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Gene Therapy

Gene Therapy for Rett Syndrome

Phase 1 & 2
Recruiting
Research Sponsored by Neurogene Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Diagnosis of typical Rett syndrome with a documented disease-causing mutation in the methyl-CpG-binding protein 2 (MECP2) gene
Be younger than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 5 years
Awards & highlights

Study Summary

This trial is testing a new gene therapy for Rett Syndrome in girls to see if it's safe and effective. #RettSyndrome #GeneTherapy

Who is the study for?
This trial is for female children with typical Rett syndrome who have a specific mutation in the MECP2 gene. They should be on a stable anti-epileptic drug regimen for 12 weeks and live close to the study center. Those with normal hand function, other significant conditions, or certain neurological disorders are excluded.Check my eligibility
What is being tested?
The trial is testing NGN-401, an investigational gene therapy designed specifically for Rett syndrome. It aims to evaluate how safe this treatment is when given to young girls suffering from this condition.See study design
What are the potential side effects?
While not explicitly listed here, potential side effects may include reactions related to gene therapy such as immune responses, issues at injection site, and general discomforts like headaches or fever due to changes within the body.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I have Rett syndrome with a confirmed MECP2 gene mutation.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and 5 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Incidence of Adverse Events of Special Interest (AESIs)
Incidence of Serious Adverse Events (SAEs)
Incidence of Treatment Emergent Adverse Events (TEAEs)
+2 more

Trial Design

2Treatment groups
Experimental Treatment
Group I: Low DoseExperimental Treatment1 Intervention
Dose Level 1
Group II: High DoseExperimental Treatment1 Intervention
Dose Level 2

Find a Location

Who is running the clinical trial?

Neurogene Inc.Lead Sponsor
5 Previous Clinical Trials
124 Total Patients Enrolled
Julie Jordan, MDStudy DirectorNeurogene Inc.
Albena Patroneva, MD, MBAStudy DirectorNeurogene Inc.

Media Library

NGN-401 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT05898620 — Phase 1 & 2
Rett Syndrome Research Study Groups: Low Dose, High Dose
Rett Syndrome Clinical Trial 2023: NGN-401 Highlights & Side Effects. Trial Name: NCT05898620 — Phase 1 & 2
NGN-401 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05898620 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Does this investigation include participants who are fifty or older?

"The requisites for this clinical trial stipulate that eligible applicants should be aged 4 to 10 years old. There are 380 trials suitable for children up to 18 and 1004 studies available for those above the age of 65."

Answered by AI

Does this research currently require volunteer participants?

"This study, which was first posted on June 1st 2023 and subsequently edited the same day, is not actively recruiting patients. However, according to clinicaltrials.gov there are 1433 other trials that may be of interest to potential participants at this time."

Answered by AI

Is there an opportunity for me to get involved in this experiment?

"Applicants to this trial must have been diagnosed with rett syndrome, and they must fall into the age range of 4-10 years old. A limited number of 5 participants are accepted in total."

Answered by AI

Who else is applying?

What site did they apply to?
Boston Children's Hospital
What portion of applicants met pre-screening criteria?
Met criteria
Recent research and studies
clinicaltrials.govStudy
A Novel, Regulated Gene Therapy (NGN-401) Study for Female Children With Rett Syndrome
2023. "A Novel, Regulated Gene Therapy (NGN-401) Study for Female Children With Rett Syndrome". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05898620.
All > Rett Syndrome
~11 spots leftby Oct 2029
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