Mode
Home
Research
Clinical Trials Drug Pipeline Weekly Digests
Reference
Conditions Medications
Community
Questions
Text Size
Log in / Sign up
Home › Conditions › Rett syndrome

Rett syndrome

Part of X-Linked Intellectual Disability

4 published articles · Updated continuously

Clinical Trial Landscape

Clinical Trials for Rett syndrome

8 trials tracked for Rett syndrome: 2 in phase 3 or 4 and 2 with published results. The most-cited published study has 190 citations.

8Trials tracked
2Phase 3 & 4
0Recruiting
2With published results
Phase distribution
Phase 3 2 Phase 2 5 Phase 1 1
  1. Phase 3 Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDER™) Completed · 190 cited
  2. Phase 3 Open-Label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome Completed · 32 cited
  3. Phase 2 Treatment of Rett Syndrome With Recombinant Human IGF-1 Completed
  4. Phase 2 Pharmacological Treatment of Rett Syndrome With Statins Completed
  5. Phase 2 Pharmacological Treatment of Rett Syndrome With Glatiramer Acetate (Copaxone) Completed
  6. Phase 2 A Study to Evaluate Ketamine for the Treatment of Rett Syndrome Completed
Show 2 more trials
  1. Phase 2 Placebo Controlled Trial of Dextromethorphan in Rett Syndrome Completed
  2. Phase 1 Treatment of Rett Syndrome With rhIGF-1 (Mecasermin [rDNA]Injection) Completed

Showing the 8 most-cited and recently-updated of 8 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.

What the trials found For clinicians

Rett syndrome: what the trials found

Trofinetide has demonstrated measurable impacts on behavioral and communication outcomes in patients with Rett syndrome. In a Phase 3 trial, Trofinetide was associated with significant improvements in the Rett Syndrome Behaviour Questionnaire (RSBQ) Total Score at week 12 1. Additionally, it showed statistically significant improvements in the Clinical Global Impression-Improvement (CGI-I) score and the Communication and Symbolic Behavior Scales Developmental Profile Infant-Toddler Checklist - Social Composite Score 1.

Longer-term data for Trofinetide indicates continued efficacy in behavioral scoring. In a Phase 3 study, patients treated with Trofinetide showed changes in RSBQ Total Scores at week 40, alongside consistent improvements in CGI-I scores compared to baseline 2.

Recent results — preliminary, needs further review

  • Ketamine (Phase 2, N=23) - Not yet corroborated.
  • Lovastatin (Phase 2, N=20) - Not yet corroborated.
  • Glatiramer Acetate (Phase 2, N=10) - Not yet corroborated.
  • Dextromethorphan (Phase 2, N=57) - Not yet corroborated.
  • rhIGF-1 (Phase 1 & 2 trials) - Not yet corroborated.

For the clinician treating this condition

  • Trofinetide is associated with statistically significant improvements in both behavioral scores (RSBQ) and clinical global impression (CGI-I) within a 12-week period.
  • Trofinetide has shown measurable impact on communication and social behavior scales in pediatric populations.
  • Evidence suggests that the benefits of Trofinetide for behavioral scoring persist through at least week 40 of treatment.

AI synthesis of 2 cited trials, updated Jun 29, 2026. Informational only — not medical advice; trial data sourced from ClinicalTrials.gov. How we use AI.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
Patient Mode — summaries use plain language, avoiding clinical jargon.
Review notes gut mycobiota dysbiosis in autism, ADHD, and Rett syndrome
Infectious Disease Sys. Review
Review notes gut mycobiota dysbiosis in autism, ADHD, and Rett syndrome Fungi in the gut may drive autism and ADHD symptoms
This narrative review synthesizes emerging evidence on gut mycobiota in autism spectrum disorder, attention-deficit/hyperactivity disorder, …
New research suggests gut fungi, not just bacteria, may play a key role in autism and ADHD.
Frontiers May 7, 2026
Review finds intranasal NGF improves symptoms in Rett syndrome mouse model
Neurology Sys. Review
Review finds intranasal NGF improves symptoms in Rett syndrome mouse model New nerve growth factor treatment could fix Rett syndrome symptoms
This review of preclinical studies in Mecp2-mutant mice reports that intranasal administration of NGF-like molecules improved behavioral sym…
A new nerve growth factor treatment might finally offer hope for Rett syndrome by fixing brain energy and calming inflammation without side …
Frontiers Apr 25, 2026
Spanish RSBQ Validation in 51 Latin American Rett Syndrome Caregivers
Neurology Phase II
Spanish RSBQ Validation in 51 Latin American Rett Syndrome Caregivers New Spanish Tool for Rett Syndrome Improves Care for Thousands
This observational linguistic validation study assessed a Spanish translation of the Rett Syndrome Behaviour Questionnaire among 51 Spanish-…
A Spanish-language behavior checklist for Rett Syndrome now matches the English version, helping Spanish-speaking families get more accurate…
medRxiv Apr 24, 2026
Trofinetide treatment shows symptom improvement in Rett syndrome observational study
Neurology Phase III
Trofinetide treatment shows symptom improvement in Rett syndrome observational study Real-World Data Shows Rett Drug Helps Most Patients
This 12-month observational study of 55 children with Rett or MECP2-related disorders found 75.9% experienced some symptom improvement with …
Real-life use of this medication shows strong results with fewer side effects than expected.
Frontiers Apr 23, 2026

Questions about Rett syndrome

Does trofinetide treatment show symptom improvement for children with Rett syndrome?

Trofinetide shows symptom improvement in Rett syndrome, with real-world studies showing about 76% of patients experiencing benefits in communication and motor skills.

Full answer →