| Idefirix (imlifidase) / Hansa Biopharma, Sarepta Therap |
2021-002640-70: Study in highly sensitised kidney transplant patients with a high degree of antibodies against the donor's kidney to see the efficacy and safety of imlifidase to temporarily remove the antibodies and enable the transplantation. |
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| Not yet recruiting | 3 | 150 | Europe | Imlifidase, [Not applicable], Powder for concentrate for solution for infusion, Idefirix | Hansa Biopharma AB, Hansa Biopharma AB | end stage chronic kidney disease (CKD), are highly sensitized and placed on the kidney transplant list awaiting a kidney transplant., Body processes [G] - Immune system processes [G12] | | | | |
ConfIdeS, NCT04935177: Renal Function in Highly Sensitized Patients 1 Year After Desensitization With Imlifidase Prior to DD Kidney Tx |
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| Active, not recruiting | 3 | 64 | US | Imlifidase, IdeS, HMED-IdeS, PLEX, Plasma exchange, PE, IVIg, Intravenous immunoglobulin, Anti-CD20 antibodies, Rituximab, Eculizumab, Soliris, Remain on wait list | Hansa Biopharma AB | Kidney Transplantation in Highly Sensitized Patients | 06/25 | 06/25 | | |
PAES, NCT05369975: Efficacy and Safety in Imlifidase Desensitized Kidney Tx Patients, Including Two Non-Comparative Reference Cohorts |
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| Recruiting | 3 | 225 | Europe, RoW | Imlifidase, IdeS, HMED-IdeS, Normal Transplantation Routine | Hansa Biopharma AB | Kidney Transplantation in Highly Sensitized Patients | 12/24 | 12/24 | | |
| Recruiting | 3 | 50 | Europe, US, RoW | Imlifidase, IdeS, HMED-IdeS, Plasma exchange (PLEX), PE, Cyclophosphamide (CYC), Glucocorticoids | Hansa Biopharma AB | Anti-Glomerular Basement Membrane Disease, Anti-Glomerular Basement Membrane Antibody Disease, Goodpasture Syndrome, Good Pasture Syndrome | 06/25 | 11/26 | | |
PAES-LTFU, NCT05937750: A Long-term Follow-up Trial of Patients Previously Treated With Imlifidase Prior to Kidney Transplantation and a Non-comparative Transplanted Patient Group |
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| Recruiting | 3 | 150 | Europe, RoW | Imlifidase administered in the 20-HMedIdeS-19 (PAES) study, IdeS, HMED-IdeS, Idefirix, Best available treatment administered in the 20-HMedIdeS-19 (PAES) study | Hansa Biopharma AB | Long Term Efficacy and Safety | 12/28 | 12/28 | | |
2018-001059-12: A clinical study investigating the safety and effect of Imlifidase (IdeS) in patients with Guillain-Barré Syndrome (GBS) |
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| Not yet recruiting | 2 | 30 | Europe | Imlifidase, Imlifidase, Powder for concentrate for solution for infusion, Idefirix | Hansa Biopharma AB, Hansa Biopharma AB | Guillain-Barré Syndrome (GBS), Guillain-Barré Syndrome (GBS), Diseases [C] - Immune System Diseases [C20] | | | | |
NCT03897205 / 2018-000022-66: An Efficacy and Safety Study of Imlifidase in Treatment of Antibody-Mediated Rejection in Kidney Transplant Patients |
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| Completed | 2 | 30 | Europe, US, RoW | Imlifidase, HMED-IdeS, IdeS, IgG endopeptidase, Plasma Exchange | Hansa Biopharma AB | Kidney Transplant Rejection | 05/22 | 11/22 | | |
2021-004706-22: Imlifidase for the treatment of autoimmune-mediated small blood vessel inflammation Imlifidase zur Behandlung von autoimmun-vermittelter Entzündung der kleinen Blutgefäße |
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| Not yet recruiting | 2 | 10 | Europe | Idefirix, Powder for concentrate for solution for infusion, Idefirix | Charité - Universitätsmedizin Berlin, Hansa Biopharma | Anti-neutrophil cytoplasmic antibody (ANCA) associated vasculitis with severe diffuse alveolar hemorrhage Anti-Neutrophile cytoplasmatische Antikörper (ANCA) assoziierte Vaskulitis mit schwerer diffuser alveolärer Hämorrhagie, Auto-immune inflammation of the lungs with severe bleeding Auto-immun vermittelte Entzündung der Lunge mit schwerer Blutung, Diseases [C] - Immune System Diseases [C20] | | | | |
