Children's Hospital of Philadelphia

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Synchronisé depuis ClinicalTrials.gov — NCT06839976

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Essais cliniques associés

Molécule	Indication / population	Phase	NCT	Titre	Statut
Eteplirsen	Myopathies	À vérifier	NCT06606340	EVOLVE — A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice	ENROLLING_BY_INVITATION
Carisbamate	Épilepsie	Phase 3	NCT05219617	Investigate Efficacy and Safety of Carisbamate as Adjunctive Treatment for Seizures Associated With LGS in Children and Adults	RECRUITING
EXCOR Active Driving System for Pediatric VAD	Insuffisance cardiaque	Non applicable	NCT05610787	EXCOR Active Driving System for the EXCOR Pediatric VAD IDE Study	ENROLLING_BY_INVITATION
zagociguat 15mg	Myopathies	Phase 2	NCT06961344	An Open-label Extension Safety Study of MELAS Patients Who Completed TIS6463-203 (PRIZM)	TERMINATED
INCB000928	Myopathies	Phase 2	NCT05090891	To Assess the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans Progressiva	RECRUITING
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
Empagliflozin	Maladie rénale chronique	Phase 3	NCT07107945	A Study to Find Out How EMPAgliflozin is Tolerated and if it Helps Children and Adolescents With Chronic KIDNEY Disease (EMPA-KIDNEY® Kids)	ACTIVE_NOT_RECRUITING
zagociguat 15mg	Accident vasculaire cérébral	Phase 2	NCT06402123	PRIZM — A Phase 2b Study of Zagociguat in Patients With MELAS	COMPLETED
Phase 1 Dose Level 1	SEP	Phase 1/2	NCT06839456	Phase 1/2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab/CD19 Depleted HSCT	RECRUITING
Ataluren	Myopathies	Phase 3	NCT01247207	Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)	TERMINATED
AZD6244	Cancer	Phase 1/2	NCT01362803	AZD6244 Hydrogen Sulfate for Children With Nervous System Tumors	ACTIVE_NOT_RECRUITING
Zavegepant	Migraine	Phase 1	NCT06995729	A Study to Learn About the Study Medicine Called Zavegepant (PF-07930207) in Children With a History of Migraine	RECRUITING
CART19	Lupus	Phase 1/2	NCT06839976	CD19-Directed Chimeric Antigen Receptor Autologous T Cells (CART19) for Lupus	RECRUITING
CliniMACS Cell Processing System for TCRαβ + T Cell and CD45RA Depleted Peripheral Stem Cell Addback	SEP	Non applicable	NCT03810196	CD45RA Depleted Peripheral Stem Cell Addback for Viral or Fungal Infections Post TCRαβ/CD19 Depleted HSCT	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
Soticlestat	Épilepsie	Phase 3	NCT05163314	A Study of Soticlestat as an Add-on Therapy in Children and Adults With Dravet Syndrome or Lennox-Gastaut Syndrome	TERMINATED
Soticlestat	Épilepsie	Phase 3	NCT04938427	A Study of Soticlestat as an Add-on Therapy in Children, Teenagers, and Adults With Lennox-Gastaut Syndrome	COMPLETED
Biopsy Procedure	Cancer	Phase 2	NCT06064097	A Study Using Nivolumab, in Combination With Chemotherapy Drugs to Treat Nasopharyngeal Carcinoma (NPC)	RECRUITING
CABA-201	Lupus	Phase 1/2	NCT06121297	RESET-SLE: A Phase 1/2 Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects With Active Systemic Lupus Erythematosus	RECRUITING
LY3295668 Erbumine	Épilepsie	Phase 1	NCT04106219	A Study of LY3295668 Erbumine in Participants With Relapsed/Refractory Neuroblastoma	ACTIVE_NOT_RECRUITING
Clemizole HCl	Épilepsie	Phase 3	NCT05066217	An Efficacy and Safety Study of Clemizole HCl in Patients With Lennox-Gastaut Syndrome	RECRUITING
NS-050/NCNP-03	Myopathies	Phase 1/2	NCT06053814	NS-050/NCNP-03 in Boys With DMD (Meteor50)	ACTIVE_NOT_RECRUITING
GPC2 CAR T cells	Cancer	Phase 1	NCT05650749	GPC2 CAR T Cells for Relapsed or Refractory Neuroblastoma and Metastatic Retinoblastoma	ENROLLING_BY_INVITATION
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
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
SGT-003	Myopathies	Phase 1/2	NCT06138639	A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)	RECRUITING
Lidocaine 4% Topical Application Cream [LMX 4]	Migraine	Phase 3	NCT03526874	Occipital Blocks for Acute Migraine	COMPLETED
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
EXCEL — A Study to Check Liver Health in Boys With XLMTM, a Serious Genetic Muscle Condition	Myopathies	À vérifier	NCT06581146	EXCEL — A Study to Check Liver Health in Boys With XLMTM, a Serious Genetic Muscle Condition	RECRUITING
Rimegepant (PF-07899801)	Migraine	Phase 3	NCT04743141	Long-term Safety Study of Rimegepant in Pediatric Subjects for the Acute Treatment of Migraine	RECRUITING
Amitriptyline	Migraine	Non applicable	NCT05889624	Responding With Evidence and Access for Childhood Headaches	RECRUITING
Rimegepant/BHV3000	Migraine	Phase 3	NCT04649242	Randomized Study in Children and Adolescents With Migraine: Acute Treatment	RECRUITING