Rett syndrome drug pipeline

Compounds in development

No compounds populated for this indication yet — see registered trials below.

Trials recruiting now (8)

8 drug-interventional rett syndrome trials recruiting now, of 46 registered interventional trials. Browse all on ClinicalTrials.gov →

• Phase 3To Investigate the Efficacy of Treatment With Oral NA-921 (Bionetide) Versus Placebo in Females With Rett Syndrome

  Bionetide, Placebo

  Biomed Industries, Inc.n=210Australia, United States · 18 sitesStarted Sep 2026NCT06840496
• Phase 3A Phase 3 Study of Fenfluramine Hydrochloride in Rett Syndrome

  fenfluramine hydrochloride

  UCB BIOSCIENCES, Inc.n=200Hungary, Japan +1 · 5 sitesStarted Jun 2026NCT07503444
• Phase 2/3Efficacy and Safety of NTI164 in Children and Young Adults With Rett Syndrome

  NTI164, Placebo

  Fenix Innovation Groupn=40Australia · 1 siteStarted Jul 2026NCT07257978
• Phase 2Lamivudine as a Novel Clinical Effort for Rett Syndrome

  Lamivudine

  Maria Denise Fernandes Carvalho de Andraden=10Brazil · 1 siteStarted Nov 2025NCT07640503
• Phase 2A Pilot Study of an Antioxidant Cocktail vs. Placebo in the Treatment of Children and Adolescents With Rett Syndrome

  Rett-T

  Holland Bloorview Kids Rehabilitation Hospitaln=35Canada · 1 siteStarted Oct 2025NCT04041713
• Phase 2Repurposing Mirtazapine in Rett Syndrome

  mirtazapine

  University of Triesten=54Italy · 4 sitesStarted Jul 2025NCT07430046
• Phase 1/2ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS)

  ION440

  Ionis Pharmaceuticals, Inc.n=48Austria, France +2 · 11 sitesStarted Oct 2024NCT06430385
• Phase 1Rett REVOLUTION Trial: An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Rett Syndrome

  Vorinostat (SAHA), Placebo

  Unravel Biosciences, Inc.n=15Colombia · 1 siteStarted Mar 2026NCT07150013