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SGT-003

Clinical trials are investigating SGT-003 in people with Duchenne muscular dystrophy. These studies aim to assess safety, tolerability, and early signs of efficacy, including changes in microdystrophin protein levels. The current trial is a Phase 1/2 study in a target group of 60 participants.

Table of Contents

Trial overview

The available trial of SGT-003 is a study in people with Duchenne muscular dystrophy, which is a serious muscle disease that gets worse over time.[1] The study is titled “A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy” and is listed as authorised.[1]

This is an interventional study, which means researchers give the study treatment and then observe what happens.[1] The trial plans to enroll 60 participants.[1]

Study design and phase

The study is a Phase 1/2 trial.[1] This early phase usually means the main goal is to learn about safety first, while also looking for early signs that the treatment may help.[1]

Participants receive a single intravenous dose of SGT-003, meaning the treatment is given once through a vein.[1] The source data does not provide more details about the visit schedule or other study procedures.[1]

Who can participate

The target population is people with Duchenne muscular dystrophy.[1] The source data does not list age limits, disease stage rules, or other detailed entry criteria.[1]

Because the trial is in an early phase, participation is usually limited to a carefully selected group, but the exact rules are not provided in the source data.[1]

What is being measured

The main safety measure is the incidence of treatment-emergent adverse events through Day 360.[1] This means the researchers will count any unwanted health problems that start or worsen after treatment begins during the first year of follow-up.[1]

The main efficacy measure is the change from baseline of microdystrophin protein levels at Day 90.[1] “Baseline” means the starting point before treatment, and microdystrophin is the protein level the researchers are checking in muscle tissue.[1]

The brief summary says the study also looks at microdystrophin expression in muscle biopsies, which are small samples of muscle taken for testing.[1]

Why this trial matters

For people with Duchenne muscular dystrophy, early trials like this help researchers learn whether a new treatment can be studied safely in humans and whether it shows a biological effect.[1] In this study, the biological effect is linked to microdystrophin levels in muscle tissue.[1]

Because the trial is early and small, it is not meant to answer every question about long-term benefit.[1] Instead, it is designed to give first important data on safety and early response in the target group.[1]

Trial ID Phase Condition studied Status Enrollment
NCT06138639 Phase 1/2 Duchenne muscular dystrophy Authorised 60

FAQ

  • What is being studied in SGT-003 clinical trials? The trial is studying a single intravenous dose of SGT-003 in Duchenne muscular dystrophy. It is mainly looking at safety, tolerability, and whether microdystrophin levels change after treatment.
  • Who can take part in the SGT-003 trial? The trial is for people with Duchenne muscular dystrophy. The source data does not give more details about age or other entry rules.
  • What phase is the SGT-003 study? This is a Phase 1/2 trial. Phase 1/2 studies usually check safety first and also look for early signs that the treatment may work.
  • What results are the researchers measuring? Researchers are measuring treatment-emergent adverse events through Day 360 and the change from baseline in microdystrophin protein levels at Day 90. These are used to track safety and early biological effect.
  • How many people are planned for the SGT-003 trial? The trial plans to enroll 60 participants. This number helps the study team collect enough information to assess the treatment in this group.

Ongoing Clinical Trials on SGT-003

  • Study of SGT-003 Gene Therapy for Safety and Tolerability in Walking Males with Duchenne Muscular Dystrophy

    Recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    Italy

  • Phase 3 Randomized Double‑Blind Placebo‑Controlled Study of Intravenous sgt-003 in Ambulatory Male Patients with Duchenne Muscular Dystrophy

    Not yet recruiting

    1 1
    Investigated drugs:
    Belgium France Germany Italy The Netherlands Spain

Glossary

  • Duchenne muscular dystrophy: A serious muscle disease that causes muscles to get weaker over time.
  • Clinical trial: A research study in people that tests a treatment to learn more about its safety and whether it may help.
  • Interventional study: A study where participants receive a treatment or procedure that the researchers are testing.
  • Phase 1/2: An early study stage that looks at safety, tolerability, and early signs of benefit.
  • Intravenous: Given through a vein, usually by a needle or IV line.
  • Safety: How well a treatment can be used without causing serious problems.
  • Tolerability: How well people can handle a treatment and its effects.
  • Efficacy: How well a treatment works for the condition being studied.
  • Microdystrophin: A shortened form of the dystrophin protein that researchers measure in muscle tissue.
  • Muscle biopsy: A small sample of muscle taken for testing in the lab.
  • Treatment-emergent adverse events: Unwanted health problems that start or get worse after treatment begins.
  • Baseline: The starting point before treatment begins, used for comparison later.

References