Rettsyndrome.org is dedicated to aggressively pursuing treatment and a cure for those living with Rett syndrome until there is a world without Rett.
There are a variety of trials available and in development. We encourage you to work with your care team to determine which trial is best for your child. The following are U.S. clinical trials for Rett syndrome. This list does not imply development or endorsement by Rettsyndrome.org. For more information, or to enroll in a clinical trial, visit ClinicalTrials.gov.
|Sarizotan||Phase 2/3: Active||4.12.19 Newron Thank You|
|Anavex 2-73||Phase 2: Recruiting||ANAVEX 3.18.19|
|Australia Update 9.4.19|
|Ketamine||Phase 2: Recruiting|
|Triheptanoin||Phase 2: Not Yet Recruiting|
|Cannabidiol Oral Solution
|Phase 3: Recruiting||Trial Criteria_9.6.19|
|GW Research May, 2019|
|Trofinetide||Phase 3: Not Yet Enrolling||ACADIA Trofinetide Update 7.31.19|
|ACADIA Trofinetide Update 5.1.19|
|ACADIA Trofinetide Update 3.27.19|
|ACADIA Trofinetide Update 2.3.19|
|ACADIA Trofinetide Update 12.20.18|
|AMO-04||FDA Approved Orphan Drug Status||AMO-04 Update 6.13.18|
|AveXis – AVXS-201 Gene Therapy||8.22.19 Avexis Update|
*Updated August 2019 For the latest information on clinical trials visit ClinicalTrials.gov.
For more information about the clinical trial process, download our Clinical Trial Guide For Participants.
- Auditory Assesment of Language and Learning
- Improving Upper Extremity Motor Skills in Persons with Rett Syndrome Using Virtual Reality Computer Games
- Rett syndrome: Tailoring treatment based on communication needs
- Communication services and support for individuals with Rett syndrome: a survey for families
- Assessing Oculomotor Control in Rett Syndrome Using Integrated EEG and Eye Tracking Technology
|Study Name||Study Focus||Status|
|Natural History Study for Rett Syndrome, MECP2 Duplication and Rett-related Disorders||gathering data to increase knowledge of RTT and develop more efficient clinical trial designs.||Enrolling|
|Biobanking of Rett Syndrome and Related Disorders||Collecting DNA, RNA, Plasma and cell lines to evaluate factors contributing to disease status, clinical severity, or responsiveness to therapeutic intervention.||Enrolling|