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Sarepta Therapeutics, Inc.
Traitements, essais et publications liés.
Traitements14programmes
Essais6liés
Publications10liées
SourceDBlocale
Traitements
14| Molécule | Indication / population | Phase | Objectif | Pays | Résultat |
|---|---|---|---|---|---|
| AntibioticsThe primary objective of this study is to evaluate acute liver injury (ALI) rates associated with ELEVIDYS with the addition of sirolimus as an adjunct prophylactic immunosuppression agent. | Myopathies | Phase 4 | À vérifier | United States | À vérifier |
| CasimersenThe 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). | Myopathies | Phase 3 | À vérifier | United States, Belgium, Bulgaria, Canada, Czechia, France, Germany, Israel, … | À vérifier |
| ELEVIDYSThe primary objective of this study is to evaluate acute liver injury (ALI) rates associated with ELEVIDYS with the addition of sirolimus as an adjunct prophylactic immunosuppression agent. | Myopathies | Phase 4 | À vérifier | United States | À vérifier |
| GlucocorticoidsThe primary objective of this study is to evaluate acute liver injury (ALI) rates associated with ELEVIDYS with the addition of sirolimus as an adjunct prophylactic immunosuppression agent. | Myopathies | Phase 4 | À vérifier | United States | À vérifier |
| GolodirsenThe 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). | Myopathies | Phase 3 | À vérifier | United States, Belgium, Bulgaria, Canada, Czechia, France, Germany, Israel, … | À vérifier |
| SRP-1001 for InjectionThe purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of SRP-1001 in participants with facioscapulohumeral muscular dystrophy Type 1 (FSHD1). In Part 1 of the study, participants will receive one dose of SRP-1001 or placebo. In Part 2 of the study, participants will receive 4 doses of SRP-1001 or placebo. Participants who complete Part 1 will have the option to re-screen and re-randomize into Part 2. All participants will undergo pre- and post-dose magnetic imaging resonance (MRI)-guided muscle biopsies (a total of 2 biopsies). Participants who complete Part 1 and enroll in Part 2 will be required to undergo an additional screening biopsy. Participants completing Part 1 or Part 2 may have the option to continue to receive drug in an open-label extension study or may be eligible to participate in later-stage clinical studies. | Myopathies | Phase 1/2 | À vérifier | Australia, Canada, Germany, Italy, Netherlands, New Zealand, Spain | À vérifier |
| SRP-1003 IV InfusionThis is a phase 1/2a double-blinded, placebo-controlled, dose-escalating study to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of single and multiple ascending doses of SRP-1003 compared to placebo in male and female participants with type 1 myotonic dystrophy (DM1). Participants who have provided written informed consent and met all protocol eligibility requirements will be randomized to receive single (Part 1) or multiple (Part 2) doses of SRP-1003 or placebo. | Myopathies | Phase 1/2 | À vérifier | Australia, Belgium, Canada, France, Germany, Italy, New Zealand, Spain, … | À vérifier |
| SRP-1003 SC InjectionThis is a phase 1/2a double-blinded, placebo-controlled, dose-escalating study to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of single and multiple ascending doses of SRP-1003 compared to placebo in male and female participants with type 1 myotonic dystrophy (DM1). Participants who have provided written informed consent and met all protocol eligibility requirements will be randomized to receive single (Part 1) or multiple (Part 2) doses of SRP-1003 or placebo. | Myopathies | Phase 1/2 | À vérifier | Australia, Belgium, Canada, France, Germany, Italy, New Zealand, Spain, … | À vérifier |
| SRP-9003The primary purpose of this study is to evaluate the safety of SRP-9003 and to quantify expression of β-SG in the skeletal muscle of participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4). The study will include both ambulatory (Cohort 1) and non-ambulatory (Cohort 2) participants. | Myopathies | Phase 1 | À vérifier | United States | À vérifier |
| CasimersenThe 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). | Myopathies | Phase 3 | À vérifier | United States, Belgium, Bulgaria, Canada, Czechia, France, Germany, Israel, Italy, Poland, Spain, Sweden, United Kingdom | À vérifier |
| SRP-9003The primary purpose of this study is to evaluate the safety of SRP-9003 and to quantify expression of β-SG in the skeletal muscle of participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4). The study will include both ambulatory (Cohort 1) and non-ambulatory (Cohort 2) participants. | Myopathies | Phase 1 | À vérifier | United States | À vérifier |
