous Clinical Trials
1,000 Total Patients Enrolled
Syneos HealthOTHER
175 Previous Clinical Trials
68,212 Total Patients Enrolled

Media Library

PXT3003 (Other) Clinical Trial Eligibility Overview. Trial Name: NCT03023540 — Phase 3
Charcot-Marie-Tooth Disease Research Study Groups: PXT3003 dose 1, PXT3003 dose 2
Charcot-Marie-Tooth Disease Clinical Trial 2023: PXT3003 Highlights & Side Effects. Trial Name: NCT03023540 — Phase 3
PXT3003 (Other) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03023540 — Phase 3
~4 spots leftby Dec 2024