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A phase I study to evaluate the safety, tolerability, and pharmacokinetics of RTX-117 for Charcot-Marie-Tooth disease (CMT)

Trial Profile

A phase I study to evaluate the safety, tolerability, and pharmacokinetics of RTX-117 for Charcot-Marie-Tooth disease (CMT)

Status: Recruiting
Phase of Trial: Phase I

Latest Information Update: 09 Mar 2026

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At a glance

  • Drugs RTX 117 (Primary)
  • Indications Charcot-Marie-Tooth disease; Leukoencephalopathies
  • Focus Adverse reactions; First in man

Most Recent Events

  • 02 Mar 2026 According to ReviR Therapeutics media release, first participant has been dosed in the First-in-Human (FIH) Phase 1 clinical trial of its lead candidate, RTX-117. This clinical progress follows the recent clearance by the Center for Drug Evaluation (CDE) of China's National Medical Products Administration (NMPA) of Investigational New Drug (IND) applications for two rare neurological indications: Charcot-Marie-Tooth disease (CMT) and Vanishing White Matter disease (VWM).
  • 02 Mar 2026 Status changed from planning to recruiting.
  • 13 Nov 2025 New trial record

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