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||Complete||4.12.19 Newron Thank You|
|Anavex 2-73||Phase 2: Recruiting||ANAVEX 3.18.19|
|Australia Update 9.4.19|
|Anavex 2-73 Update 9.16.19|
|Ketamine||Phase 2: Recruiting|
|Triheptanoin||Phase 2: Recruiting|
|Cannabidiol Oral Solution
|Phase 3: Recruiting||Trial Criteria_9.6.19|
|GW Research May, 2019|
|Trofinetide||Phase 3: Recruiting||ACADIA Trofinetide Update 6.15.20|
|ACADIA Trofinetide Update 3.23.20|
|ACADIA Trofinetide Update 2.24.20|
|ACADIA Trofinetide Update 10.30.19|
|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||AMO-04 Update 6.13.18|
|AveXis – AVXS-201 Gene Therapy||8.22.19 Avexis Update|
*Updated June 2020. 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.
|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|