Gainesville, FLNCT05693142Now EnrollingIRB Ready

Duchenne Muscular Dystrophy Clinical Trial in Gainesville, FL

Access cutting-edge duchenne muscular dystrophy treatment through this clinical trial at a research site in Gainesville. Study-provided care at no cost to qualified participants.

Sponsored by REGENXBIO Inc.

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Expert Care in Gainesville

Access duchenne muscular dystrophy specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related duchenne muscular dystrophy treatment provided free

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Check if you qualify for this duchenne muscular dystrophy clinical trial in Gainesville, FL

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Why Participate?

  • No-Cost Study Care

  • Local to Gainesville

    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 Gainesville site if eligible
  4. 4Begin participation

About This Duchenne Muscular Dystrophy Study in Gainesville

RGX-202 is a gene therapy designed to deliver a transgene for a novel microdystrophin that includes functional elements of naturally-occurring dystrophin including the C-Terminal (CT) domain. This is a multicenter, open-label dose evaluation clinical study to assess the safety, tolerability, and clinical efficacy of a one-time intravenous (IV) dose of RGX-202 in participants with Duchenne. For additional information on how to participate (or be considered for the study), please follow this lin

Sponsor: REGENXBIO Inc.

Who Can Participate

Inclusion Criteria

The participant's legal guardian(s) is (are) willing and able to provide written, signed informed consent prior to any study-related procedures; and, where applicable, the minor participant has provided written or verbal assent according to local requirements.
Is a male at least 4 years of age and less than 12 years of age at consent or 1 to \<4 years of age at the time of dosing and ≥ 10 kg at the time of screening.
Must meet any of the following criteria:
DMD gene mutation in exons 18 and above, and a clinical picture consistent with typical DMD with the exception of a participant (Cohort 1b) with DMD gene mutation in exons 12-17.
Participant is able to walk 100 meters independently without assistive devices. Cohort 2c participant must be able to walk 10 meters independently without assistive devices. Cohort 1b participant must be able to walk with or without assistive devices.
Participant is able to complete the TTSTAND per protoc

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 Gainesville?

Yes, this clinical trial (NCT05693142) has an active research site in Gainesville, 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.

Duchenne Muscular Dystrophy Treatment Options in Gainesville, FL

If you're searching for duchenne muscular dystrophy treatment options in Gainesville, FL, this clinical trial (NCT05693142) 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 Gainesville research site is actively enrolling participants for this clinical trial. You'll receive care from experienced duchenne muscular dystrophy 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 duchenne muscular dystrophy clinical trials near you to find additional studies recruiting in your area.

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