| SRP-1003 IV InfusionThis is a phase 1/2a double-blinded, placebo-controlled, dose-escalating study to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of single and multiple ascending doses of SRP-1003 compared to placebo in male and female participants with type 1 myotonic dystrophy (DM1). Participants who have provided written informed consent and met all protocol eligibility requirements will be randomized to receive single (Part 1) or multiple (Part 2) doses of SRP-1003 or placebo. | Myopathies | Phase 1/2 | À vérifier | Australia, Belgium, Canada, France, Germany, Italy, New Zealand, Spain, Taiwan, Thailand, United Kingdom | À vérifier |
| SRP-1001 for InjectionThe purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of SRP-1001 in participants with facioscapulohumeral muscular dystrophy Type 1 (FSHD1). In Part 1 of the study, participants will receive one dose of SRP-1001 or placebo. In Part 2 of the study, participants will receive 4 doses of SRP-1001 or placebo. Participants who complete Part 1 will have the option to re-screen and re-randomize into Part 2. All participants will undergo pre- and post-dose magnetic imaging resonance (MRI)-guided muscle biopsies (a total of 2 biopsies). Participants who complete Part 1 and enroll in Part 2 will be required to undergo an additional screening biopsy. Participants completing Part 1 or Part 2 may have the option to continue to receive drug in an open-label extension study or may be eligible to participate in later-stage clinical studies. | Myopathies | Phase 1/2 | À vérifier | Australia, Canada, Germany, Italy, Netherlands, New Zealand, Spain | À vérifier |
| ELEVIDYSThe primary objective of this study is to evaluate acute liver injury (ALI) rates associated with ELEVIDYS with the addition of sirolimus as an adjunct prophylactic immunosuppression agent. | Myopathies | Phase 4 | À vérifier | United States | À vérifier |
Essais cliniques
6| Molécule | Indication / population | Phase | NCT | Titre | Statut |
|---|---|---|---|---|---|
| ELEVIDYS | Myopathies | Phase 4 | NCT07542314 | ENHANCE — Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting | NOT_YET_RECRUITING |
| delandistrogene moxeparvovec | Myopathies | Phase 1 | NCT04626674 | ENDEAVOR — A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) - Non-Ambulatory Cohort | RECRUITING |
| SRP-1001 for Injection | Myopathies | Phase 1/2 | NCT06131983 | Study of SRP-1001 in Adult and Adolescent Participants With Facioscapulohumeral Muscular Dystrophy Type 1 | RECRUITING |
| SRP-1003 IV Infusion | Myopathies | Phase 1/2 | NCT06138743 | Study of SRP-1003 in Participants With Type 1 Myotonic Dystrophy | RECRUITING |
| SRP-9003 | Myopathies | Phase 1 | NCT05876780 | A Gene Transfer Single Dose Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2E/R4 (Beta-Sarcoglycan [β-SG] Deficiency) | ACTIVE_NOT_RECRUITING |
| Casimersen | Myopathies | Phase 3 | NCT03532542 | An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy | TERMINATED |
Publications
10| Molécule | Indication / population | Titre | Journal | Date |
|---|---|---|---|---|
| Antibiotics | Non-prescription antibiotic dispensing in community pharmacies in jordan: a simulated patient study. | The Libyan journal of medicine | ||
| Antibiotics | Biomimetic stress granules replenish lysosomal repair to reinstate macrophage immunometabolic antibacterial programs. | Bioactive materials | ||
| Casimersen | A Real-World Target Trial Emulation of Eteplirsen, Casimersen, and Golodirsen to Evaluate Survival Among Patients with Duchenne Muscular Dystrophy. | Advances in therapy | ||
| SRP-1003 SC Injection | Degradation-Robust Hue Prior Network for Low-Light Rainy Image Restoration. | Sensors (Basel, Switzerland) | ||
| SRP-1003 SC Injection | Elegans-Inspired Magnetic Polyurethane Soft Robot (MPSR) for Closed Visceral Secondary Hemostasis In Situ. | Advanced healthcare materials | ||
| SRP-1003 IV Infusion | Transient Neonatal Myasthenia Gravis in a Newborn Infant Presenting to an Emergency Department. | Cureus | ||
| SRP-1003 IV Infusion | Remifentanil attenuates LPS-induced hepatic injury by modulating NRF2/HO-1 and necroptosis-related gene expression. | Naunyn-Schmiedeberg's archives of pharmacology | ||
| SRP-9003 | Limb-Girdle Muscular Dystrophy Scientific Workshop: A Multistakeholder Discussion Focused on Charting the Path Forward for Drug Development. | Neurology. Clinical practice | ||
| Casimersen | FDA-approved antisense oligonucleotide therapies for duchenne muscular dystrophy: current status and future outlook. | RNA biology | ||
| Casimersen | RNA Therapeutics Targeting Skeletal Muscle: Emerging Antisense and Gene-Modifying Strategies. | Biomolecules |