2026-09-10
minor
Study Status, Eligibility, Contacts/Locations v15
Trial sites expanded: 7 → 12 locations
Eligibility criteriarevised
[...] ening, has a performance upper limb (PUL) entry item score ≥32 at the Screening visit and has a total PUL score of ≥2012 and ≤40 at the time of Screening.
Cohorts 1, 2, 3, 5, 7 and [...]
2026-06-19
minor
Study Identification, Study Status, Study Description, Conditions, Eligibility v14
Eligibility criteria+19 characters
[...] on documented clinical findings and prior genetic testing.
Cohort 8: Non-ambulatory per protocol-specified criteria at the time of Screening, has a performance upper limb (PUL) entry item score ≥3 at the Screening visit and has a total PUL score of ≥20 and ≤40 at the time of Screening.
⇅ moved Cohorts 1, 2, 3, 5, 7 and 8 only: Stable dose equivalent of oral glucocorticoids for at least 12 weeks before screening and the dose is expected to remain constant (except for modifications to accommodate changes in weight) throughout the first year of the study.
Cohort 1: Is ambulatory, and ≥4 to <8 years of a [...] [...]
Study description-104 characters
Enrollment for Cohorts 1 through 7 has been completed.
Cohort 8 is currently enrolling new participants.
Study title+24 characters
[...] 9001) in Participants With Duchenne Muscular Dystrophy (DMD) - Non-Ambulatory Cohort
2025-12-19
notable
Active, Not Recruiting→Recruiting Study Status, Study Design, Outcome Measures, Eligibility, Contacts/Locations v11
Trial statusActive, Not Recruiting→Recruiting
Primary completion date2024-07-23→2027-12-31
Completion date2026-07-31→2028-02-29
Enrollment target55→83
Eligibility criteria+450 characters
Inclusion Criteria:
For Cohorts 1-78: Has a definitive diagnosis of DMD based on documented clin [...] creening.
Cohort 5a: Is ambulatory and ≥4 to <9 years of age with time to rise from the floor ≤7 seconds at the screening visit.
Cohort 5b: Non-ambulatory per protocol specified criteria [...] y per protocol-specified criteria at the time of Screening.
Cohort 8: Non-ambulatory per protocol-specified criteria at the time of Screening, has a performance upper limb (PUL) entry item score ≥3 at the Screening visit and has a total PUL score of ≥20 and ≤40 at the time of Screening.
Ability to cooperate with mot [...] [...]
Secondary endpoints7 to 11 entries
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Adverse Events (AEs) of Special Interest (AESIs)
Cohort 8: Number of Participants With Infections, Edema, Wound-healing Complications, Hyperlipidemia, Angioedema, and Intestinal Lung Disease/ Non-infectious Pneumonitis
Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12 as Measured by IF Fiber Intensity
Cohort 8: Number of Participants With Hepatic AEs, Hepatic Biomarkers, and Laboratory Assessments Indicative of Either Acute Hepatocellular Injury or Acute Live [...]
Primary endpoints2 to 3 entries
Cohort 8: Number of Participants with Acute Liver Injury (ALI)
Part 1 (Cohorts 1 to 5): Change from Baseline in Quantity of Delandistrogene Moxepa [...] in Expression at Week 12, as Measured by Western Blot
Part 1 (Cohorts 6 to 8): Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western Blot
Study sitesdetails revised at 5 of 5 sites
Show 10 earlier versions
2025-08-08
minor
Study Status v10
Re-verified, no change to tracked fields
2024-08-22
notable
Enrolling by Invitation→Active, Not Recruiting Study Status, Study Design v9
Trial statusEnrolling by Invitation→Active, Not Recruiting
Primary completion dateestimated 2024-11-30→confirmed 2024-07-23
Enrollment targetestimated 58→confirmed 55
2023-07-28
minor
Study Status, Study Description, Study Design, Outcome Measures, Eligibility v8
Primary completion date2023-08-31→2024-11-30
Completion date2028-01-31→2026-07-31
Enrollment target46→58
Eligibility criteria+197 characters
Inclusion Criteria:
For Cohorts 1-7: Has a definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
Cohort 1 only: Is ambulatory, and ≥4 to <8 years of age at the time of Screening.
