What side effects are associated with this type of intervention?

Common treatments for Rett Syndrome include symptomatic treatments such as antiepileptic drugs, medications for gastrointestinal issues, and physical therapy. These treatments can have side effects like drowsiness, gastrointestinal discomfort, and fatigue. Gene therapy, such as the investigational NGN-401, aims to address the underlying genetic cause of Rett Syndrome. Potential side effects of gene therapy can include immune reactions, insertional mutagenesis, and off-target effects, although specific data on NGN-401's side effects are still being evaluated.

What prior FDA approvals exist?

As of now, there are no FDA-approved gene therapies specifically for the treatment of Rett Syndrome. Traditional treatments have focused on managing symptoms and improving quality of life, including medications for seizures, muscle stiffness, and breathing irregularities. NGN-401 represents a novel approach by targeting the genetic root of the disorder, which is a significant step forward compared to existing symptomatic treatments.

What other types of research exist?

As of now, there are no specific alternatives to NGN-401 mentioned in the provided context. However, patients and caregivers should consult with their healthcare providers to explore other emerging gene therapies or advanced pharmacological treatments that may be in development or clinical trials for Rett Syndrome.

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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

Must not have

Presence of a concomitant medical condition that precludes intracerebroventricular administration, or use of anesthetics needed for study related procedures

Grossly abnormal psychomotor development in the first 6 months of life

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 5 years

Awards & highlights

No Placebo-Only Group

Summary

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.

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.

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.

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

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I have Rett syndrome with a confirmed MECP2 gene mutation.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I do not have any health conditions that prevent me from receiving treatments directly into my brain or using anesthesia.

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My child had significant developmental delays in the first 6 months.

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My hands function normally or almost normally.

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I have a history of genetic disorders or neurological conditions.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 5 years

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

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Incidence of Adverse Events of Special Interest (AESIs)

Incidence of Serious Adverse Events (SAEs)

Incidence of Treatment Emergent Adverse Events (TEAEs)

+2 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups

Experimental Treatment

Group I: Low DoseExperimental Treatment1 Intervention

Dose Level 1

Group II: High DoseExperimental Treatment1 Intervention

Dose Level 2

0 / 5Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Gene therapy, such as NGN-401, aims to address the underlying genetic cause of Rett Syndrome by introducing a functional copy of the MECP2 gene, which is mutated in most patients. This therapy works by delivering the correct gene sequence to the patient's cells, potentially restoring normal function and alleviating symptoms. This approach is significant for Rett Syndrome patients because it targets the root cause of the disorder, offering the possibility of more effective and long-lasting improvements compared to symptomatic treatments.