Recruiting Rett Syndrome Studies in Boston
To Investigate the Efficacy of Treatment With Oral NA-921 (Bionetide) Versus Placebo in Females 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 • To investigate the efficacy of treatme...
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 and Efficacy of TSHA-102 in Adolescent and 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 p...
A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome
This study will evaluate the safety profile of the investigational gene therapy, NGN-401, in females with typical Rett syndrome....
About Rett Syndrome Clinical Trials in Boston
Rett syndrome is a rare genetic neurological disorder that primarily affects girls, causing a regression in development after initially normal growth. It is caused by mutations in the MECP2 gene. Treatment is supportive, with gene therapies under investigation.
There are currently 4 rett syndrome clinical trials recruiting participants in Boston, MASSACHUSETTS. These studies are seeking a combined 262 participants. Research is being sponsored by Biomed Industries, Inc., Taysha Gene Therapies, Inc., Neurogene Inc.. Clinical trial participation is free and participants receive study-related medical care at no cost.
Rett Syndrome Clinical Trials in Boston — FAQ
Are there rett syndrome clinical trials in Boston?
Yes, there are 4 rett syndrome clinical trials currently recruiting in Boston, MASSACHUSETTS. Browse the studies on this page to find one that fits.
How do I join a clinical trial in Boston?
Browse the studies listed above, click one that interests you, and complete the free eligibility form. A study coordinator at the Boston research site will contact you about next steps.
Are clinical trials in Boston free?
Yes, clinical trial participation is always free. Study-related treatment, medical tests, and doctor visits are provided at no cost. Many Boston studies also compensate for your time and travel.
What rett syndrome treatments are being tested?
The 4 active trials in Boston are testing new therapies including novel drugs, biologics, and treatment approaches for rett syndrome.
Data updated March 2, 2026 from ClinicalTrials.gov