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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

PREMIER Trial Summary

This trial will have 2 parts: a double-blind treatment period where some people will get the drug and some will get a placebo, and an open-label extension period where everyone will get the drug. The primary endpoint will be assessed at Month 15.

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.Check my eligibility
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.See study design
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.

PREMIER Trial 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.

PREMIER Trial Timeline

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
modified Overall Neuropathy Limitation Scale (mONLS)
Secondary outcome measures
10-Meter Walk Test
CMTNS-V2
Patient Global Impression of Change (PGI-C)
+3 more

PREMIER 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

Find a Location

Who is running the clinical trial?

Pharnext S.C.A.Lead Sponsor
6 Previous Clinical Trials
3,182 Total Patients Enrolled
4 Trials studying Charcot-Marie-Tooth Disease
3,090 Patients Enrolled for Charcot-Marie-Tooth Disease
Pharnext SALead Sponsor
6 Previous Clinical Trials
3,182 Total Patients Enrolled
4 Trials studying Charcot-Marie-Tooth Disease
3,090 Patients Enrolled for Charcot-Marie-Tooth Disease
Worldwide Clinical TrialsOTHER
61 Previous Clinical Trials
14,037 Total Patients Enrolled

Media Library

Placebo (Other) Clinical Trial Eligibility Overview. Trial Name: NCT04762758 — Phase 3
Charcot-Marie-Tooth Disease Research Study Groups: PXT3003, Placebo
Charcot-Marie-Tooth Disease Clinical Trial 2023: Placebo Highlights & Side Effects. Trial Name: NCT04762758 — Phase 3
Placebo (Other) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04762758 — Phase 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Does this study have an age limit? Question: What is the expected primary outcome of the trial?Rewritten Question: What are researchers looking to learn from this trial?

"The age requirement to participate in this clinical trial is between 16-65 years old."

Answered by AI

In how many countries is this research being conducted?

"The University of Florida Health - Norman Fixel Institute for Neurological Diseases in Gainesville, Florida, Hopital de l'Enfant-Jesus in Québec, Minnesota, Advent Health Medical Group Neurology at Winter Park in Winter Park, North carolina, and 34 other locations are all running this study."

Answered by AI

What are the known risks associated with (RS)-baclofen, naltrexone hydrochloride and D-sorbitol?

"There is some data to support the efficacy of (RS)-baclofen, naltrexone hydrochloride and D-sorbitol, and this medication has been through multiple rounds of safety testing. Consequently, our team rates the safety of this drug as a 3."

Answered by AI

What are common indications for (RS)-baclofen, naltrexone hydrochloride and D-sorbitol?

"(RS)-baclofen, naltrexone hydrochloride and D-sorbitol can all be used to treat spinal cord, multiple sclerosis, and severe cerebral origin spasticity."

Answered by AI

Are there other instances where (RS)-baclofen, naltrexone hydrochloride and D-sorbitol have been studied together?

"(RS)-baclofen, naltrexone hydrochloride and D-sorbitol was first looked into in 2007 by Columbia Addiction Services and Psychotherapy Intervention Research. So far, 18434 studies have been completed with 56 more presently underway. Gainesville, Florida is conducting a large number of these active studies."

Answered by AI

Are individuals still being enrolled in this clinical research project?

"Unfortunately, this study is not currently looking for more participants. Although, it is worth noting that this study was last updated on 10/11/2022 and there are other, similar studies that are actively recruiting. There are 25 clinical trials actively recruiting participants with hmsn type ii and 56 studies for (RS)-baclofen, naltrexone hydrochloride and D-sorbitol enrolling patients as of right now."

Answered by AI

Are there any demographics that are ineligible for this research?

"The trial is looking for 350 people with hmsn type ii between the ages of 16 and 65. The most important criteria for candidates are as follows: male and non-pregnant female subjects, aged 16 to 65 years with a genetically proven diagnosis of CMT1A, able to provide written informed consent/assent and comply with study procedures, mild-to-moderate severity assessed by a CMTNS-V2 score >2 and ≤18, muscle weakness in at least foot dorsiflexion on clinical assessment, ulnar nerve motor conduction time of at least 15 m/s, If taking prescribed"

Answered by AI

Who else is applying?

How old are they?
18 - 65
What site did they apply to?
UC Davis Health Department of Physical Medicine and Rehabilitation
Hospital for Special Care
Colombia University Department of Neurology
Other
What portion of applicants met pre-screening criteria?
Did not meet criteria
Met criteria
What state do they live in?
Connecticut

How responsive is this trial?

Typically responds via
Email
Most responsive sites:
  1. Clinical Research Unit Montreal Neurological Institute and Hospital: < 24 hours
Average response time
  • < 1 Day
Recent research and studies
clinicaltrials.govStudy
Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients
2021. "Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04762758.
~87 spots leftby Apr 2025
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