A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome

Launched by NEUROGENE INC. · Jun 1, 2023

Keywords

ClinConnect Summary

This clinical trial is studying a new gene therapy called NGN-401, which is designed for female children diagnosed with Rett syndrome, a rare genetic disorder that affects brain development and can cause a variety of challenges, including problems with movement and communication. The main goal of the study is to find out if NGN-401 is safe for these children. The trial is currently looking for participants aged between 4 and 10 years old who have a specific mutation in the MECP2 gene, which is known to cause Rett syndrome. To be eligible, participants must also have had a stable treatment plan with anti-epileptic drugs for at least 12 weeks, and they should live within a two-hour drive of the study center.

If a child is accepted into the trial, they will receive the gene therapy and will be monitored closely by the research team for safety. It's important to note that participants should not have normal hand function or significant other health issues that could interfere with the study. Additionally, they should not have had other serious neurological conditions. The study may also allow children who have previously taken another treatment, trofinetide, to participate if they stopped due to side effects or lack of effectiveness. For families considering this trial, it’s an opportunity to help advance research on Rett syndrome while contributing to a better understanding of new treatment options.

Gender

FEMALE

Eligibility criteria

• Inclusion Criteria:
• • Diagnosis of typical Rett syndrome with a documented disease-causing mutation in the methyl-CpG-binding protein 2 (MECP2) gene
• • Current anti-epileptic drug regimen has been stable for at least 12 weeks
• • Participant and caregiver should reside within a 2-hour drive of the study center for at least 3 months following treatment
• • Participant must have never taken trofinetide or have taken trofinetide and discontinued due to lack of tolerability, lack of efficacy, or other reasons. Following NGN-401 dosing, trofinetide may be initiated after a specified time period and with the support of the treating clinician
• Exclusion Criteria:
• • Normal or near normal hand function
• • Has a current clinically significant condition other than Rett syndrome
• • Presence of a concomitant medical condition that precludes intracerebroventricular administration, or use of anesthetics or immune suppression needed for study related procedures
• • Grossly abnormal psychomotor development in the first 6 months of life
• • A history of other genetic disorders or neurological conditions, such as stroke, brain tumor, or autoimmune processes affecting the central nervous system
• • Other inclusion and exclusion criteria apply.