Miami, FLNCT05898620Now EnrollingIRB Ready

Rett Syndrome Clinical Trial in Miami, FL

Access cutting-edge rett syndrome treatment through this clinical trial at a research site in Miami. Study-provided care at no cost to qualified participants.

Sponsored by Neurogene Inc.

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Expert Care in Miami

Access rett syndrome specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related rett syndrome treatment provided free

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Check if you qualify for this rett syndrome clinical trial in Miami, FL

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Your information is protected and will only be shared with the research team.

Why Participate?

  • No-Cost Study Care

  • Local to Miami

    Convenient for FL residents

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3Visit Miami site if eligible
  4. 4Begin participation

About This Rett Syndrome Study in Miami

This study will evaluate the safety profile of the investigational gene therapy, NGN-401, in females with typical Rett syndrome.

Sponsor: Neurogene Inc.

Who Can Participate

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

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in Miami?

Yes, this clinical trial (NCT05898620) has an active research site in Miami, FL that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

Q:Can I leave the trial if I change my mind?

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

Still have questions? Our study coordinators are here to help.

Rett Syndrome Treatment Options in Miami, FL

If you're searching for rett syndrome treatment options in Miami, FL, this clinical trial (NCT05898620) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our Miami research site is actively enrolling participants for this clinical trial. You'll receive care from experienced rett syndrome specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all rett syndrome clinical trials near you to find additional studies recruiting in your area.

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Secure · Expert Care · Miami, FL