Find your next paid study
12 recruiting studies matching your filters
Promoting Prosocial Behavior in Syndromic Intellectual and Developmental Disabilities
The purpose of this study is to evaluate the effectiveness of an adapted, telehealth functional behavioral therapy (FBTsIDD) specifically focused on promoting …
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 addi…
Auditory EEG and Behavioral Assessments in Individuals With Rett Syndrome
This observational research study is designed to learn more about how the brain processes sound in individuals with Rett syndrome. Participants will complete a…
Assessing the Effectiveness of Customized Computer Activities on Reducing Stereotypies and Improving Independent Upper Extremity Motor Skills in Persons with 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 rea…
Rett Syndrome Real World Data Observational Registry
The Rett Syndrome Registry is a longitudinal observational study of individuals with MECP2 mutations and a diagnosis of Rett syndrome. Designed together with t…
Characterization of Translatable Neurophysiological Biomarkers to Enhance Therapeutic Development in Rett Syndrome
The goal of this observational study is to identify candidate biomarkers in individuals with Rett Syndrome (RTT). The main questions it aims to answer are: * …
Cognitive Function in Rett Syndrome During Trofinetide Treatment
Assessing cognitive functions among individuals with severe intellectual and developmental disabilities (IDD), including RTT, is often challenging due to floor…
An Open-label Phase 1/2/3 Study Consisting of a Phase 1/2 Safety and Dose-escalation and Phase 3 Dose-expansion Study to Evaluate Safety and Efficacy of a Single Intrathecal Administration of TSHA-102, an AAV9-Delivered Gene Therapy in Females With Rett Syndrome
The primary objectives of this study are to evaluate the safety of a single intrathecal (IT) dose of TSHA-102 in females with typical Rett syndrome, to select …
Functional and Neurodevelopmental Outcomes Following Intensive Multimodal Neurorehabilitation in Pediatric Patients With Neurodevelopmental Disorders, Including Chromosomal Abnormalities
This observational study evaluates functional and developmental outcomes in pediatric participants undergoing a two week intensive multimodal neurorehabilitati…
Validation of Innovative Biosensors for Rett Autonomic Symptom Tracking
The VIBRANT study aims to validate biosensors to objectively and directly measure symptoms in Rett syndrome and create confidence in the use of these devices i…
A Baseline-Controlled, Open-Label, Multicenter, Single-Arm, Pivotal Study to Evaluate the Efficacy, Safety, and Tolerability of NGN-401 in Subjects With Rett Syndrome (Embolden)
This study will evaluate the efficacy and safety profiles of the investigational gene therapy, NGN-401, in females with typical Rett syndrome.
A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Phase 3 Study of Bionetide for the Treatment of Girls and Women With Rett Syndrome
Primary Objective • To investigate the efficacy of treatment with oral Bionetide versus placebo in girls and women with Rett syndrome Key Secondary Objective …