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10 Rett Syndrome Trials

Power is an online platform that helps thousands of Rett Syndrome patients discover FDA-reviewed trials every day. Every trial we feature meets safety and ethical standards, giving patients an easy way to discover promising new treatments in the research stage.

Primary Objective • To investigate the efficacy of treatment with oral Bionetide versus placebo in girls and women with Rett syndrome Key Secondary Objective • To investigate the efficacy of treatment with oral Bionetide versus placebo on ability to communicate in girls and women with Rett syndrome
Pivotal Trial
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:5 - 20
Sex:Female
210 Participants Needed
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.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:5 - 8
Sex:Female
20 Participants Needed
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.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:4 - 10
Sex:Female
16 Participants Needed
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.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:12+
Sex:Female
18 Participants Needed
The purpose of this study is to evaluate the effectiveness of an adapted, telehealth functional behavioral therapy (FBTsIDD) specifically focused on promoting appropriate communication and behavioral strategies in individuals with syndromic intellectual and developmental disorders. Participants will be asked to complete virtual study assessments at intake and then on a monthly basis for the duration of 3-6 months. In addition, participants will attend weekly or biweekly virtual intervention visits with a study therapist.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Unphased
Age:2 - 12
Sex:All
92 Participants Needed
This study will examine the potential efficacy and safety of Rett-T for core motor deficits of Rett syndrome, and will explore biological markers of safety and treatment response.
Trial Details
Trial Status:Not Yet Recruiting
Trial Phase:Phase 2
Age:2 - 21
Sex:Female
35 Participants Needed
Image of trial facility.

Virtual Reality Gaming for Rett Syndrome

Washington, District of Columbia
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.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Unphased
Age:4 - 60
Sex:All
10 Participants Needed
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PIXI for Neurogenetic Disorders

Research Triangle Park, North Carolina
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.
No Placebo Group
Trial Details
Trial Status:Enrolling By Invitation
Trial Phase:Unphased
Age:< 99
Sex:All
120 Participants Needed
Image of trial facility.

ION440 for MECP2 Syndrome

Saint Paul, Minnesota
The primary purpose of this study is to evaluate the safety and tolerability of ION440.
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:2 - 65
Sex:Male
48 Participants Needed
The objective of this study is to determine the effects of a 6-month, home-based personalized transcranial direct current stimulation (tDCS) intervention targeting the left dorsolateral prefrontal cortex on cognitive function, dual task standing and walking, and other metrics of mobility in older adults with motoric cognitive risk syndrome (MCR).
No Placebo Group
Trial Details
Trial Status:Not Yet Recruiting
Trial Phase:Unphased
Age:65 - 90
Sex:All
128 Participants Needed
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Frequently Asked Questions

How much do Rett Syndrome clinical trials pay?

Each trial will compensate patients a different amount, but $50-100 for each visit is a fairly common range for Phase 2–4 trials (Phase 1 trials often pay substantially more). Further, most trials will cover the costs of a travel to-and-from the clinic.

How do Rett Syndrome clinical trials work?

After a researcher reviews your profile, they may choose to invite you in to a screening appointment, where they'll determine if you meet 100% of the eligibility requirements. If you do, you'll be sorted into one of the treatment groups, and receive your study drug. For some trials, there is a chance you'll receive a placebo. Across Rett Syndrome trials 30% of clinical trials have a placebo. Typically, you'll be required to check-in with the clinic every month or so. The average trial length for Rett Syndrome is 12 months.

How do I participate in a study as a "healthy volunteer"?

Not all studies recruit healthy volunteers: usually, Phase 1 studies do. Participating as a healthy volunteer means you will go to a research facility several times over a few days or weeks to receive a dose of either the test treatment or a "placebo," which is a harmless substance that helps researchers compare results. You will have routine tests during these visits, and you'll be compensated for your time and travel, with the number of appointments and details varying by study.

What does the "phase" of a clinical trial mean?

The phase of a trial reveals what stage the drug is in to get approval for a specific condition. Phase 1 trials are the trials to collect safety data in humans. Phase 2 trials are those where the drug has some data showing safety in humans, but where further human data is needed on drug effectiveness. Phase 3 trials are in the final step before approval. The drug already has data showing both safety and effectiveness. As a general rule, Phase 3 trials are more promising than Phase 2, and Phase 2 trials are more promising than phase 1.

Do I need to be insured to participate in a Rett Syndrome medical study ?

Clinical trials are almost always free to participants, and so do not require insurance. The only exception here are trials focused on cancer, because only a small part of the typical treatment plan is actually experimental. For these cancer trials, participants typically need insurance to cover all the non-experimental components.

What are the newest Rett Syndrome clinical trials ?

Most recently, we added Bionetide for Rett Syndrome, Antioxidant Cocktail for Rett Syndrome and Transcranial Direct Current Stimulation for Dementia to the Power online platform.