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Rett Syndrome clinical trials

View clinical trials related to Rett Syndrome.

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NCT ID: NCT05012475 Recruiting - Rett Syndrome Clinical Trials

Measuring Impact of Computer Gaming on Arm Use in Rett Syndrome

Start date: May 1, 2021
Phase: N/A
Study type: Interventional

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, we are able to recruit from multiple countries and all states and territories of the USA.

NCT ID: NCT04988867 Completed - Rett Syndrome Clinical Trials

An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett Syndrome

DAFFODILâ„¢
Start date: September 22, 2021
Phase: Phase 2/Phase 3
Study type: Interventional

To investigate the safety and tolerability of long-term treatment with oral trofinetide in girls with Rett syndrome

NCT ID: NCT04920110 Completed - Rett Syndrome Clinical Trials

Development of the ORCA Communication Measure for Rett Syndrome

Rett-ORCA
Start date: July 2, 2021
Phase:
Study type: Observational

This measurement validation study will use qualitative and quantitative methodology to evaluate the Observer-Reported Communication Ability Measure (ORCA), to appropriately capture communication abilities in individuals with Rett syndrome. The ORCA Measure is a caregiver-reported questionnaire that collects caregiver observations of their child's communication abilities including expressive, receptive and pragmatic communication types. Caregivers will participate via phone interviews and online surveys. Approximately 270 participants will be enrolled.

NCT ID: NCT04900493 Recruiting - Rett Syndrome Clinical Trials

The Rett Syndrome Global Registry

Start date: January 31, 2022
Phase:
Study type: Observational [Patient 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 complete medical record consolidation and summary. Qualified researchers and therapeutic developers may request access to de-identified aggregate information to further Rett research, or assist with clinical development planning to facilitate and expedite more effective clinical trials.

NCT ID: NCT04776746 Completed - Rett Syndrome Clinical Trials

Open-Label Extension Study of Trofinetide for Rett Syndrome

Start date: November 8, 2020
Phase: Phase 3
Study type: Interventional

To investigate the safety and tolerability of continued long-term treatment with oral trofinetide in girls and women with Rett syndrome

NCT ID: NCT04514549 Completed - Rett Syndrome Clinical Trials

ASSESSING EMERALD AND MC10 BIOSTAMP nPOINT BIOSENSORS FOR RETT SYNDROME

Start date: November 23, 2020
Phase:
Study type: Observational

This is a pilot study of the Emerald device in Rett syndrome patients diagnosed with a confirmed MECP2 mutation. MC10 BioStamp nPoint patches will also be assessed with the goal to develop Rett-specific breathing algorithms

NCT ID: NCT04463316 Recruiting - Clinical trials for Prader-Willi Syndrome

GROWing Up With Rare GENEtic Syndromes

GROW UR GENES
Start date: October 1, 2018
Phase:
Study type: Observational

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. Increased life expectancy Although many genetic syndromes used to cause premature death, improvement of medical care has improved life expectancy. More and more patients are now reaching adult age, and the complexity of the syndrome persists into adulthood. However, until recently, multidisciplinary care was not available for adults with rare genetic syndromes. Ideally, active and well-coordinated health management is provided to prevent, detect, and treat comorbidities that are part of the syndrome. However, after transition from pediatric to adult medical care, patients and their parents often report fragmented poor quality care instead of adequate and integrated health management. Therefore, pediatricians express the urgent need for adequate, multidisciplinary adult follow up of their pediatric patients with rare genetic syndromes. Medical guidelines for adults not exist and the literature on health problems in these adults is scarce. Although there is a clear explanation for the absence of adult guidelines (i.e. the fact that in the past patients with rare genetic syndromes often died before reaching adult age), there is an urgent need for an overview of medical issues at adult age, for 'best practice' and, if possible, for medical guidelines. The aim of this study is to get an overview of medical needs of adults with rare genetic syndromes, including: 1. comorbidities 2. medical and their impact on quality of life 3. medication use 4. the need for adaption of medication dose according to each syndrome Methods and Results This is a retrospective file study. Analysis will be performed using SPSS version 23 and R version 3.6.0.

NCT ID: NCT04304482 Completed - Rett Syndrome Clinical Trials

ANAVEX2-73 Study in Pediatric Patients With Rett Syndrome

EXCELLENCE
Start date: July 1, 2020
Phase: Phase 2/Phase 3
Study type: Interventional

ANAVEX2-73-RS-003 is a Phase 2/3, double-blind, randomized, placebo-controlled dose escalation safety, tolerability and efficacy study in patients 5-17 years of age with RTT using endpoints including multiple clinical and exploratory molecular and biochemical measures.

NCT ID: NCT04279314 Completed - Rett Syndrome Clinical Trials

Open-Label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome

LILACâ„¢
Start date: January 29, 2020
Phase: Phase 3
Study type: Interventional

To investigate the safety and tolerability of long-term treatment with oral trofinetide in girls and women with Rett syndrome

NCT ID: NCT04252586 Terminated - Rett Syndrome Clinical Trials

A Long-term Safety Study of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome

Start date: February 28, 2020
Phase: Phase 3
Study type: Interventional

This study will be conducted to evaluate the long-term safety of cannabidiol oral solution (GWP42003-P, CBD-OS) in participants with Rett syndrome.