Cohort 2 only: Is ambulatory, and ≥8 to <18 years of age at the time of Screening.
Cohort 3 and Cohort 5b: Non-ambulatory per protocol specified criteria at the time of Screening.
Cohort 4 only: Is ambulatory and ≥3 to <4 years of age at the time of Screening.
Cohort 5a only: Is ambulatory and ≥4 to <9 years of age.
Cohort 5b: Non-ambulatory per protocol specified criteria at the [...] [...]
Secondary endpoints5 to 7 entries
Change from Baseline in Quantity of Delandistrogene Moxeparvovec Micro-Dystrophin Protein Expression at Week 12, as Measured by Imm [...] e from Baseline in Quantity of Delandistrogene Moxeparvovec Micro-Dystrophin Protein Expression at Week 12, as Measured by IF Percent Dystrophin Positive Fibers (PDPF)
Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12 as Measured by IF Fiber Intensity:
Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12 as Measured by IF PDPF
Primary endpoints1 to 2 entries
Part 1: Change from Baseline in Quantity of Delandistrogene Moxeparvovec Micro-Dystrophin Protein Expression at Week 12, as Measured by Western Blot
Part 1: Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western Blot
2023-06-30
minor
Study Identification, Study Status, Study Description, Conditions, Arms and Interventions, Outcome Measures v7
Primary completion date2023-05-31→2023-08-31
InterventionsSRP-9001 (Genetic)→delandistrogene moxeparvovec (Genetic)
Secondary endpoints5 entries, revised
Change Fromfrom Baseline in Quantity of Delandistrogene Moxeparvovec Micro-Dystrophin Protein Expression at Week 12, as Measured by Immunofluorescence (IF) Fiber Intensity
Change Fromfrom Baseline in Quantity of Delandistrogene Moxeparvovec Micro-Dystrophin Protein Expression at Week 12, as Measured by IF Percent Dystrophin Positive Fibers (PDPF)
Primary endpoints1 entry, revised
Part 1: Change Fromfrom Baseline in Quantity of Delandistrogene Moxeparvovec Micro-Dystrophin Protein Expression at Week 12, as Measured by Western Blot
Study titlerevised
A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001 (Delandistrogene Moxeparvovec) in Participants With Duchenne Muscular Dystrophy (DMD)
2022-09-28
minor
Study Status, Conditions, Study Design, Eligibility v6
Primary completion date2022-10-31→2023-05-31
Completion date2026-07-31→2028-01-31
Enrollment target38→46
Eligibility criteria+137 characters
[...] d ≥8 to <18 years of age at the time of Screening.
Cohort 3 onlyand Cohort 5b: Non-ambulatory per protocol specified criteria at the time of Screening.
Cohort 4 only: Is ambulatory, and ≥3 to <4 years of age at the time of Screening.
DefinitiveCohort 5a only: Is ambulatory and ≥4 to <9 years of age at the time of Screening
For Cohort 1-5: Has a definitive diagnosis of DMD based on documented clinical findings and [...] y to cooperate with motor assessment testing.
Cohorts 1, 2, 3, and 35 only: Stable dose equivalent of oral corticosteroids for at [...] specified requirements.
Genetic mutation inclusion cri [...]
2022-06-05
minor
Study Identification, Study Status, Study Description, Arms and Interventions, Eligibility v5
Primary completion date2022-03-31→2022-10-31
Completion date2026-07-30→2026-07-31
Eligibility criteria+54 characters
[...] rs are not elevated as per protocol-specified requirements.
Genetic mutation inclusion criteria varies by cohort.
Exclusion Criteria:
Has a concomitant illness, autoimmune di [...]
