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NMDA Receptor Antagonist

1.5 mg/kg for Rett Syndrome

Phase 2
Waitlist Available
Led By Jeffrey Neul, MD, PhD
Research Sponsored by Rett Syndrome Research Trust
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be younger than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up day 1, day 15
Awards & highlights
No Placebo-Only Group

Summary

This 2 cohort, sequential, ascending dose study will assess the safety, tolerability and efficacy of oral ketamine dosed in a single 5-day BID regimen in addition to placebo, in a 4-week cross-over design in patients with Rett Syndrome. Approximately 12 patients per cohort are anticipated to participate for approximately 8-10 weeks at approximately 7 US study centers.

Eligible Conditions
  • Rett Syndrome

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~day 1, day 15
This trial's timeline: 3 weeks for screening, Varies for treatment, and day 1, day 15 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Dose-Limiting Adverse Events
Secondary study objectives
Continuous biosensor data
Other study objectives
Children's Sleep Habits Questionnaire
Clinical Global Impression of Improvement
Clinician Domain Likert Scale
+6 more

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups
Experimental Treatment
Group I: 1.5 mg/kgExperimental Treatment1 Intervention
ketamine will be dosed orally twice daily for 5 days at 0.75 mg/kg, in addition to 5 days of placebo
Group II: 0.75 mg/kgExperimental Treatment1 Intervention
ketamine will be dosed orally twice daily for 5 days at 0.75 mg/kg, in addition to 5 days of placebo
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Ketamine
2011
Completed Phase 4
~1120

Find a Location

Who is running the clinical trial?

Rett Syndrome Research TrustLead Sponsor
7 Previous Clinical Trials
5,365 Total Patients Enrolled
7 Trials studying Rett Syndrome
5,365 Patients Enrolled for Rett Syndrome
Vanderbilt University Medical CenterOTHER
900 Previous Clinical Trials
939,483 Total Patients Enrolled
3 Trials studying Rett Syndrome
8,202 Patients Enrolled for Rett Syndrome
Jana von Hehn, PhDStudy DirectorRett Syndrome Research Trust
1 Previous Clinical Trials
5,000 Total Patients Enrolled
1 Trials studying Rett Syndrome
5,000 Patients Enrolled for Rett Syndrome
~3 spots leftby Nov 2025
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