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Gene therapy trial aims to stop duchenne in its tracks

NCT ID NCT06138639

Disease control Sponsor: Solid Biosciences Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 26, 2026 · Last updated Jul 09, 2026 · Updated 3 times

Summary

This study tests a single dose of SGT-003, a gene therapy that delivers a working version of the dystrophin gene to muscle cells. About 60 boys with Duchenne muscular dystrophy, aged from infancy to under 18, will receive the treatment and be followed for 5 years. The goal is to see if it is safe and can restore enough dystrophin to improve muscle strength and function.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
SGT-003 (a gene therapy using a modified virus to deliver a shortened dystrophin gene)
What this could lead to
If successful, this could provide a one-time treatment that slows or stops muscle decline in Duchenne muscular dystrophy, potentially improving quality of life and mobility.
What could go wrong
This is an early-phase trial with only 60 participants, so results may not apply to all. Gene therapies can cause immune reactions or liver issues, and the long-term effects are unknown.
Why investors are watching

Solid Biosciences is a very small company, so this early-stage trial of its one-time gene therapy SGT-003 for Duchenne muscular dystrophy is its main value driver. The trial tests safety and whether a single infusion helps boys with the disease, with results from the youngest age groups expected first. For a micro-cap, this readout will largely determine how investors view the company's future.

If it works: If the therapy shows a good safety profile and signs that it helps muscle function, the company could advance to later-stage testing and attract partnership interest. A positive result would validate its main asset and support the company's continued operation.

If it fails: Gene therapies often fail in early trials, and safety problems or weak efficacy data could force the company to halt development. A delay in enrollment or a poor readout could sharply reduce the company's prospects, since it has few other products in the pipeline.

AI-written from the trial record. Speculative, and not investment advice.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 60 people

The number the study aims to enrol. It can still change while the study runs.

Started

May 2024

Expected to finish

May 2031

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

0 to 17 years

Sex

Male participants only

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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: * Cohorts 1, 2, 4, and 5: A stable daily oral steroid regimen of at least 0.5 mg/kg/day of prednisone or 0.75 mg/kg/day of deflazacort for ≥12 weeks prior to Screening Part A or Rescreening, allowing for weight-based modifications consistent with clinical practice. * Cohort 3: N/A * Meet 10-meter walk/run time criteria * Meet time to rise from supine criteria * Cohort 5: Meet Performance of Upper Limb (PUL) 2.0 criteria * Participant has body weight: ≤ 90 kg Exclusion Criteria: * Treatment with dystrophin modifying drugs within 3 months prior to screening. * Current or prior treatment with an approved or investigational gene transfer drug. * Exposure to certain approved or investigational drugs within 3 months prior to screening or 5 half-lives since last administration, whichever is longer. * Established clinical diagnosis of DMD that is associated with any deletion mutation invariant or variant predicted to not express exons 1 to 11 or, exons 42 to 45, or exons 57 to 69, inclusive, in the DMD gene as documented by a genetic report and confirmed by Sponsor genetic testing. Other inclusion or exclusion criteria apply.

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Conditions

The condition(s) this trial relates to.

Duchenne muscular dystrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Duchenne muscular dystrophy

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    15 sites in 4 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    RECRUITING

    Chicago, Illinois, 60611-2605, United States

  • Arkansas Children's Hospital

    RECRUITING

    Little Rock, Arkansas, 72202, United States

  • Children's Hospital of Philadelphia

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • Children's Hospital of the King's Daughters

    RECRUITING

    Norfolk, Virginia, 23510, United States

  • Fondazione Policlinico Universitario Agostino Gemelli IRCCS

    RECRUITING

    Rome, 00168, Italy

  • Great Ormond Street Hospital

    RECRUITING

    London, WC1N 3JH, United Kingdom

  • Nationwide Children's Hospital

    RECRUITING

    Columbus, Ohio, 43215, United States

  • Oregon Health and Sciences University

    RECRUITING

    Portland, Oregon, 97239, United States

  • Rare Disease Research

    RECRUITING

    Atlanta, Georgia, 30329, United States

    Contact Email: •••••@•••••

  • Seattle Children's Hospital

    RECRUITING

    Seattle, Washington, 98105, United States

  • The Hospital for Sick Children

    RECRUITING

    Toronto, Ontario, M5G 0A4, Canada

  • University of California

    RECRUITING

    San Diego, California, 92037, United States

  • University of California, Davis

    RECRUITING

    Sacramento, California, 95817, United States

  • University of California, Los Angeles Medical Center

    RECRUITING

    Los Angeles, California, 90095, United States

  • Washington University in St. Louis

    RECRUITING

    St Louis, Missouri, 63110, United States

    Contact Email: •••••@•••••

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