Clinical Trials

Clinical trials and studies are important aspects of developing treatments for diseases. To learn more about clinical trials please visit the website.


Below is a list of all the current clinical trials for Rett Syndrome. The trials are listed in random order. Inclusion below does not imply endorsement on the part of RSRT.

A Study to Evaluate Ketamine for the Treatment of Rett Syndrome

Study of ANAVEX 2-73 in Patients with Rett Syndrome (USA)

ANAVEX2-73 Study in Patients With Rett Syndrome (Australia)

Efficacy and Safety of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome (ARCH)

Evaluation of the Efficacy, Safety, and Tolerability of Sarizotan in Rett Syndrome With Respiratory Symptoms

Open Label Trial of Triheptanoin in Treatment of Rett Syndrome 

Treatment of Mitochondrial Dysfunction in Rett Syndrome With Triheptanoin

A Pilot Study of an Antioxidant Cocktail vs. Placebo in the Treatment of Children and Adolescents With Rett Syndrome (Canada)

Biobanking of Rett Syndrome and Related Disorders

Development of a Behavioral Outcome Measure for Rett Syndrome (RettBe)

Natural History of Rett Syndrome & Related Disorders

Rare Disease Clinical Research Network: Neurophysiological Correlates

The Role of Probiotic PS128 in Movement Disorders

Angelman & Rett Syndrome: Interaction and Communication 

Patients With RETT Syndrome (France)




Study to Assess Safety and Efficacy of Fingolimod in Children With Rett Syndrome

Osteoporosis in Rett Syndrome

Treatment of Rett Syndrome With Recombinant Human IGF-1

Trial of Dextromethorphan in Rett Syndrome

Pharmacological Treatment of Rett Syndrome with Statins

A Safety Study of NNZ-2566 in Pediatric Rett Syndrome

Pharmacological Treatment of Rett Syndrome with Glatiramer Acetate (Copaxone)

An Open Label, Exploratory Study to Investigate the Treatment Effect of Glatiramer Acetate on Girls With Rett Syndrome

Phase 2 Study of EPI-743 in Rett Syndrome

A Safety Study of NNZ-2566 in Patients with Rett Syndrome

Pilot Study of the Effects of the Desipramine on the Neurovegetative Parameters of the Child With Rett Syndrome


Intervention Rationale Study Details Results
Folate, Betaine Dietary methyl supplementation Randomized double blinded placebocontrolled 73 individuals No clinical improvement although parents reported improvement in children under 5 years old
Folinic Acid Improve folate levels in CNS Randomized double blinded placebocontrolled crossover trial 8 individuals Increased 5-MTHF levels, no clinical change
Creatine DNA methylation Randomized double blinded placebocontrolled crossover trial 18 individuals in stage III or IV 11% increase in DNA methylation, no clinical change
Melatonin Improve sleep disturbances Open label 10 individuals Increased total sleep time and efficiency in subjects with worst baselines
L-carnitine Improve Carnitine deficiency Randomized double blinded placebocontrolled crossover trial 35 individuals Improvement in hand apraxia, some measures of well-being in subgroup
Naltrexone Improve respiratory function Randomized double blind placebocontrolled crossover trial 25 individuals Modification of some breathing issues, more rapid decline in 40% on drug
Lamotrigine Seizure control and dyspraxia Open pilot study 12 individuals Well tolerated, better seizure control in 2/3, better behavior in 4/12
Levetiracetam Seizure control Open label 8 individuals Reduced seizure frequency and improved quality of life

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