Comparing 5 approaches for duchenne muscular dystrophy
Official title: A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
A Phase 1/2, Multicenter, Open-Label Study to Investigate the Safety, Tolerability, and Efficacy of a Single Intravenous Dose of SGT-003 in Males With Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
- Phase 1
- 5 groups
- One site, in Toronto
- Recruiting
Interventions
- Genetic therapy
SGT-003
Adeno-associated virus serotype SLB101 containing the human microdystrophin gene (h-µD5)
Canadian Sites (1)
1 of 1 recruiting
- Recruiting
The Hospital for Sick Children
Toronto, Ontario
Eligibility Criteria
See who this study is looking for23 criteria
The trial’s own eligibility text, word for word from the registry. Only the trial site can say who takes part.
Inclusion
- +CohortCohortA group of participants sharing a characteristic, followed together.Read more → 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 ScreeningScreeningThe checks done before joining, to see whether a study fits.Read more → 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 SponsorSponsorThe organisation responsible for the study overall.Read more → 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 baselineBaselineYour starting measurements, taken before treatment begins.Read more → muscle biopsies may be required to determine eligibilityEligibility criteriaThe full list of requirements for taking part in a study.Read more → 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
- −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 SponsorSponsorThe organisation responsible for the study overall.Read more → genetic testing.
- −Treatment with dystrophin modifying drugs within 3 months prior to screeningScreeningThe checks done before joining, to see whether a study fits.Read more →.
- −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.
- −Other inclusionInclusion criteriaThe things you must have or be for a study to consider you.Read more → or exclusion criteriaExclusion criteriaThe things that would prevent someone from taking part.Read more → apply.
In plain language
Assembled directly from this trial’s registry record. Every sentence traces to a field below — nothing here is generated or interpreted.
What this study is
This study is testing a treatment for a condition.
Phase 1Phase 1The earliest stage of human testing, in a small group, focused on safety.Read more →/2 — a combined study that starts with early safety and continues into what the treatment does.
From the trial registry
Built from these fields:
- designModule.designInfo.primaryPurpose
- designModule.phases
Who receives what
There are 5 groups in this study.
Groups A, B, C, D and E receive SGT-003.
Registry label: A: Cohort 1: SGT-003 · B: Cohort 2: SGT-003 · C: Cohort 3: SGT-003 · D: Cohort 4: SGT-003 · E: Cohort 5: SGT-003
From the trial registry
Built from these fields:
- armsInterventionsModule.armGroups[].label
- armsInterventionsModule.armGroups[].type
- armsInterventionsModule.armGroups[].interventionNames
How the study is run
Groups are assigned by the study team using set rules, rather than by chance.
This study is open-labelOpen-labelEveryone knows which treatment is being given — nothing is hidden.Read more →: everyone knows which treatment is being given, including you and the study team.
From the trial registry
Built from these fields:
- designModule.designInfo.allocation
- designModule.designInfo.maskingInfo.masking
Is there a placebo?
This study does not list a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group.
From the trial registry
Built from these fields:
- armsInterventionsModule.armGroups[].type
- armsInterventionsModule.armGroups[].interventionNames
Who the study is looking for
The study lists an age range of 0 years to 17 years.
The study lists male participants only.
The study does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
These are the criteria the study lists. Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part.
From the trial registry
Built from these fields:
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How big and how long
The study aims to enrol about 60 people.
The study is currently expected to finish around May 2031.
The main measurement is taken over: Day 360.
From the trial registry
Built from these fields:
- designModule.enrollmentInfo.count
- statusModule.completionDateStruct.date
- outcomesModule.primaryOutcomes[].timeFrame
What the study measures
Incidence of treatment-emergent adverse eventsAdverse eventAny medical problem that happens during a study, whether or not the treatment caused it.Read more → (AEs) — measured over Day 360.
Change from baselineBaselineYour starting measurements, taken before treatment begins.Read more → in Microdystrophin Protein Levels — measured over Day 90.
From the trial registry
Built from these fields:
- outcomesModule.primaryOutcomes[].measure
- outcomesModule.primaryOutcomes[].timeFrame
Source: NCT06138639 on ClinicalTrials.gov. The full registry text is further down this page — this summary never replaces it.
Not medical advice. What do these terms mean?
What is being tested — in plain terms
About SGT-003Genetic
Adeno-associated virus serotype SLB101 containing the human microdystrophin gene (h-µD5)
From the trial registry — its own words, unedited.
What a genetic is here: A gene-based treatment — such as gene transfer or gene editing — being studied.
Read the full explanation → · in clinical review
Common questions
Answered from this trial’s registry record. Where the record doesn’t say, these answers say so rather than filling the gap.
Am I eligible for this trial?
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part. Concord cannot make that determination, and neither can any tool that has not examined you.
What the study lists: an age range of 0 years to 17 years.
The full inclusionInclusion criteriaThe things you must have or be for a study to consider you.Read more → and exclusion criteriaExclusion criteriaThe things that would prevent someone from taking part.Read more → are published on this page, exactly as the study team wrote them.
The useful next step is to bring this trial to your doctor. Answering a few questions first gives them something concrete to review.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.eligibilityCriteria
Is there a placebo?
This study does not list a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group.
From the trial registry
- armsInterventionsModule.armGroups[].type
Would I know which treatment I am getting?
This study is open-labelOpen-labelEveryone knows which treatment is being given — nothing is hidden.Read more →: everyone knows which treatment is being given, including you and the study team.
Groups are assigned by the study team using set rules, rather than by chance.
From the trial registry
- designModule.designInfo.maskingInfo.masking
- designModule.designInfo.allocation
Who can join?
The study lists an age range of 0 years to 17 years.
It lists male participants only.
It does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can confirm whether a particular person can take part.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How long would this take?
The study's main measurement is taken over: Day 360.
The study as a whole is currently expected to finish around 2031-05-06.
How long any one person takes part can differ from the study length. The study team can tell you what the schedule looks like in practice.
From the trial registry
- outcomesModule.primaryOutcomes[].timeFrame
- statusModule.completionDateStruct.date
How many people are taking part?
The study aims to enrol about 60 people.
From the trial registry
- designModule.enrollmentInfo.count
Where is this happening?
This study lists 2 locations, including: Toronto, Ontario, Canada; Seattle, Washington, United States.
Sites can open and close during a study, so confirm with the team before travelling.
From the trial registry
- trial_locations
About This Trial
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. Cohort 5 will include participants 10 to \< 18 years of age. Initiation of participant enrollment in Cohorts 4 and 5 will be subject to the accrual of safety and efficacy data from Cohorts 1-3. All participants will receive SGT-003 and will be enrolled in the study for 5 total years for long-term follow up.
Other Sites (1)
Seattle Children's Hospital
Seattle, Washington, United States
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See if this trial could fit youThis page is for informational purposes only and does not constitute medical advice. Clinical trial eligibility can only be determined by the trial site after proper screening. Trial information is sourced from ClinicalTrials.gov and may not reflect the most current status. Always consult your healthcare provider before making decisions about clinical trial participation.