Rettsyndrome.org is dedicated to aggressively pursuing treatment and a cure for those living with Rett syndrome until there is a world without Rett.

Drug Trials

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.

Rett Syndrome Clinical TrialsDownload the list of active Rett Syndrome clinical trials & studies.

Intervention Name Status Updates
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
(GWP42003-P; CBD-OS)
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
AMO 04QA
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.

Neuro-Habilitative Studies

Observational Studies

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