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NCT02579759 · ClinicalTrials.gov registry record · Phase 3

Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients (PLEO-CMT)

A Phase 3 study, sponsored by Pharnext S.C.A..

Completed
Registry status
Phase 3
Development phase
323
Enrollment target

NCT02579759: Completed Phase 3 study, sponsored by Pharnext S.C.A..

NCT02579759 is a Phase 3 study that has completed, run by Pharnext S.C.A.. The registered enrollment target is 323 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (58% lower). According to ClinicalTrials.gov, the official US trial registry.

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The verdict

NCT02579759, a Phase 3 study, has completed, sponsored by Pharnext S.C.A..

COMPLETED
Registry status
Phase 3
Development phase
323 participants
Enrollment target

Study Summary

The purpose of this study is to determine whether PXT3003 is effective and safe in the treatment of Charcot-Marie-Tooth disease - Type 1 A (CMT1A). This double-blind study will assess in parallel groups 2 doses of PXT3003 compared to Placebo in CMT1A patients treated for 15 months.

Primary Outcome

The primary efficacy variable used in the main analysis is the mean of the available ONLS values at month 12 and month 15. The ONLS is a disability scale that was derived and improved from the Overall Disability Sum Score (ODSS) to measure limitations in the everyday activities of the upper limbs (rated on 5 points) and the lower limbs (rated on 7 points). The total score is a 12-point scale: 0 (no disability) to 12 (maximum disability). Lower values in the ONLS indicate a better clinical condi

Interventions

  • DRUG placebo
  • DRUG PXT3003 dose 1
  • DRUG PXT3003 dose 2

Trial Details

FieldValue
Enrollment Target 323 participants
Start Date 2015-12
Est. Completion 2018-08
Phase Phase 3
Pharnext S.C.A.

2 total trials

What the finished NCT02579759 record still lists

NCT02579759 is an interventional study that assigns participants to a tested intervention. The registered 323 participants enrollment target is mid-sized for trials with a published cap, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (58% lower).

The record links to 0 conditions, and to 3 interventions - of which placebo is the first listed.

NCT02579759 does not publish any study locations in the registry export this page uses.

Frequently Asked Questions

What is clinical trial NCT02579759 about?

NCT02579759 is a clinical study titled "Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients (PLEO-CMT)". The purpose of this study is to determine whether PXT3003 is effective and safe in the treatment of Charcot-Marie-Tooth disease - Type 1 A (CMT1A). This double-blind study will assess in parallel groups 2 doses of PXT3003 compared to Placebo in CMT1A patients treated for 15 months.

What is the current status of trial NCT02579759?

This trial is currently completed. It is a Phase 3 study. The enrollment target is 323 participants. The study started on 2015-12. Estimated completion is 2018-08.

What interventions are being tested in trial NCT02579759?

The interventions under investigation include: placebo (DRUG), PXT3003 dose 1 (DRUG), PXT3003 dose 2 (DRUG).

Who is sponsoring clinical trial NCT02579759?

This trial is sponsored by Pharnext S.C.A., which has 2 total clinical trials registered on ClinicalTrials.gov.

How this trial's enrollment target compares

Where NCT02579759's enrollment target sits among peer trials

323 1081st of 2000 higher than 920 of 2,000 other Phase 3 trials

participants (enrollment target), bucketed by value

Each bar is a band; taller bars hold more other Phase 3 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.

Source ClinicalTrials.gov registry export · 2026-08-08

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Source: ClinicalTrials.gov NCT02579759, the US trial registry maintained by the National Library of Medicine. NCT02579759 (mid enrollment · none site footprint · completed) retrieved and formatted by PlainTrial, see methodology.