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

Phase 3
Waitlist Available
Led By Mark Roberts, MD
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

PLEO-CMT-FU Trial Summary

This trial is for patients who have completed the primary study CLN-PXT3003-02, which was testing PXT3003 (a potential treatment for Charcot-Marie-Tooth Type 1A) against placebo. In the extension study, CLN-PXT3003-03, all patients from the primary study are eligible to continue, and will be given PXT3003 at either dose 1 (5 mL) or dose 2 (5 mL) depending on what they received in the primary study. In Period 2 of the extension study, all patients will receive PXT3003 at twice dose 1 (2x5

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.Check my eligibility
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.See study design
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.

PLEO-CMT-FU Trial 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.

PLEO-CMT-FU Trial 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

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

PLEO-CMT-FU 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)

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
172 Previous Clinical Trials
68,267 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

Frequently Asked Questions

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

Are there any other notable cases of PXT3003 being used for medicinal purposes?

"PXT3003's first dose was studied in 2017 at the Departement of Neurology, UZ Leuven. In total, there have been 2 completed clinical trials since then with 2 more ongoing as of now. These latter studies are based in Ann Arbor, Michigan."

Answered by AI

Are there any current openings for this clinical trial research program?

"Data from clinicaltrials.gov shows that this particular study is not currently taking on any more patients. Although, there are 27 other trials with open enrollment at the moment."

Answered by AI

How do I sign up for the research program?

"This clinical trial is seeking 187 individuals that have charcot-marie-tooth disease type ia and are between the ages of 16 and 67. Eligible patients must meet the following requirements: they must have been randomized to study CLN-PXT3003-02 under placebo or dose 1 in the past and completed 15 months of double-blind treatment including all procedures required at Study Termination visit (V6), they must be able to provide informed consent specific to this extension study, female patients must agree to continue using an approved method of birth control throughout the duration of the study, and finally patients must have completed"

Answered by AI

Can elderly individuals participate in this research project?

"Eligibility requirements for this research indicate that potential participants must between 16 and 67 years old."

Answered by AI

Is this research the first to test this hypothesis?

"As of now, there are 2 ongoing trials for PXT3003 dose 1 in 10 metropolises and 8 countries. The first study happened in 2017 and completed its Phase 3 drug approval stage that year. This trial was sponsored by Premier Research Group plc, involved 187 patients, and is the earliest known study for PXT3003 dose 1 ."

Answered by AI

How many human test subjects are involved in this experiment?

"As of right now, this study is not looking for new patients to enroll. The original posting date was March 7th, 2017 and the most recent update was on March 9th, 2021. If you're interested in other studies, there are currently 25 trials involving charcot-marie-tooth disease, type ia that are actively recruiting participants as well as 2 PXT3003 dose 1 trials."

Answered by AI

Under what conditions has PXT3003 been linked with adverse health effects?

"PXT3003 has undergone Phase 3 trials, meaning that there is both some evidence of efficacy and multiple rounds of data supporting safety. Consequently, our team at Power rates the safety of PXT3003 as a 3."

Answered by AI

Could you tell me how many hospitals are participating in this clinical trial?

"There are 10 sites currently running this clinical trial. A few notable locations include University of Michigan Health System in Ann Arbor, Department of Neurology at Cedars-Sinai Medical Center in Los Angeles, and University of Kansas Medical Center in Kansas City."

Answered by AI

Who else is applying?

What state do they live in?
Texas
How old are they?
18 - 65
What site did they apply to?
Department of Neurology, Cedars-Sinai Medical Center
University of Kansas Medical Center
University Hospital of Quebec
What portion of applicants met pre-screening criteria?
Did not meet criteria
Met criteria
Recent research and studies
clinicaltrials.govStudy
Assessing Long Term Safety and Tolerability of PXT3003 in Patients With Charcot-Marie-Tooth Disease Type 1A
2017. "Assessing Long Term Safety and Tolerability of PXT3003 in Patients With Charcot-Marie-Tooth Disease Type 1A". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03023540.
~16 spots leftby Dec 2024
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