What side effects are associated with this type of intervention?

The most common treatments for Charcot-Marie-Tooth Disease, particularly those similar to the fixed-dose combination of [RS]-baclofen, naltrexone hydrochloride [HCl], and D-sorbitol (PXT3003), have known side effects. Baclofen can cause drowsiness, dizziness, weakness, and fatigue. Naltrexone may lead to nausea, headache, dizziness, and fatigue. D-sorbitol often results in gastrointestinal issues such as bloating, gas, and diarrhea. When combined, these treatments may exhibit a cumulative effect of these side effects.

What prior FDA approvals exist?

As of now, there are no specific FDA-approved treatments for Charcot-Marie-Tooth Disease (CMT) that are similar to the fixed-dose combination of [RS]-baclofen, naltrexone hydrochloride [HCl], and D-sorbitol being studied in the PXT3003 trial. Current management of CMT primarily focuses on symptomatic treatment, including physical therapy, occupational therapy, and orthopedic devices to manage symptoms and improve quality of life. Research is ongoing to find more targeted therapies for this condition.

What other types of research exist?

Promising novel alternatives to PXT3003 for Charcot-Marie-Tooth Disease patients include gene therapy approaches targeting specific genetic mutations associated with CMT, antisense oligonucleotide therapies, and other pharmacological agents such as HDAC6 inhibitors and neurotrophic factors. These treatments are currently under investigation and have shown potential in preclinical and early clinical trials.

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PXT3003 for Charcot-Marie-Tooth Disease (PREMIER Trial)

Phase 3

Waitlist Available

Led By Mario Saporta, MD

Research Sponsored by Pharnext S.C.A.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Male and non-pregnant female subjects, aged 16 to 65 years with a genetically proven diagnosis of CMT1A.

Muscle weakness in at least foot dorsiflexion on clinical assessment.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from baseline to month 15

Awards & highlights

Pivotal Trial

Summary

This trial tests PXT3003, a liquid medicine combining three drugs, on patients with Charcot-Marie-Tooth disease type 1A (CMT1A). The goal is to see if it can improve nerve function and reduce symptoms in these patients. PXT3003 is a promising treatment option for patients with Charcot-Marie-Tooth disease type 1A.

Who is the study for?

This trial is for people aged 16-65 with a genetic diagnosis of Charcot-Marie-Tooth disease type 1A (CMT1A), showing specific nerve and muscle weakness symptoms. Participants should have mild-to-moderate disease severity, be on stable doses of psychoactive drugs if used, and not be pregnant.

What is being tested?

The study tests PXT3003—a mix of baclofen, naltrexone HCl, and D-sorbitol—against a placebo to see if it's effective in treating CMT1A. After the initial double-blind phase where neither doctors nor patients know who gets what treatment, all participants can receive PXT3003 in an open-label extension.

What are the potential side effects?

While the trial aims to assess safety and tolerability as secondary objectives, potential side effects are not detailed here. Typically such medications could cause digestive issues, mood changes or drowsiness.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I am between 16 and 65 years old and have a confirmed diagnosis of CMT1A.

Select...

I have weakness in lifting my foot upwards.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from baseline to month 15

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from baseline to month 15

This trial's timeline: 3 weeks for screening, Varies for treatment, and from baseline to month 15 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

modified Overall Neuropathy Limitation Scale (mONLS)

Secondary study objectives

10-Meter Walk Test

CMTNS-V2

Patient Global Impression of Change (PGI-C)

+3 more

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: PXT3003Experimental Treatment1 Intervention

Liquid oral solution, 10 mL twice a day, morning and evening with food

Group II: PlaceboPlacebo Group1 Intervention

Liquid oral solution, 10 mL twice a day, morning and evening with food

0 / 2Eligibility 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

The treatment PXT3003 for Charcot-Marie-Tooth Disease (CMT) combines [RS]-baclofen, naltrexone hydrochloride (HCl), and D-sorbitol. [RS]-baclofen is a muscle relaxant that acts on GABA-B receptors to reduce muscle spasticity, which can help alleviate muscle stiffness and cramps in CMT patients. Naltrexone HCl is an opioid receptor antagonist that may modulate pain and inflammation, potentially improving nerve function and reducing neuropathic pain. D-sorbitol is a sugar alcohol that may have neuroprotective effects, although its exact role in this combination is less clear. Together, these components aim to address multiple symptoms of CMT, including muscle spasticity, pain, and nerve degeneration, thereby improving overall patient quality of life.