NCT05049850: A Study to Investigate DSA Rebound in Patients Treated with Imlifidase Prior to Transplantation |
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| Terminated | 2 | 3 | NA | Imlifidase, IdeS, HMED-IdeS | Hansa Biopharma AB | Kidney Transplantation in Highly Sensitized Patients | 02/24 | 05/24 | | |
NCT03943589: A Study of Imlifidase in Patients With Guillain-Barré Syndrome |
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| Completed | 2 | 30 | Europe | Imlifidase, Hansa Medical-Immunoglobulin G degrading enzyme of Streptococcus pyogenes (HMED-IdeS), IdeS, IgG endopeptidase | Hansa Biopharma AB | Guillain-Barré Syndrome (GBS) | 02/24 | 02/24 | | |
| Recruiting | 2 | 10 | Europe | Imlifidase, IdeS, HMED-IdeS | Hansa Biopharma AB | Kidney Transplantation in Highly Sensitized Patients | 09/24 | 09/29 | | |
NCT06518005: Efficacy and Safety of GNT0003 Following Imlifidase Pre-treatment in Severe Crigler-Najjar Syndrome |
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| Not yet recruiting | 2 | 3 | Europe | Imlifidase, GNT0003 | Genethon, Hansa Biopharma AB | Crigler-Najjar Syndrome | 09/26 | 09/30 | | |
| Not yet recruiting | 2 | 10 | NA | Imlifidase | Fundacion Clinic per a la Recerca Biomédica | Kidney Transplant Candidates | 07/26 | 12/26 | | |
2022-003407-15: An Open-Label, Systemic Gene Delivery Study to Evaluate the Safety, Tolerability and Expression of SRP-9001 in association with imlifidase in Subjects with Duchenne Muscular Dystrophy with pre-existing Antibodies to rAAVrh74 Estudio abierto de liberación génica sistémica para evaluar la seguridad, la tolerabilidad y la expresión de SRP-9001 en asociación con imlifidasa en sujetos con distrofia muscular de Duchenne con anticuerpos preexistentes contra rAAVrh74 |
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| Not yet recruiting | 1 | 6 | Europe | Imlifidase, SRP-9001, Solution for infusion, Powder for concentrate for solution for infusion, Idefirix | Sarepta Therapeutics, Inc, Sarepta Therapeutics, Inc | Duchenne muscular dystrophy Distrofia muscular de Duchenne, Duchenne muscular dystrophy Distrofia muscular de Duchenne, Diseases [C] - Nervous System Diseases [C10] | | | | |
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) |
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| Enrolling by invitation | 1 | 6 | Europe | delandistrogene moxeparvovec, SRP-9001, delandistrogene moxeparvovec-rokl, ELEVIDYS, imlifidase, Idefirix | Sarepta Therapeutics, Inc., Hansa Biopharma AB | Duchenne Muscular Dystrophy | 01/25 | 09/26 | | |
NCT03611621: A Follow up Study of Patients Treated With Imlifidase Prior to Kidney Transplantation |
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| Completed | N/A | 39 | Europe, US | Imlifidase, IdeS, HMED-IdeS | Hansa Biopharma AB | Kidney Transplant Failure and Rejection | 02/23 | 02/23 | | |
| Terminated | N/A | 18 | Europe | Imlifidase, IdeS, HMED-IdeS | Hansa Biopharma AB | Kidney Transplant Rejection | 03/23 | 03/23 | | |
ConfIdeSLTFU, NCT05714514: Survival and Dialysis Independency in Highly Sensitized Patients After Desensitization With Imlifidase and Tx of Kidneys |
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| Recruiting | N/A | 64 | US | Imlifidase administered in the ConfIdeS study, IdeS, HMED-IdeS, Best available treatment administered in the ConfIdeS study | Hansa Biopharma AB | Kidney Transplantation in Highly Sensitized Patients | 12/27 | 12/29 | | |
| ezutromid (SMT C1100) / Sarepta Therap |
