Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients
Public ClinicalTrials.gov record NCT04762758. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Multi-center, Randomized, Double-blind, Placebo Controlled Phase III Study to Assess the Efficacy, Safety, and Tolerability of PXT3003 in Charcot-Marie-Tooth Type 1A (CMT1A)
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
The study will consist of 2 periods: Double-blind Treatment and Open-Label Extension(OLE) Period. -Double-blind Treatment Period - This will be randomized, double-blind, placebo-controlled part of the study which will be conducted in parallel groups, ie,1 group receiving the active treatment (PXT3003) and the other group receiving placebo. Primary endpoint of the study will be assessed at Month 15. -Open-label Extension (OLE) Period - All subjects completing Double-blind Treatment Period will be given an opportunity to enter the OLE Period of the study and receive the active treatment (PXT3003). The duration of the OLE Period will be based on Sponsor discretion, ie, Sponsor intends to keep the study open until the study drug PXT3003 is commercially available. During this period, the long-term safety and efficacy of PXT3003 will be assessed as an exploratory objective. Double-blind Treatment Period Objectives: Primary: To evaluate the efficacy of treatment with PXT3003 (a fixed-dose combination of \[RS\]-baclofen, naltrexone hydrochloride \[HCl\], and D-sorbitol) compared to placebo in subjects with Charcot-Marie-Tooth disease type 1A (CMT1A). Secondary: To evaluate the safety and tolerability of PXT3003 treatment in subjects with CMT1A. Exploratory: To characterize the relationship between plasma biomarkers and response to PXT3003 treatment. OLE Period Objective: Exploratory: To evaluate the long-term safety and efficacy of PXT3003.
Study identification
- NCT ID
- NCT04762758
- Recruitment status
- Not listed
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 350 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 16 Years to 65 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Mar 29, 2021
- Primary completion
- Apr 18, 2024
- Completion
- Apr 18, 2024
- Last update posted
- Apr 2, 2024
2021 – 2024
United States locations
- U.S. sites
- 20
- U.S. states
- 14
- U.S. cities
- 19
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Cedars-Sinai Medical Center | Los Angeles | California | 90048 | — |
| UCLA Department of Psychiatry and Biobehavioral Sciences | Los Angeles | California | 90095 | — |
| UC Davis Health Department of Physical Medicine and Rehabilitation | Sacramento | California | 95817 | — |
| Hospital for Special Care | New Britain | Connecticut | 06053 | — |
| University of Florida Clinical Research Center | Gainesville | Florida | 32610 | — |
| University of Miami Leonard M. Miller School of Medicine | Miami | Florida | 33136 | — |
| Advent Health Medical Group Neurology Orlando | Orlando | Florida | 32803 | — |
| University of Kansas Medical Center Research Institute | Fairway | Kansas | 66205 | — |
| Massachusetts General Hospital Neuromuscular Diagnostic Center | Boston | Massachusetts | 02114 | — |
| University of Minnesota Health | Minneapolis | Minnesota | 55414 | — |
| MU Health Care Neurology and Sleep Disorders Clinic | Columbia | Missouri | 65212 | — |
| Hackensack Meridian Health Hackensack University Medical Center | Hackensack | New Jersey | 07601 | — |
| Colombia University Department of Neurology | New York | New York | 10032 | — |
| UNC Department of Neurology Peripheral Neuropathy Center | Chapel Hill | North Carolina | 27514 | — |
| Atrium Health Neurosciences Institute | Charlotte | North Carolina | 28207 | — |
| The Ohio State University Wexner Medical Center | Columbus | Ohio | 43210 | — |
| Oregon Neurology | Springfield | Oregon | 97477 | — |
| National Neuromuscular Research Institute | Austin | Texas | 78759 | — |
| Neurology Clinic at University of Washington Medical Center | Seattle | Washington | 98195 | — |
| Providence St. Luke's Rehabilitation Medical Center | Spokane | Washington | 99202 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 30 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04762758, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Apr 2, 2024 · Synced Sep 3, 2026
Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.
Open the official record
The complete protocol, eligibility criteria, and contact information for NCT04762758 live on ClinicalTrials.gov.