Study Results
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View full resultsBasic Information
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COMPLETED
PHASE1/PHASE2
39 participants
INTERVENTIONAL
2015-01-13
2019-03-25
Brief Summary
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Detailed Description
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Part 2: Open-label evaluation of SRP-4053 in patients from Part 1, along with newly enrolled DMD patients with deletions amenable to exon 53 skipping and an untreated group of DMD patients with deletions not amenable to exon 53 skipping.
Safety, including adverse event monitoring and routine laboratory assessments, will be followed on an ongoing basis for all patients.
Clinical efficacy, including functional tests such as the six-minute walk test (6MWT), will be assessed at regularly scheduled study visits. Patients in the treated groups will undergo one baseline and one follow-up muscle biopsy. Patients in the untreated group will not undergo biopsies and will follow an abbreviated schedule of study assessments.
Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Part 1: SRP-4053
Patients will receive SRP-4053 (golodirsen) intravenous (IV) infusions, weekly, at escalating dose levels as follows: Weeks 1-2, 4 mg/kg/week; Weeks 3-4, 10 mg/kg/week; Weeks 5-6, 20 mg/kg/week; Weeks 7-12, 30 mg/kg/week. Dosing will be interrupted or halted if specific predefined stopping criteria are met or if warranted at the discretion of the Sponsor or Investigator.
SRP-4053
SRP-4053 (golodirsen) solution for IV infusion.
Part 1: Placebo
Patients will receive SRP-4053 placebo-matching IV infusions, weekly, for 12 weeks. Dosing will be interrupted or halted if specific predefined stopping criteria are met or if warranted at the discretion of the Sponsor or Investigator.
Placebo
SRP-4053 placebo-matching solution for IV infusion.
Part 2: SRP-4053
All eligible patients from Part 1, as well as new patients, will receive SRP-4053 (golodirsen) 30 mg/kg/week IV infusions, weekly, for up to 168 weeks.
SRP-4053
SRP-4053 (golodirsen) solution for IV infusion.
Part 2: Untreated Group
Patients with DMD who have a genotypically confirmed deletion of exon(s) not amenable to treatment by exon 53 skipping, but who otherwise meet the same eligibility criteria as treated patients newly recruited to Part 2, will undergo the same study assessments as treated Patients (except for pharmacokinetic \[PK\] sampling and muscle biopsies), but at a reduced schedule through Week 144. The untreated patients are not considered as control group.
No interventions assigned to this group
Interventions
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Placebo
SRP-4053 placebo-matching solution for IV infusion.
SRP-4053
SRP-4053 (golodirsen) solution for IV infusion.
Eligibility Criteria
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Inclusion Criteria
* Intact right and left biceps muscles or an alternative upper arm muscle group.
* Stable pulmonary and cardiac function.
* Minimum performance on 6MWT, North Star Ambulatory Assessment, and rise (Gowers) test as specified in the study protocol.
* On a stable dose of corticosteroids for at least 6 months.
Exclusion Criteria
* Current or previous treatment with any other experimental treatments within 12 weeks prior to study entry.
* Major surgery within the last 3 months.
* Presence of other clinically significant illness.
* Major change in physical therapy regime within the last 3 months.
6 Years
15 Years
MALE
No
Sponsors
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Institut de Myologie, France
OTHER
Consultants for Research in Imaging and Spectroscopy
OTHER
Great Ormond Street Hospital for Children NHS Foundation Trust
OTHER
Catholic University of the Sacred Heart
OTHER
Royal Holloway University
OTHER
SYSNAV
INDUSTRY
University College, London
OTHER
University of Newcastle Upon-Tyne
OTHER
Sarepta Therapeutics, Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Medical Director
Role: STUDY_DIRECTOR
Sarepta Therapeutics, Inc.
Locations
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Boston Children's Hospital
Boston, Massachusetts, United States
Institute de Myologie
Paris, , France
Policlinico Universitario A Gemelli
Rome, , Italy
Great Ormond Street Hospital for Children NHS Foundation Trust
London, , United Kingdom
Newcastle University Hospital
Newcastle, , United Kingdom
Countries
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References
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Frank DE, Schnell FJ, Akana C, El-Husayni SH, Desjardins CA, Morgan J, Charleston JS, Sardone V, Domingos J, Dickson G, Straub V, Guglieri M, Mercuri E, Servais L, Muntoni F; SKIP-NMD Study Group. Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy. Neurology. 2020 May 26;94(21):e2270-e2282. doi: 10.1212/WNL.0000000000009233. Epub 2020 Mar 5.
Servais L, Mercuri E, Straub V, Guglieri M, Seferian AM, Scoto M, Leone D, Koenig E, Khan N, Dugar A, Wang X, Han B, Wang D, Muntoni F; SKIP-NMD Study Group. Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A First-in-human, Multicenter, Two-Part, Open-Label, Phase 1/2 Trial. Nucleic Acid Ther. 2022 Feb;32(1):29-39. doi: 10.1089/nat.2021.0043. Epub 2021 Nov 17.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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4053-101
Identifier Type: -
Identifier Source: org_study_id
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