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rett-syndrome Clinical Trials

A listing of rett-syndrome medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.

Found 14 clinical trials

Esketamine for the Treatment of Rett Syndrome

The goal of this interventional study is to learn about the efficacy and safety of Esketamine for treating children with Rett syndrome (RTT). The main questions it aims to answer are: whether Esketamine treatment is effective in improving symptom severity for RTT. whether Esketamine is safe in the treatment of …

5 - 10 years of age Female Phase N/A

Measuring Impact of Computer Gaming on Arm Use in Rett Syndrome

Using a tele-research approach, we will recruit, enroll, guide and support carers and participants to engage in computer based activities (modified virtual reality) with the primary outcome of reducing stereotypies and increasing independent arm and hand use and secondary outcome of improving quality of living. Because of our virtual approach, …

4 - 60 years of age Both Phase N/A

A Novel, Regulated Gene Therapy (NGN-401) Study for Female Children With Rett Syndrome

This study will evaluate the safety profile of the investigational gene therapy, NGN-401, in female children with typical Rett syndrome.

4 - 10 years of age Female Phase 1/2
S Samantha Dreyer

Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study)

The REVEAL Pediatric Study is a multi-center, Phase 1/2 open-label, dose-escalation and dose-expansion study of TSHA-102, an investigational gene therapy, in pediatric females with Rett Syndrome. The safety, tolerability, and preliminary efficacy of two dose levels will be evaluated. The study duration is up to 6 years.

5 - 8 years of age Female Phase 1

Safety and Efficacy of TSHA-102 in Adult Females With Rett Syndrome (REVEAL Adult Study)

The REVEAL Adult Study is a multi-center, Phase 1/2 open-label, dose-escalation study of TSHA-102, an investigational gene therapy, in adult females with Rett syndrome. The safety, tolerability, and preliminary efficacy of two dose levels will be evaluated. The study duration is estimated to be up to 63 months.

12 - 100 years of age Female Phase 1/2
J Jana von Hehn, PhD

The Rett Syndrome Global Registry

The Rett Global Registry is a fully remote, global, caregiver-reported registry to collect information about caring for a loved one with Rett syndrome. In addition, caregivers have the ability to track and graph their loved one's symptoms and care strategies over time, store information for central access, and opt-in to …

- 100 years of age Both Phase N/A

Rett Syndrome Registry

The Rett Syndrome Registry is a longitudinal observational study of individuals with MECP2 mutations and a diagnosis of Rett syndrome. Designed together with the IRSF Rett Syndrome Center of Excellence Network medical directors, this study collects data on the signs and symptoms of Rett syndrome as reported by the Rett …

- 99 years of age Both Phase N/A
L Laura CG de Graaff, MD, PhD

GROWing Up With Rare GENEtic Syndromes

Introduction Rare complex syndromes Patients with complex genetic syndromes, by definition, have combined medical problems affecting multiple organ systems, and intellectual disability is often part of the syndrome. During childhood, patients with rare genetic syndromes receive multidisciplinary and specialized medical care; they usually receive medical care from 3-4 medical specialists. …

18 - 100 years of age Both Phase N/A
U US GSK Clinical Trials Call Center

A Study to Evaluate the Safety, Effectiveness and Impact of the GlaxoSmithKline Biologicals' Malaria Vaccine- RTS, S/AS01E in Young Children in Sub-Saharan Africa

The RTS, S/AS01E vaccine has been developed for routine immunization of children living in malaria- endemic countries of sub-Saharan Africa. This study is intended as a post-implementation safety study (after vaccine implementation), with the primary objective to evaluate the safety of vaccine after its administration. In addition to the primary …

- 5 years of age Both Phase N/A
V Viola Doccini

Biomarkers in Rett Syndrome

Rett syndrome (RTT) is an X-linked genetic disorder that causes severe neurological development disorder. In its classic form, it seems to affect almost exclusively females with an incidence of up to one in 10,000 females. Patients affected by Rett Syndrome can present a wide range of symptoms, in different combinations …

- 18 years of age Female Phase N/A

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