2014-003100-78: A clinical trial to test if the drug SMT C1100 is safe and well absorbed compared to placebo when given to children with Duchenne Muscular Dystrophy (DMD), who follow a balanced diet. |
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| Ongoing | 1 | 12 | Europe | SMT C1100, Oral suspension | Summit (Oxford) Limited, Summit (Oxford) Limited | Duchenne Muscular Dystrophy, An inherited disease causing muscle degeneration., Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | | | | |
2015-001967-38: A research project for boys with Duchenne muscular dystrophy aged 5-9 years. It aims to find out how safe and well tolerated different doses of the experimental medicine are, when given by mouth after breakfast and evening meal. |
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| Ongoing | 1 | 24 | Europe | SMT C1100 (F5; nanosuspension), SMT C1100 (F6; Powder for Oral Suspension), SMT C1100 (F5), SMT C1100 (F6), Oral suspension, Powder for oral suspension | Summit (Oxford) Limited | Duchenne muscular dystrophy (DMD), A progressive, lethal muscle wasting disease, characterised by a generalised weakness and progressive loss of muscle strength; cardiac and respiratory difficulties present, leading to death, Diseases [C] - Musculoskeletal Diseases [C05] | | | | |
| Elevidys (delandistrogene moxeparvovec-rokl) / Sarepta Therap |
2020-002372-13: Study to evaluate the Safety and Efficacy of SRP-90001 in non ambulatory and ambulatory Subjects with Duchenne Muscular Dystrophy Etude destinée à évaluer la sécurité d’emploi et l’efficacité du SRP9001 chez des patients non ambulatoires et ambulatoires atteints de dystrophie musculaire de Duchenne |
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| Not yet recruiting | 3 | 148 | Europe | Corticosteroide, NA, SRP-9001, Solution for infusion | Sarepta Therapeutics, Inc., SAREPTA THERAPEUTICS, INC., Sarepta Therapeutics, Inc. | Duchenne Muscular Dystrophy Dystrophie musculaire de Duchenne, Duchenne Muscular Dystrophy Dystrophie musculaire de Duchenne, Diseases [C] - Musculoskeletal Diseases [C05] | | | | |
2019-003374-91: Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular Dystrophy Étude visant à évaluer la sécurité d’emploi et l’efficacité du SRP-9001 chez des sujets atteints de dystrophie musculaire de Duchenne |
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| Not yet recruiting | 3 | 120 | Europe | NA, SRP-9001, Solution for infusion | Sarepta Therapeutics, Inc., SAREPTA THERAPEUTICS, INC., Sarepta Therapeutics, Inc., | Duchenne Muscular Dystrophy Dystrophie musculaire de Duchenne, Duchenne Muscular Dystrophy Dystrophie musculaire de Duchenne, Diseases [C] - Musculoskeletal Diseases [C05] | | | | |
EMBARK, NCT05096221: A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) |
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| Active, not recruiting | 3 | 126 | Europe, Japan, US, RoW | delandistrogene moxeparvovec, SRP-9001, delandistrogene moxeparvovec-rokl, ELEVIDYS, placebo | Sarepta Therapeutics, Inc., Hoffmann-La Roche | Duchenne Muscular Dystrophy | 10/23 | 11/24 | | |
ENVISION, NCT05881408: A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD) |
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| Recruiting | 3 | 148 | Europe, Japan, US, RoW | delandistrogene moxeparvovec, SRP-9001, delandistrogene moxeparvovec-rokl, ELEVIDYS, placebo | Sarepta Therapeutics, Inc., Hoffmann-La Roche | Duchenne Muscular Dystrophy | 01/26 | 01/27 | | |
EXPEDITION, NCT05967351: A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study |
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| Enrolling by invitation | 3 | 400 | Europe, US | delandistrogene moxeparvovec, SRP-9001, delandistrogene moxeparvovec-rokl, ELEVIDYS | Sarepta Therapeutics, Inc., Hoffmann-La Roche | Duchenne Muscular Dystrophy | 11/30 | 11/30 | | |
