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 6/14/18 

 

 Drug Trials  

Trial Name

click title for study details

Trial Location  Contact Info  
Evaluation of the Efficacy, Safety, and Tolerability of Sarizotan in Rett Syndrome With Respiratory Symptoms 

University of Alabama at Birmingham

jlane@uab.edu 1-205-996-4927

 

Altman Clinical & Translation Research Institute - Rady Children's Hospital/UCSD

kditslear@ucsd.edu  1-858-246-2288

 

Rush University Medical Center

susan_rohde@rush.edu  1-312-942-0079

 

Texas Children's Hospital

Mohammad.Naqvi@bcm.edu
 

Gillette Children's Specialty Healthcare

RKatoch@gillettechildrens.com  1-651-325-2331 

ANAVEX 2-73  Pre-qualifying online survey availableHERE

TBD TBD 

 

Observational & Non-Pharmaceutical Based Trials

 
Trial Name

Trial Location

Contact Information

Natural History Study

University of Alabama at Birmingham

 jlane@uab.edu  1-800-822-2472 x 7

 

Baylor College of Medicine

jobarris@texaschildrens.org 1-832-822-7388

 

Children's Hospital Boston

Grace.Bazin@childrens.harvard.edu  1-312-942-0079

 

Greenwood Genetics Center

fran@ggc.org  1-864-941-8100 

 

Children's Hospital of Philadelphia

GormanC@email.chop.edu  1-267-426-5171 

 

University of Rochester Medical Center

Laurie.Seltzer@urmc.rochester.edu  1-585-275-2808

 

Cleveland Clinic (*CDKL5 only)

 CARISTI@ccf.org  1-246-444-0173

 

Rady Children's Hospital/UCSD

kditslear@ucsd.edu  1-858-246-2288 

 

Vanderbilt University Medical Center

 nicole.i.thompson@vanderbilt.edu  1-615-343-4586

 

Rush University Medical Center

 susan_rohde@rush.edu  1-312-942-0079

 

UCSF Benioff Children's Hospital

 Erobertson@mail.cho.org  1-925-979-4055

 

Cincinnati Children's Hospital

Maxwell.mays@cchmc.org  1-513-803-7935 

 

Gillette Children's Specialty Healthcare

RKatoch@gillettechildrens.com  1-651-325-2331  

 

St. Louis Children's Hospital

rettresearch@neuro.wustl.edu  1-314-454-4267

 

University of Colorado

     Gina.VanderVeen@childrenscolorado.org  1-720-777-5514 

Biobanking of Rett Syndrome and Related Disorders

University of Alabama at Birmingham

  jlane@uab.edu  1-205-934-1130

 

Children's Hospital Boston

      Lindsay.Swanson@childrens.harvard.edu   1-617-355-8994

 

 Gillette Children's Specialty Healthcare

 1-651-325-2331  
 

Vanderbilt University Medical Center

 nicole.i.thompson@vanderbilt.edu  1-615-343-4586

 

Baylor College of Medicine

 jg20@bcm.edu  1-832-822-1781

 

Rush University Medical Center

susan_rohde@rush.edu  1-312-942-0079 

 

University of California, San Diego

 kditslear@ucsd.edu  1-858-246-2288

 

Neuro-Habilitation Studies

  

Visual Attention to Print in Children with Rett Syndrome

Appalachian State University

koppenhaverd@appstate.edu 
 

UNC Center for Literacy and Disability

 clds@unc.edu
Auditory Assesment of Language and Learning

 Vanderbilt University 

 dorita.jones@vanderbilt.edu  1-615-343-1961

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

 Georgetown University

psd23@georgetown.edu  1-240-381-1763 

Rett syndrome: Tailoring treatment based on communication needs

 University of Minnesota - Dept. of Educational Psychology

sima0034@unm.edu
 
Communication services and support for inidviduals with Rett syndrome: a survey for families

Maastricht University

               g.townend@maastrichtuniversity.nl                            Take the Survey

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

University of Minnesota

byier001@unm.edu   1-612-626-7892

 

Trial Results & Updates   

 

Treatment of Rett Syndrome with Recombinant Human IGF-1(Phase 2) Read Results   
Trofinetide Phase 3 Update from Neuren 2-5-18  Read the Update  

 

International Trials

 

Evaluation of the Efficacy, Safety, and Tolerability of Sarizotan in Rett Syndrome With Respiratory Symptoms  Murdoch, Western Australia Jenny Downs, PhD  61894897774  Jenny.Downs@telethonkids.org.au
  Kochi, Kerala, India Vinayan KP, MD    91 9447800303  vinayankp@aims.amrita.edu
  Chennai, Tamilnadu, India Sandhiya Thangaraj  917871881270 sandhiyadevi12345@gmail.com
  Kolkata, West Bengal, India Arijit Chattopadhyay, MD  91 9830133133   arijitchatto@hotmail.com
  Mumbai, India Vrajesh Udani, MD    +9819596661  vrajesh.udani@gmail.com 
  Mumbai, India Anaita Hegde, MD    +91 982 018-6155           docanaita@rediffmail.com
  New Delhi, India

Sheffali Gulati    +91 986 839 7532     sheffaligulati@gmail.com

  Siena, Tuscany, Italy Silvia Leoncini    +390577586534   s.leoncini74@gmail.com
  Milano, Italy  Miriam Nella Savini    390281844201   miriam.savini@yahoo.it
  London, United Kingdom

Asimina Tsirka  440207848069  asimina.tsirka@kcl.ac.uk      Efisia Saia 4402078480358    Efisia.Sais@nhs.net