CLINICAL TRIALS PROFILE FOR GOLODIRSEN
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All Clinical Trials for golodirsen
| Trial ID | Title | Status | Sponsor | Phase | Start Date | Summary |
|---|---|---|---|---|---|---|
| NCT02500381 ↗ | Study of SRP-4045 and SRP-4053 in Participants With Duchenne Muscular Dystrophy (DMD) | Recruiting | Sarepta Therapeutics | Phase 3 | 2016-09-28 | The main objective of this study is to evaluate the efficacy of SRP-4045 and SRP-4053 compared to placebo in participants with DMD with out-of-frame deletion mutations amenable to skipping exon 45 and exon 53, respectively. |
| NCT02500381 ↗ | Study of SRP-4045 and SRP-4053 in Participants With Duchenne Muscular Dystrophy (DMD) | Recruiting | Sarepta Therapeutics, Inc. | Phase 3 | 2016-09-28 | The main objective of this study is to evaluate the efficacy of SRP-4045 and SRP-4053 compared to placebo in participants with DMD with out-of-frame deletion mutations amenable to skipping exon 45 and exon 53, respectively. |
| NCT03532542 ↗ | An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy | Enrolling by invitation | Sarepta Therapeutics | Phase 3 | 2018-08-02 | The main objective of this study is to evaluate the safety and tolerability of long-term treatment with casimersen or golodirsen in patients with Duchenne muscular dystrophy (DMD). |
| NCT03532542 ↗ | An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy | Enrolling by invitation | Sarepta Therapeutics, Inc. | Phase 3 | 2018-08-02 | The main objective of this study is to evaluate the safety and tolerability of long-term treatment with casimersen or golodirsen in patients with Duchenne muscular dystrophy (DMD). |
| NCT04179409 ↗ | A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of Casimersen, Eteplirsen and Golodirsen in Subjects With Duchenne Muscular Dystrophy Carrying Eligible DMD Duplications | Active, not recruiting | Sarepta Therapeutics, Inc. | Phase 2 | 2020-02-18 | This is an 48-week open-label study to determine the efficacy and safety of casimersen, eteplirsen, or golodirsen for the treatment of boys with duchenne muscular dystrophy who have a single exon duplication of either exon 45, 51 or 53, respectively. There will be weekly infusions and two muscle biopsies at baseline and at month 12. |
| NCT04179409 ↗ | A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of Casimersen, Eteplirsen and Golodirsen in Subjects With Duchenne Muscular Dystrophy Carrying Eligible DMD Duplications | Active, not recruiting | Kevin Flanigan | Phase 2 | 2020-02-18 | This is an 48-week open-label study to determine the efficacy and safety of casimersen, eteplirsen, or golodirsen for the treatment of boys with duchenne muscular dystrophy who have a single exon duplication of either exon 45, 51 or 53, respectively. There will be weekly infusions and two muscle biopsies at baseline and at month 12. |
| NCT04708314 ↗ | An Open-Label Study of Golodirsen in Non-Ambulant Patients With Duchenne Muscular Dystrophy | Terminated | Sarepta Therapeutics, Inc. | Phase 4 | 2020-10-31 | This is an open-label study to evaluate the safety and tolerability of golodirsen injection in Non-ambulant DMD patients with confirmed genetic mutations amenable to treatment by exon 53 skipping (Golodirsen). Golodirsen 30 mg/kg will be administered as an intravenous (IV) infusion over approximately 35 to 60 minutes once a week during the treatment period (up to 96 weeks). After the treatment period, patients can go into a safety extension period (not to exceed 48 weeks) until the patient is able to transition to commercially available drug or a separate golodirsen study. Safety will be regularly assessed throughout the study via the collection of adverse events (AEs), laboratory tests, electrocardiograms (ECGs), echocardiograms (ECHOs), vital signs, and physical examinations. Exploratory assessments, including pulmonary function tests (PFTs), upper extremity testing, and other measurements of functional status, will occur at functional assessment visits every 12 weeks over the first year of treatment and approximately every 24 weeks over the second year of treatment. |
| >Trial ID | >Title | >Status | >Sponsor | >Phase | >Start Date | >Summary |
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