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NCT04762758 · ClinicalTrials.gov registry record · Phase 3
Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients
A Phase 3 study of Charcot Marie Tooth Disease, sponsored by Pharnext S.C.A..
- Active
- Registry status
- Phase 3
- Development phase
- 350
- Enrollment target
- 20
- Study locations
NCT04762758: Active Phase 3 study of Charcot Marie Tooth Disease, sponsored by Pharnext S.C.A..
NCT04762758 is a Phase 3 study of Charcot Marie Tooth Disease that is active but no longer recruiting, run by Pharnext S.C.A.. The registered enrollment target is 350 participants, below the 2,315-participant average among 7 other Charcot Marie Tooth Disease trials with a reported enrollment target (85% lower). The trial reports 20 study locations across 14 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04762758, a Phase 3 study of Charcot Marie Tooth Disease, is active but no longer recruiting, sponsored by Pharnext S.C.A..
- ACTIVE NOT RECRUITING
- Registry status
- Phase 3
- Development phase
- 350 participants
- Enrollment target
- 20
- Study locations
Study Summary
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.
Conditions Studied
Interventions
- DRUG Placebo
- DRUG (RS)-baclofen, naltrexone hydrochloride and D-sorbitol
Study Locations (20)
California
- Cedars-Sinai Medical Center - Los Angeles
- UCLA Department of Psychiatry and Biobehavioral Sciences - Los Angeles
- UC Davis Health Department of Physical Medicine and Rehabilitation - Sacramento
Florida
- University of Florida Clinical Research Center - Gainesville
- University of Miami Leonard M. Miller School of Medicine - Miami
- Advent Health Medical Group Neurology Orlando - Orlando
North Carolina
- UNC Department of Neurology Peripheral Neuropathy Center - Chapel Hill
- Atrium Health Neurosciences Institute - Charlotte
Washington
- Neurology Clinic at University of Washington Medical Center - Seattle
- Providence St. Luke's Rehabilitation Medical Center - Spokane
Connecticut
- Hospital for Special Care - New Britain
Kansas
- University of Kansas Medical Center Research Institute - Fairway
Massachusetts
- Massachusetts General Hospital Neuromuscular Diagnostic Center - Boston
Minnesota
- University of Minnesota Health - Minneapolis
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 350 participants |
| Start Date | 2021-03-30 |
| Est. Completion | 2024-04-19 |
| Phase | Phase 3 |
What the registry record for NCT04762758 still lists
NCT04762758 is an interventional study that assigns participants to a tested intervention. The registered 350 participants enrollment target is mid-sized for trials with a published cap, below the 2,315-participant average among 7 other Charcot Marie Tooth Disease trials with a reported enrollment target (85% lower).
The record links to 1 condition, with Charcot Marie Tooth Disease appearing as the primary indexed condition, and to 2 interventions - of which Placebo is the first listed.
NCT04762758 names 20 study sites across 14 states, led by California, Florida, North Carolina.
Frequently Asked Questions
What is clinical trial NCT04762758 about?
NCT04762758 is a clinical study titled "Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients". 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 (PXT30...
What is the current status of trial NCT04762758?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 350 participants. The study started on 2021-03-30. Estimated completion is 2024-04-19.
What conditions does trial NCT04762758 study?
This clinical trial studies the following conditions: Charcot Marie Tooth Disease.
What interventions are being tested in trial NCT04762758?
The interventions under investigation include: Placebo (DRUG), (RS)-baclofen, naltrexone hydrochloride and D-sorbitol (DRUG).
Who is sponsoring clinical trial NCT04762758?
This trial is sponsored by Pharnext S.C.A., which has 2 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04762758 being conducted?
This trial has 20 study locations across California, Connecticut, Florida, Kansas, Massachusetts. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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