2022-000691-19: Study to assess the safety and expression of RO7494222 (SRP-9001) in participants under the age of 4 with Duchenne muscular dystrophy Estudio para evaluar la seguridad y la expresión de RO7494222 (SRP-9001) en sujetos menores de cuatro años con Distrofia Muscular de Duchenne |
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| Not yet recruiting | 2 | 21 | Europe | delandistrogene moxeparvovec, RO7494222 (SRP-9001), Solution for infusion | F. Hoffmann-La Roche Ltd, F. HOFFMANN - LA ROCHE LTD., F. Hoffmann-La Roche Ltd | Duchenne Muscular Dystrophy Distrofia muscular de Duchenne, Duchenne Muscular Dystrophy Distrofia muscular de Duchenne, Diseases [C] - Musculoskeletal Diseases [C05] | | | | |
ENVOL, NCT06128564: 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) |
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| Recruiting | 2 | 21 | Europe | delandistrogene moxeparvovec, SRP-9001, ELEVIDYS, RO7494222, delandistrogene moxeparvovec-rokl | Hoffmann-La Roche, Sarepta Therapeutics, Inc. | Duchenne Muscular Dystrophy | 11/32 | 11/32 | | |
NCT03769116 / 2021-000078-27: A Randomized, Double-blind, Placebo-controlled Study of Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy (DMD) |
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| Completed | 1/2 | 41 | US | delandistrogene moxeparvovec, SRP-9001, delandistrogene moxeparvovec-rokl, ELEVIDYS, placebo | Sarepta Therapeutics, Inc., Sarepta Therapeutics, Inc. | Muscular Dystrophy, Duchenne | 12/20 | 08/23 | | |
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| Completed | 1/2 | 4 | US | delandistrogene moxeparvovec, SRP-9001, delandistrogene moxeparvovec-rokl, ELEVIDYS | Sarepta Therapeutics, Inc., Sarepta Therapeutics, Inc. | Duchenne Muscular Dystrophy | 04/23 | 04/23 | | |
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ENDEAVOR, NCT04626674: A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) |
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| Active, not recruiting | 1 | 55 | US | delandistrogene moxeparvovec, SRP-9001, delandistrogene moxeparvovec-rokl, ELEVIDYS | Sarepta Therapeutics, Inc., Hoffmann-La Roche | Muscular Dystrophy, Duchenne | 07/24 | 07/26 | | |
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ENDURE, NCT06270719: An Observational Study Comparing Delandistrogene Moxeparvovec With Standard of Care in Participants With Duchenne Muscular Dystrophy |
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| Recruiting | N/A | 500 | US | delandistrogene moxeparvovec, SRP-9001, delandistrogene moxeparvovec-rokl, ELEVIDYS, Standard of Care, Chronic glucocorticoid treatment | Sarepta Therapeutics, Inc. | Duchenne Muscular Dystrophy | 12/29 | 12/38 | | |
| Amondys 45 (casimersen) / Sarepta Therap |
| Terminated | 3 | 171 | Europe, Canada, US, RoW | Casimersen, SRP-4045, AMONDYS 45, Golodirsen, SRP-4053, VYONDYS 53 | Sarepta Therapeutics, Inc. | Duchenne Muscular Dystrophy | 07/23 | 07/23 | | |
| Active, not recruiting | 3 | 228 | Europe, Canada, US, RoW | SRP-4045, Casimersen, AMONDYS 45, SRP-4053, Golodirsen, VYONDYS 53, Placebo | Sarepta Therapeutics, Inc. | Duchenne Muscular Dystrophy | 10/25 | 10/25 | | |
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NCT04179409: A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 in Subjects With DuchenneMuscular Dystrophy Carrying Eligible DMD Duplications. |
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| Completed | 2 | 3 | US | Amondys 45, SRP-4045, Casimersen, Exondys 51, Eteplirsen, AVI-4658, Vyondys 53, SRP-4053, Golodirsen | Kevin Flanigan, Sarepta Therapeutics, Inc. | Duchenne Muscular Dystrophy | 09/21 | 09/23 | | |
| Exondys 51 (eteplirsen) / Sarepta Therap |
NCT03992430 / 2018-001762-42: A Study to Compare Safety and Efficacy of a High Dose of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON) |
