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Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients

NCT04762758 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 350

Last updated 2024-04-03

No results posted yet for this 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

  • Charcot-Marie-Tooth Disease

Interventions

DRUG

(RS)-baclofen, naltrexone hydrochloride and D-sorbitol

oral fixed dose combination

DRUG

Placebo

liquid oral solution

Sponsors & Collaborators

  • Worldwide Clinical Trials

    collaborator OTHER

  • Pharnext S.C.A.

    lead OTHER

Principal Investigators

  • Sharam Attarian, MD · CHU la Timone, Marseille , France

  • Mario Saporta, MD · University of Miami Miller School of Medicine, USA

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Model
PARALLEL

Eligibility

Min Age
16 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-03-30
Primary Completion
2024-04-19
Completion
2024-04-19
FDA Drug
Yes

Countries

  • United States
  • Belgium
  • Canada
  • Denmark
  • France
  • Germany
  • Israel
  • Italy
  • Spain

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04762758 on ClinicalTrials.gov