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Synchronisé depuis ClinicalTrials.gov — NCT06138639

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27
MoléculeIndication / populationPhaseNCTTitreStatut
Mycophenolate Mofetil Lupus Phase 2 NCT05538208 The Pediatric Lupus Nephritis Mycophenolate Mofetil (PLUMM) Study RECRUITING
Biopsy Procedure Cancer Phase 1/2 NCT05099003 A Study of the Drug Selinexor With Radiation Therapy in Patients With Newly-Diagnosed Diffuse Intrinsic Pontine (DIPG) Glioma and High-Grade Glioma (HGG) RECRUITING
Biopsy Procedure Cancer Phase 2 NCT06064097 A Study Using Nivolumab, in Combination With Chemotherapy Drugs to Treat Nasopharyngeal Carcinoma (NPC) RECRUITING
Observation Study in Patients Age 0-5 Years With LAMA2-related Congenital Muscular Dystrophy Myopathies À vérifier NCT06503367 Observation Study in Patients Age 0-5 Years With LAMA2-related Congenital Muscular Dystrophy RECRUITING
The Baby Duchenne Study: Characterizing Developmental and Clinical Outcomes in the First Three Years in Children With Duchenne Muscular Dystrophy Myopathies À vérifier NCT07092540 The Baby Duchenne Study: Characterizing Developmental and Clinical Outcomes in the First Three Years in Children With Duchenne Muscular Dystrophy RECRUITING
GRASP-01-002 — Defining Endpoints in Becker Muscular Dystrophy Myopathies À vérifier NCT05257473 GRASP-01-002 — Defining Endpoints in Becker Muscular Dystrophy ACTIVE_NOT_RECRUITING
GRASP-01-003 — LGMD R1 Natural History Study Myopathies À vérifier NCT05618080 GRASP-01-003 — LGMD R1 Natural History Study ACTIVE_NOT_RECRUITING
Biospecimen Collection Cancer Phase 3 NCT05675410 A Study to Compare Standard Therapy to Treat Hodgkin Lymphoma to the Use of Two Drugs, Brentuximab Vedotin and Nivolumab RECRUITING
Prednisolone Myopathies Phase 4 NCT05412394 Once Weekly Infant Corticosteroid Trial for DMD RECRUITING
Bone Scan Cancer Phase 2/3 NCT05691478 A Study to Test the Addition of the Drug Cabozantinib to Chemotherapy in Patients With Newly Diagnosed Osteosarcoma SUSPENDED
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
SGT-003 Myopathies Phase 1/2 NCT06138639 A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE) RECRUITING
ETX101 Épilepsie Phase 1/2 NCT05419492 ENDEAVOR — A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome RECRUITING
RGX-202 Myopathies Phase 2/3 NCT05693142 AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD) ACTIVE_NOT_RECRUITING
SAT-3247 Myopathies Phase 2 NCT07287189 BASECAMP — Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients RECRUITING
Sevasemten 10 mg Myopathies Phase 2 NCT05291091 Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy (GRAND CANYON) ACTIVE_NOT_RECRUITING
Sevasemten Myopathies Phase 2 NCT06066580 MESA — Open-Label Extension of EDG-5506 in Participants With Becker Muscular Dystrophy ENROLLING_BY_INVITATION
DYNE-251 Myopathies Phase 1/2 NCT05524883 DELIVER — Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping ACTIVE_NOT_RECRUITING
delpacibart zotadirsen Myopathies À vérifier NCT07250737 Managed Access Program for Del-zota in Participants With DMD Mutations Amenable to Exon 44 Skipping AVAILABLE
zorevunersen Épilepsie Phase 3 NCT06872125 A Double-blind Study Evaluating the Efficacy, Safety, and Tolerability of Zorevunersen in Patients With Dravet Syndrome RECRUITING
S230815- Starting dose A Épilepsie Phase 1/2 NCT07227857 KANDLE — A First-in-human Study of S230815 in Pediatric Participants With KCNT1-related Developmental and Epileptic Encephalopathy RECRUITING
Clemizole HCl Épilepsie Phase 3 NCT04462770 ARGUS — A Study of EPX-100 (Clemizole Hydrochloride) in Participants With Dravet Syndrome RECRUITING
Limb Girdle Muscular Dystrophy Type 2E Recruitment Study Myopathies À vérifier NCT03492346 Limb Girdle Muscular Dystrophy Type 2E Recruitment Study UNKNOWN
Sevasemten Dose 1 Myopathies Phase 2 NCT06100887 FOX — Phase 2 Study of EDG-5506 in Children and Adolescents With Duchenne Muscular Dystrophy Previously Treated With Gene Therapy ACTIVE_NOT_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
Rimegepant/BHV3000 Migraine Phase 3 NCT04649242 Randomized Study in Children and Adolescents With Migraine: Acute Treatment RECRUITING
Ataluren Myopathies Phase 2 NCT01009294 Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD) TERMINATED