Clinical Trials for Rett syndrome

A Participant's Guide to Drug Research in Rett Syndrome

"Does participating in a clinical trial prevent my daughter from participating in other future clinical trials?"

Detailed information on the Natural History Study


Drug Trials|Observational & Non- Pharmaceurtical Trials |Neuro-Habilitation Studies|Trial Results & Updates|International Trials


Updated 4/4/19 


Download the Available Rett Syndrome Clinical Trials & Studies List


 Drug Trials  

Trial Name

click title for study details

Trial Location  Contact Info  
Efficacy and Safety of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome (ARCH) University of Alabama
Rady Children's Hospital
Children's Hospital Colorado
Rush University Medical Center
Kennedy Krieger Institute/Johns Hopkins Medicine
Boston Children's Hospital/Harvard Medical School
Gillette Children's Specialty Healthcare
St. Louis Children's Hospital/Washington Univ. School of Medicine
Montefiore Medical Center/Albert Einstein College of Medicine
Cinncinnati Children's Hospital/Univ. Cincinnati
Children's Hospital of Philadelphia (CHoP)/Univ. Pennsylvania
Greenwood Genetic Center
Vanderbilt University Medical Center
Texas Children's Hospital/Baylor College of Medicine

GW Research May 2019 Update

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

4.12.19 Newron Thank You

University of Alabama at Birmingham 1-205-996-4927


Altman Clinical & Translation Research Institute - Rady Children's Hospital/UCSD  1-858-246-2288


Rush University Medical Center  1-312-942-0079


Texas Children's Hospital

Gillette Children's Specialty Healthcare  1-651-325-2331 

ANAVEX 2-73  Pre-qualifying online survey availableHERE

See  Anavex 2-73 Update: 3.28.19

Trofinetide Phase 3

TBD ACADIA Trofinetide Update 5.1.19
Trofinetide Phase 2 Published Results 3.27.19
ACADIA Trofinetide Update 2.3.19
ACADIA Trofinetide Update 12.20.18


Observational & Non-Pharmaceutical Based Trials

Trial Name

Trial Location

Contact Information

Natural History Study

University of Alabama at Birmingham  1-800-822-2472 x 7


Baylor College of Medicine 1-832-822-7388


Children's Hospital Boston  1-312-942-0079


Greenwood Genetics Center  1-864-941-8100 


Children's Hospital of Philadelphia  1-267-426-5171 


University of Rochester Medical Center  1-585-275-2808


Cleveland Clinic (*CDKL5 only)  1-246-444-0173


Rady Children's Hospital/UCSD  1-858-246-2288 


Vanderbilt University Medical Center  1-615-343-4586


Rush University Medical Center  1-312-942-0079


UCSF Benioff Children's Hospital  1-925-979-4055


Cincinnati Children's Hospital  1-513-803-7935 


Gillette Children's Specialty Healthcare  1-651-325-2331  


St. Louis Children's Hospital  1-314-454-4267


University of Colorado  1-720-777-5514 

Biobanking of Rett Syndrome and Related Disorders

University of Alabama at Birmingham  1-205-934-1130


Children's Hospital Boston   1-617-355-8994


 Gillette Children's Specialty Healthcare


Vanderbilt University Medical Center  1-615-343-4586


Baylor College of Medicine  1-832-822-1781


Rush University Medical Center  1-312-942-0079 


University of California, San Diego  1-858-246-2288


Neuro-Habilitation Studies


Visual Attention to Print in Children with Rett Syndrome

Appalachian State University 

UNC Center for Literacy and Disability
Auditory Assesment of Language and Learning

 Vanderbilt University  1-615-343-1961

Improving Upper Extremity Motor Skills in Persons with Rett Syndrome Using Virtual Reality Computer Games

 Georgetown University  1-240-381-1763 

Rett syndrome: Tailoring treatment based on communication needs

 University of Minnesota - Dept. of Educational Psychology
Communication services and support for inidviduals with Rett syndrome: a survey for families

Maastricht University

                                 Take the Survey

Assessing Oculomotor Control in Rett Syndrome Using Integrated EEG and Eye Tracking Technology

University of Minnesota   1-612-626-7892


Trial Results & Updates   


Treatment of Rett Syndrome with Recombinant Human IGF-1(Phase 2) Read Results   


International Trials


Evaluation of the Efficacy, Safety, and Tolerability of Sarizotan in Rett Syndrome With Respiratory Symptoms  Murdoch, Western Australia Jenny Downs, PhD  61894897774
  Kochi, Kerala, India Vinayan KP, MD    91 9447800303
  Chennai, Tamilnadu, India Sandhiya Thangaraj  917871881270
  Kolkata, West Bengal, India Arijit Chattopadhyay, MD  91 9830133133
  Mumbai, India Vrajesh Udani, MD    +9819596661 
  Mumbai, India Anaita Hegde, MD    +91 982 018-6155 
  New Delhi, India

Sheffali Gulati    +91 986 839 7532

  Siena, Tuscany, Italy Silvia Leoncini    +390577586534
  Milano, Italy  Miriam Nella Savini    390281844201
  London, United Kingdom

Asimina Tsirka  440207848069      Efisia Saia 4402078480358