
Clinical Trials
Clinical trials and studies are important aspects of developing treatments for diseases. To learn more about clinical trials please visit the clinicaltrials.gov website.
CURRENT TRIALS and STUDIES
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)
Open Label Trial of Triheptanoin in Treatment of Rett Syndrome
Treatment of Mitochondrial Dysfunction in Rett Syndrome With Triheptanoin
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)
RECENTLY FINISHED TRIALS
Study to Assess Safety and Efficacy of Fingolimod in Children With 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)
Phase 2 Study of EPI-743 in Rett Syndrome
PAST CLINICAL TRIALS AND STUDIES
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 |