Fiche société
Sarepta Therapeutics, Inc.
Traitements, essais et publications liés.
Traitements23programmes
Essais11liés
Publications11liées
SourceDBlocale
Traitements
23| 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 | À vérifier | À vérifier | United States | À vérifier |
| delandistrogene moxeparvovecThis open-label, single-arm study will evaluate the safety and expression of delandistrogene moxeparvovec in participants with DMD. Participants will be in the study for approximately 264 weeks. | Myopathies | Phase 1 | À vérifier | 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 |
| EteplirsenThis is a phase 4, multicenter, prospective, observational study designed to collect both medical history data and prospective data on Duchenne Muscular Dystrophy (DMD) treatment outcomes in participants receiving eteplirsen, golodirsen, and casimersen in routine clinical practice. Participants in this study will have been prescribed eteplirsen, golodirsen, or casimersen commercially prior to entry into the study. | Myopathies | À vérifier | 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 | À vérifier | À vérifier | United States | À vérifier |
| imlifidaseThis is a gene transfer therapy study evaluating the safety of delandistrogene moxeparvovec and delandistrogene moxeparvovec dystrophin expression in association with imlifidase, in participants with DMD with pre-existing antibodies to rAAVrh74 over a period of 104 weeks. | Myopathies, SEP | Phase 1 | À vérifier | Spain | 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 |
| delandistrogene moxeparvovecThis open-label, single-arm study will evaluate the safety and expression of delandistrogene moxeparvovec in participants with DMD. Participants will be in the study for approximately 264 weeks. | Myopathies | Phase 2 | À vérifier | Belgium, France, Germany, Italy, Spain, United Kingdom | À vérifier |
| delandistrogene moxeparvovecThis open-label, single-arm study will evaluate the safety and expression of delandistrogene moxeparvovec in participants with DMD. Participants will be in the study for approximately 264 weeks. | Myopathies | Phase 1 | À vérifier | United States | 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 |
| delandistrogene moxeparvovecThis open-label, single-arm study will evaluate the safety and expression of delandistrogene moxeparvovec in participants with DMD. Participants will be in the study for approximately 264 weeks. | Myopathies | À vérifier | À vérifier | United States | À vérifier |
| delandistrogene moxeparvovecThis open-label, single-arm study will evaluate the safety and expression of delandistrogene moxeparvovec in participants with DMD. Participants will be in the study for approximately 264 weeks. | Myopathies | Phase 1 | À vérifier | Spain | À vérifier |
| EteplirsenThis is a phase 4, multicenter, prospective, observational study designed to collect both medical history data and prospective data on Duchenne Muscular Dystrophy (DMD) treatment outcomes in participants receiving eteplirsen, golodirsen, and casimersen in routine clinical practice. Participants in this study will have been prescribed eteplirsen, golodirsen, or casimersen commercially prior to entry into the study. | Myopathies | À vérifier | À vérifier | United States | À vérifier |
| imlifidaseThis is a gene transfer therapy study evaluating the safety of delandistrogene moxeparvovec and delandistrogene moxeparvovec dystrophin expression in association with imlifidase, in participants with DMD with pre-existing antibodies to rAAVrh74 over a period of 104 weeks. | SEP | Phase 3 | À vérifier | United States | À vérifier |
Essais cliniques
11| Molécule | Indication / population | Phase | NCT | Titre | Statut |
|---|---|---|---|---|---|
| imlifidase | SEP | Phase 3 | NCT04935177 | ConfIdeS — Renal Function in Highly Sensitized Patients 1 Year After Desensitization With Imlifidase Prior to DD Kidney Tx | COMPLETED |
| Eteplirsen | Myopathies | À vérifier | NCT06606340 | EVOLVE — A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice | ENROLLING_BY_INVITATION |
| delandistrogene moxeparvovec | Myopathies | Phase 1 | NCT06241950 | A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Imlifidase Infusion in Participants With Duchenne Muscular Dystrophy (DMD) Determined to Have Pre-existing Antibodies to Recombinant Adeno-Associated Virus Serotype (rAAVrh74) | TERMINATED |
| delandistrogene moxeparvovec | Myopathies | À vérifier | NCT06270719 | ENDURE — An Observational Study Comparing Delandistrogene Moxeparvovec (ELEVIDYS) With Standard of Care in Participants With Duchenne Muscular Dystrophy | ENROLLING_BY_INVITATION |
| 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 | 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 |
| delandistrogene moxeparvovec | Myopathies | Phase 2 | NCT06128564 | ENVOL — A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD) | ACTIVE_NOT_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 | ENROLLING_BY_INVITATION |
| SRP-1003 IV Infusion | Myopathies | Phase 1/2 | NCT06138743 | Study of SRP-1003 in Participants With Type 1 Myotonic Dystrophy | ENROLLING_BY_INVITATION |
| 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
11| 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 | ||
| delandistrogene moxeparvovec | Safety, Tolerability, and Efficacy of a Prophylactic Sirolimus Protocol for Patients Receiving Delandistrogene Moxeparvovec-Rokl Gene Therapy. | Human gene 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 |