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.

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

Treatment of Mitochondrial Dysfunction in Rett Syndrome With Triheptanoin

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

Biobanking of Rett Syndrome and Related Disorders

Osteoporosis in Rett Syndrome

The Findings of MR Imaging 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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