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A Pilot Phase 2 Study with Long-Term Extension to Assess the Safety and Efficacy of Sirolimus in Patients with Leigh Syndrome Caused by Genetically-Confirmed Mitochondrial Respiratory Chain Deficiency

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Trial Profile

A Pilot Phase 2 Study with Long-Term Extension to Assess the Safety and Efficacy of Sirolimus in Patients with Leigh Syndrome Caused by Genetically-Confirmed Mitochondrial Respiratory Chain Deficiency

Status: Recruiting
Phase of Trial: Phase II

Latest Information Update: 10 Mar 2025

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At a glance

  • Drugs Sirolimus (Primary)
  • Indications Leigh disease
  • Focus Adverse reactions

Most Recent Events

  • 05 Mar 2025 Status changed from not yet recruiting to recruiting.
  • 04 Mar 2025 New trial record

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