Study title+31 characters
A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From SRP-9001 (Delandistrogene Moxeparvovec) in Participants With Duchenne Muscular Dystrophy (DMD)
2021-11-05
minor
Study Identification, Study Status, Sponsor/Collaborators, Study Description, Study Design, Eligibility v4
Primary completion date2021-12-31→2022-03-31
Enrollment target32→38
Eligibility criteria+442 characters
[...] ort 3 only: Non-ambulatory per protocol specified criteria.
Cohort 4 only: Is ambulatory, and ≥3 to <4 years of age at the time of Screening.
Definitive diagnosis of DMD based on documented clinical fin [...] esting.
Ability to cooperate with motor assessment testing.
Cohorts 1, 2, and 3 only: Stable dose equivalent of oral corticosteroids for at least 12 weeks before screening and the dose is expected to remain constant (except for modifications to accommodate changes in weight) throughout the first year of the study.
Cohort 4: Do not yet require use of chronic steroids for treatment of their DMD, i [...] [...]
Collaborators1 entry, revised
Hoffmann-La Roche Pharma AG
Study title+26 characters
A Gene DeliveryTransfer Therapy Study to Evaluate the Safety of and Expression From SRP-9001 in Participants With Duchenne Muscular Dystrophy (DMD)
2021-06-16
notable
Recruiting→Enrolling by Invitation Study Status, Study Description, Study Design, Arms and Interventions, Outcome Measures, Eligibility, Contacts/Locations v3
Trial statusRecruiting→Enrolling by Invitation
Primary completion date2021-07→2021-12-31
Completion date2026-03→2026-07-30
Enrollment target20→32
Study sites4→5
Eligibility criteria+115 characters
Inclusion Criteria:
Cohort 1 only: Is male at birthambulatory, ambulatory and from ≥4 to under <8 years of age at the time of randomizationScreening.
Cohort 2 only: Is ambulatory, and ≥8 to <18 years of age at the time of Screening.
Cohort 3 only: Non-ambulatory per protocol specified criteria.
Definitive diagnosis of Duchenne Muscular DystrophyDMD based on documented clinical findings and prior genetic testing.
Ability to cooperate with motor assessment testing.
Stable [...]
Secondary endpoints6 to 5 entries
Vector Shedding as, Measured by Polymerase Chain Reaction in Urine, Saliva, and Stool Samples Post-infusionInfusion
Immunogenicity of SRP-9001 Determined by Level of Antibody Titers to Recombinant Adeno-Associated Virus Serotype rh74 (rAAVrh74)
IncidenceNumber of Participants With Treatment Emergent Adverse Events (TEAEs), Serious AEsAdverse Events (SAEs) and Adverse Events (AEs) of Special Interest
Number of Participants with Clinically Significant Abnormalities in Vital Signs, Physical Examinations, Laboratory Parameters, Electrocardiograms (ECG) and Echocardiograms (ECHO) Findings
Change Fr [...] [...]
Primary endpoints1 entry, revised
Part 1: Change From Baseline in Quantity of Micro-dystrophinDystrophin Protein Expression at Week 12, as Measured by Western Blot
2021-03-03
minor
Study Status, Study Design, Outcome Measures, Contacts/Locations v2
Primary completion date2021-05-31→2021-07
Completion date2025-12→2026-03
Enrollment target10→20
Secondary endpoints4 to 6 entries
Change From Baseline in Quantity of Micro-dystrophin Protein Expression Measured by Immunofluorescence (IF) Fiber Intensity
Change From Baseline in Quantity of Micro-dystrophin Expression Measured by Immunofluorescence Percent Dystrophin Positive Fibers (PDPF)
Study sitesdetails revised at 1 of 4 sites
2020-12-22
notable
Not Yet Recruiting→Recruiting Study Status, Study Description, Arms and Interventions, Outcome Measures, Contacts/Locations v1
Trial statusNot Yet Recruiting→Recruiting
Start dateestimated 2020-11-11→confirmed 2020-11-23
Study sites0→4
Secondary endpoints4 entries, revised
Immunogenicity of SRP-9001 as AssessedDetermined by Level of Antibody Titers to rAAVrh74