Popular Trials
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Trials With No Placebo
Behavioural Intervention
Virtual Therapy for Developmental Disabilities
This trial tests a virtual therapy program to help people with intellectual and developmental disorders better communicate and manage their behavior. Participants will do assessments and have weekly or biweekly virtual visits with a therapist.
Gene Therapy
Gene Therapy for Rett Syndrome
This trial tests NGN-401, a gene therapy using a virus to deliver a healthy gene, in female children with Rett syndrome. It aims to improve brain cell function by providing the correct version of a missing or faulty gene.
Gene Therapy
Gene Therapy (TSHA-102) for Rett Syndrome
This trial is testing a new gene therapy called TSHA-102 in adult women with Rett syndrome. The therapy aims to fix the genetic problems causing the disorder by adding healthy genes to their cells. Researchers will study its safety and effectiveness over several years. TSHA-102 is a gene therapy aimed at addressing the genetic deficiencies in Rett syndrome by adding healthy MECP2 genes to the cells.
Behavioral Intervention
Virtual Reality Gaming for Rett Syndrome
This trial uses special computer games with wristbands to help individuals with Rett syndrome move their arms and hands more independently. The wristbands track arm movements, allowing participants to control the games. The goal is to reduce repetitive hand movements and improve quality of life.
Behavioural Intervention
PIXI for Neurogenetic Disorders
This trial tests a program called PIXI, which helps parents of infants with genetic disorders. The program educates parents about their baby's condition, coaches them on how to interact with their baby, and provides family support. The goal is to improve the baby's development and family well-being.
Frequently Asked Questions
Introduction to rett syndrome
What are the top hospitals conducting rett syndrome research?
When it comes to advancing our understanding and treatment options for Rett Syndrome, several top hospitals are leading the way. In Aurora, Colorado, Children's Hospital stands out with its dedication to research in this rare neurological disorder. Although currently conducting just one active clinical trial for Rett Syndrome, they have made significant progress with a total of six trials since their first recorded study in 2019. Similarly, Boston Children's Hospital has been at the forefront of Rett Syndrome research since 2010 when they initiated their inaugural trial. With one ongoing trial and an impressive tally of ten previous studies, this institution remains committed to finding breakthroughs.
In Houston, Texas Children's Hospital has also played a crucial role in advancing knowledge about Rett Syndrome. Their efforts include seven completed trials alongside their current active trial dedicated to this condition - all stemming from their initial study recorded in 2019. Furthermore Georgetown University School of Medicine joined the pursuit more recently but nonetheless quickly established themselves as key contributors with their first-ever trial on record conducted just last year (2021).
North of the border in Toronto lies Holland Bloorview Kids Rehabilitation Hospital – an institute that focuses not only on providing care but also endeavors towards expanding scientific knowledge surrounding conditions such as Rett Syndrome.This Canadian hospital may possess only one active clinical trial along with another singular past endeavor; both traceable back to 2019 Yet despite these numbers being relatively small compared to others mentioned earlier—it must be remembered that every piece adds up towards uncovering answers which hold transformative potential for those affected by such diseases.
These hospitals exemplify how medical professionals around the world are joining forces and dedicating resources toward better understanding and treating Rett Syndrome—a complex neurodevelopmental disorder that primarily affects girls early in life.Studies like these contribute valuable insights into causes,symptoms,and management strategies while simultaneously offering hope for patients,families,and researchers alike.As we continue down this path,the collective efforts across borders and institutions bring us closer to unraveling the mysteries of Rett Syndrome and ultimately improving the lives of those living with this condition.
Which are the best cities for rett syndrome clinical trials?
Aurora, Colorado; Boston, Massachusetts; Houston, Texas; Washington, District of Columbia; and Toronto, Ontario are the top cities for rett syndrome clinical trials. Each city has one active trial dedicated to researching different aspects of this condition. From Cohort 1 studies in Aurora, Boston, and Houston to Modified Virtual Reality Gaming research in Washington and Rett T investigations in Toronto. These cities offer opportunities for individuals affected by rett syndrome to participate in cutting-edge clinical trials that could contribute to advancements in treatment and understanding of the disorder.
Which are the top treatments for rett syndrome being explored in clinical trials?
Promising treatment options for Rett syndrome are currently being explored in clinical trials. One such option is modified Virtual Reality Gaming, which has shown potential in one active trial specifically dedicated to rett syndrome. This innovative approach was first introduced and listed in 2021 with the aim of improving outcomes for individuals with Rett syndrome. Another notable contender is triheptanoin, a substance under investigation in one ongoing trial solely focused on this rare disorder. First listed in 2016, triheptanoin holds promise as a potential therapeutic intervention for Rett syndrome patients. As research progresses, these treatments offer hope for improved quality of life and management strategies within the realm of Rett syndrome care.
What are the most recent clinical trials for rett syndrome?
Exciting progress is being made in the realm of rett syndrome research, with recent clinical trials offering hope for improved treatments. Notably, Cohort 1 has shown promise in both Phase 1 and Phase 2 studies, presenting potential avenues for therapeutic intervention. Additionally, Rett T and triheptanoin have been investigated in Phase 2 trials as potential treatment options. These efforts demonstrate a commitment to finding effective strategies to address the challenges faced by individuals living with rett syndrome. While further investigation is needed, these advancements provide optimism for enhanced care and quality of life for those affected by this disorder.
What rett syndrome clinical trials were recently completed?
In the realm of Rett syndrome research, several significant clinical trials have recently reached completion, bringing new hope to patients and their families. In September 2021, ACADIA Pharmaceuticals Inc.'s trial investigating Trofinetide wrapped up, adding valuable insights into its potential as a treatment option. Another trial sponsored by the same company concluded in November 2020 with promising results for trofinetide. Additionally, Anavex Life Sciences Corp.'s study on ANAVEX2-73 oral liquid finished in July 2020, showcasing another avenue of exploration in tackling this complex disorder. These recent advancements underscore the dedication of researchers towards finding effective therapies for Rett syndrome and improving lives affected by it.