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| Active, not recruiting | 3 | 160 | Europe, US, RoW | Eteplirsen, AVI-4658, EXONDYS 51, EXONDYS | Sarepta Therapeutics, Inc., Sarepta Therapeutics, Inc. | Muscular Dystrophy, Duchenne | 11/24 | 11/24 | | |
MOMENTUM, NCT04004065 / 2019-000601-77: Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Efficacy (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment |
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| Active, not recruiting | 2 | 62 | Europe, Canada, US | Vesleteplirsen, SRP-5051 | Sarepta Therapeutics, Inc. | Duchenne Muscular Dystrophy | 10/23 | 01/29 | | |
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| Galgt2 gene therapy / Nationwide Children's, Sarepta Therap |
NCT03333590: Gene Transfer Clinical Trial to Deliver rAAVrh74.MCK.GALGT2 for Duchenne Muscular Dystrophy |
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| Active, not recruiting | 1/2 | 2 | US | rAAVrh74.MCK.GALGT2 | Kevin Flanigan | Duchenne Muscular Dystrophy | 11/20 | 10/23 | | |
| LYS-SAF302 / Lysogene, Sarepta Therap |
| Active, not recruiting | 2/3 | 20 | Europe, US | LYS-SAF302, AAVrh10-h.SGSH | LYSOGENE | Mucopolysaccharidosis Type IIIA | 03/22 | 03/22 | | |
| GNT 0004 / Sarepta Therap |
2020-002093-27: A clinical study in 3 parts with a microdystrophin (called GNT0004), a new gene therapy in boys with Duchenne disease who can still walk. The study will start with finding the proper treatment dose (part 1).After that, a comparative study versus placebo will start to assess the safety and the effectiveness of the proper dose of this therapy (part 2).In the end, a follow up period will continue to investigate the treatment safety and efficacy over longer time (part 3). |
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| Not yet recruiting | 2/3 | 51 | Europe | rAAV8-hMD1, GNT0004, Concentrate for solution for infusion | Genethon, Genethon | Duchenne Muscular Dystrophy, Duchenne's disease, Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | | | | |
| bidridistrogene xeboparvovec (SRP-9003) / Sarepta Therap |
NCT06246513: A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4 (EMERGENE) |
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| Recruiting | 3 | 15 | US | SRP-9003, scAAVrh74.MHCK7.hSGCB, bidridistrogene xeboparvovec, Glucocorticoid, Prednisone or equivalent | Sarepta Therapeutics, Inc. | Limb-girdle Muscular Dystrophy | 01/25 | 11/29 | | |
NCT03652259: Gene Delivery Clinical Trial of SRP-9003 (Bidridistrogene Xeboparvovec) for Participants With Limb-Girdle Muscular Dystrophy, Type 2E (LGMD2E) (Beta-Sarcoglycan Deficiency) |
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| Active, not recruiting | 1/2 | 6 | US | SRP-9003, LGMD2E vector, bidridistrogene xeboparvovec | Sarepta Therapeutics, Inc. | Limb-Girdle Muscular Dystrophy, Type 2E | 02/27 | 02/27 | | |
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) |
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| Active, not recruiting | 1 | 6 | US | SRP-9003, scAAVrh74.MHCK7.hSGCB | Sarepta Therapeutics, Inc. | Limb Girdle Muscular Dystrophy | 08/28 | 08/28 | | |
| Pompe Disease therapeutic / Sarepta Therap |
| No trials found |
| STRX-240 / Sarepta Therap, StrideBio |
| No trials found |
| Gene Targets / Codiak, Sarepta Therap |
| No trials found |
| CNS-1 program / Sarepta Therap, Lacerta Therap |
| No trials found |
| STRX-210 / Sarepta Therap, StrideBio |
| No trials found |
| STRX-230 / Sarepta Therap, StrideBio |
| No trials found |
| Emery-Dreifuss muscular dystrophy therapeutic / Sarepta Therap, Columbia University |
| No trials found |
| scAAV1.tMCK.NTF3 / Sarepta Therap |
| No trials found |
| multiple sclerosis therapeutic / Sarepta Therap, University of Florida |
| No trials found |
| AAV based Rett syndrome therapy / Sarepta Therap, University of Massachusetts Boston |
| No trials found |
