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NCT06138639RECRUITINGIRB Ready

A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)

Sponsored by Solid Biosciences Inc.

Quick Self-Assessment

See if you qualify and express your interest

Preparing your pre-screening questions...
๐Ÿ“Š
1 of 40 participants interested
3% interest

Express Your Interest Today

Interested in learning more? Complete the form below.

Secure & Confidential

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

Why Participate?

  • No-Cost Study Care

  • Advance Medical Science

    Contribute to groundbreaking research

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel (varies by study)

  • Help Others

    Benefit future patients

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3In-person assessment if eligible
  4. 4Begin participation

About This Study

This is a multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single intravenous (IV) infusion of SGT-003 in participants with Duchenne muscular dystrophy. There will be 5 cohorts in this study. Cohort 1 will include participants 4 to \< 7 years of age. Cohort 2 will include participants 7 to \< 12 years of age. Cohort 3 will include participants 0 to \< 4 years of age. Cohort 4 will include participants 12 to \< 18 years of age.

Who Can Participate

Inclusion Criteria

Cohort 1: 4 to \<7 years of age
Cohort 2: 7 to \<12 years of age
Cohort 3: 0 to \< 4 years of age
Cohort 4: 12 to \< 18 years of age
Cohort 5: 10 to \< 18 years of age
Participant ambulatory status at the time of Screening Part A or Rescreening, as defined by the ability to complete a 10-meter walk/run test in \< 30 seconds:
Cohorts 1, 2, and 4: Ambulatory
Cohort 3: Either ambulatory or non-ambulatory
Cohort 5: Non-ambulatory, but having been previously ambulatory by history
Established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype confirmed by Sponsor genetic testing. In cases where a genotype may be predictive of residual dystrophin production and/or a clear clinical diagnosis of DMD cannot be made (e.g., due to age), evaluation of dystrophin levels in baseline muscle biopsies may be required to determine eligibility under this criterion.
Negative for AAV antibodies.
Steroid regimen:

โ“ Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility during the screening process.

Study Details

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Study Type

INTERVENTIONAL

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Target Enrollment

40 participants

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Age Range

0 Years - 17 Years

โšง

Gender

MALE

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Sponsor

Solid Biosciences Inc.

Study Locations

Choose your preferred location or select flexible during enrollment

RECRUITING

Little Rock

Arkansas

Location available
View Little Rock location page
RECRUITING

Los Angeles

California

Location available
View Los Angeles location page
RECRUITING

Sacramento

California

Location available
View Sacramento location page
RECRUITING

San Diego

California

Location available
View San Diego location page
RECRUITING

Atlanta

Georgia

Location available
View Atlanta location page
RECRUITING

Chicago

Illinois

Location available
View Chicago location page
RECRUITING

St Louis

Missouri

Location available
View St Louis location page
RECRUITING

Columbus

Ohio

Location available
View Columbus location page
RECRUITING

Portland

Oregon

Location available
View Portland location page

๐Ÿ“ And 3 more locations available

Frequently Asked Questions

Q:What is a clinical trial?

A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.

Q:Will I be compensated?

Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.

Q:Will I receive a placebo instead of treatment?

When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.

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

Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.

Q:How long does a clinical trial last?

Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.

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

Ready to Make a Difference?

Express your interest in this groundbreaking clinical trial

๐Ÿ”’ Secure ยท ๐Ÿฅ Expert Care

Looking for Duchenne Muscular Dystrophy Treatment in Little Rock?

Join others in Arkansas exploring innovative treatment options through clinical research

Duchenne Muscular Dystrophy Treatment Options in Little Rock, Arkansas

If you're searching for Duchenne Muscular Dystrophy treatment in Little Rock, participating in a clinical research study may provide access to innovative approaches under expert medical supervision. This study is actively recruiting participants in Little Rock, Los Angeles, Sacramento and surrounding areas.

Clinical trials offer participants the opportunity to receive cutting-edge treatments while contributing to medical research that may help future patients with Duchenne Muscular Dystrophy. All study-related care is provided at no cost to participants.

Local Sites
3 locations in Arkansas
Now Enrolling
Up to 40 participants
Quick Start
Screening available now

Why Consider a Clinical Trial for Duchenne Muscular Dystrophy?

Potential Benefits

  • โœ“Access to new treatment approaches before public availability
  • โœ“Close monitoring by experienced medical professionals
  • โœ“Study-related care provided at no cost
  • โœ“Contribute to medical research for Duchenne Muscular Dystrophy

What to Expect

  • โ†’Initial screening to determine eligibility
  • โ†’Regular check-ups and monitoring visits
  • โ†’Possible compensation for time and travel
  • โ†’You can withdraw at any time

Frequently Asked Questions About This Duchenne Muscular Dystrophy Study

Important Clinical Trial Information

This information is provided for educational purposes and does not constitute medical advice. Clinical trial participation involves potential risks and benefits. Eligibility requirements apply and will be assessed during the screening process.

Study identifier: NCT06138639. For complete study details, visit ClinicalTrials.gov. Always consult with your healthcare provider before making decisions about your medical care or participating in clinical research.