What side effects are associated with this type of intervention?

The most common treatments for Charcot-Marie-Tooth Disease (CMT), particularly those similar to the combination of baclofen, naltrexone, and sorbitol (as in PXT3003), can have several side effects. Baclofen may cause drowsiness, dizziness, weakness, and fatigue. Naltrexone can lead to nausea, headache, dizziness, and fatigue. Sorbitol often results in gastrointestinal issues such as bloating, gas, and diarrhea. When combined, these medications may produce a cumulative effect of these side effects. Additionally, other treatments for CMT, such as neuropathic pain medications and physical therapy, can cause sedation, gastrointestinal disturbances, and muscle weakness.

What prior FDA approvals exist?

There is no specific mention of prior FDA approvals for treatments of Charcot-Marie-Tooth Disease (CMT) in the provided context. The trial PXT3003, which combines baclofen, naltrexone, and sorbitol, aims to reduce PMP22 overexpression, a novel approach for CMT1A. Generally, treatments for CMT focus on managing symptoms and improving quality of life, but no specific FDA-approved drugs similar to PXT3003 are detailed in the context.

What other types of research exist?

Promising novel alternatives to PXT3003 for Charcot-Marie-Tooth Disease patients include treatments that target neuropathy or neuroprotection. One potential alternative is the use of semaphorin 3A inhibitors like SM-216289 (xanthofulvin), which has shown efficacy in promoting nerve growth. Another option could be exploring the use of alpha-lipoic acid, which has been effective in treating diabetic polyneuropathy and may offer neuroprotective benefits. Further research into these and other neuroprotective agents could provide new therapeutic options for CMT patients.

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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 weeks3 visits

Treatment ~ Varies

Follow Up ~ 9 or 24 months

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)

0 / 1Eligibility criteria met

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.