Phoenix Children's Hospital
Phoenix Children's Hospital
Phoenix, Arizona, United States95 results · Page 1 of 4
Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, X-linked disease, blood coagulation disease, coagulation protein disease, hematologic disorder, hemophilia B, hemorrhagic disease, hereditary disease, inherited blood coagulation disorder | Recruiting | Phase 1 / Phase 2 | 2026-10-08 | NCT06379789 | |
| Silmitasertib (CX-4945) in Combination With Chemotherapy for Relapsed Refractory Solid Tumors | Ewing sarcoma, Myosarcoma, Neoplasms by Histologic Type, Neoplasms, Adipose Tissue, Neoplasms, Bone Tissue, Neoplasms, Connective and Soft Tissue, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Neuroectodermal Tumors, Primitive, Neuroectodermal Tumors, Primitive, Peripheral, connective tissue neoplasm, epithelial neoplasm, liposarcoma, myomatous neoplasm, neoplasm, neuroblastoma, neuroepithelial neoplasm, osteosarcoma, primitive neuroectodermal tumor, rhabdomyosarcoma, sarcoma | Recruiting | Phase 1 / Phase 2 | 2026-10-06 | NCT06541262 | |
| A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Atrophy, Spinal, Type II, Neurologic Manifestations, Neuromuscular Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Spinal Muscular Atrophies of Childhood, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, proximal spinal muscular atrophy, spinal cord disorder, spinal muscular atrophy, spinal muscular atrophy, type III | Recruiting | Phase 2 | 2026-10-02 | NCT07047144 | |
| A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, X-linked disease, connective tissue disorder, cutaneous mucinosis, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, inherited neurodegenerative disorder, intellectual disability, lysosomal storage disease, metabolic disease, mucopolysaccharidosis, mucopolysaccharidosis type 2, nervous system disorder | Active, not recruiting | Phase 3 | 2026-10-02 | NCT04573023 | |
| A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous Emicizumab in Participants From Birth to 12 Months of Age With Hemophilia A Without Inhibitors | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, blood coagulation disease, coagulation protein disease, hematologic disorder, hemophilia A, hemorrhagic disease, hereditary disease, inherited blood coagulation disorder, severe hemophilia A | Active, not recruiting | Phase 3 | 2026-10-01 | NCT04431726 | |
| A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Regularly Transfused, Followed by a 5-Year Extension Period | Hemic and Lymphatic Diseases, Nutritional and Metabolic Diseases, anemia, hematologic disorder, metabolic disease, pyruvate kinase deficiency of red cells | Active, not recruiting | Phase 3 | 2026-09-28 | NCT05144256 | |
| Metastatic Ewing's Trial Testing Schedule Enhancement to Improve Outcomes | Ewing sarcoma, Neoplasms by Histologic Type, Neoplasms, Bone Tissue, Neoplasms, Connective and Soft Tissue, connective tissue neoplasm, neoplasm, osteosarcoma, sarcoma | Recruiting | Phase 1 | 2026-09-25 | NCT07194044 | |
| Open-Label Study to Assess Safety and Time to Catheter-related Bloodstream Infections (CRBSI) in Subjects From Birth to < 18 Years of Age | Catheter-Related Infections, Infections | Recruiting | Phase 4 | 2026-09-25 | NCT06714864 | |
| A Trial Comparing Unrelated Donor BMT With IST for Pediatric and Young Adult Patients With Severe Aplastic Anemia (TransIT, BMT CTN 2202) | Bone Marrow Failure Disorders, Hemic and Lymphatic Diseases, anemia, aplastic anemia, bone marrow disorder, hematologic disorder | Active, not recruiting | Phase 3 | 2026-09-25 | NCT05600426 | |
| HCRN Endoscopic Versus Shunt Treatment of Hydrocephalus in Infants | brain disorder, central nervous system disorder, hydrocephalus, nervous system disorder | Active, not recruiting | Not applicable | 2026-09-25 | NCT04177914 | |
| A Study of Selexipag as Add-On Treatment to Standard of Care in Children With Pulmonary Arterial Hypertension | cardiovascular disorder, hypertensive disorder, lung disorder, pulmonary hypertension, respiratory system disorder, vascular disorder | Active, not recruiting | Phase 3 | 2026-09-25 | NCT04175600 | |
| Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMIT | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne muscular dystrophy, Muscular Disorders, Atrophic, X-linked disease, hereditary disease, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neuromuscular disease | Recruiting | Not stated | 2026-09-24 | NCT06564974 | |
| Campath/Fludarabine/Melphalan Transplant Conditioning for Non-Malignant Diseases | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Nutritional and Metabolic Diseases, hematologic disorder, hereditary disease, inherited hemoglobinopathy, metabolic disease | Recruiting | Phase 1 / Phase 2 | 2026-09-16 | NCT00920972 | |
| A Study to Evaluate the Safety and Efficacy of Ruxolitinib Cream in Pediatric Participants With Nonsegmental Vitiligo | NonSegmental Vitiligo | Recruiting | Phase 3 | 2026-09-14 | NCT06548360 | |
| A Trial of Dabrafenib, Trametinib and Hydroxychloroquine for Patients With Recurrent LGG or HGG With a BRAF Aberration | Neoplasms by Histologic Type, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, epithelial neoplasm, glioma, neoplasm, neuroepithelial neoplasm, primitive neuroectodermal tumor | Active, not recruiting | Phase 1 / Phase 2 | 2026-09-11 | NCT04201457 | |
| Prenatally-initiated Psychological Intervention for Mothers of Infants With Congenital Heart Disease (CHD) | Cardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, cardiovascular disorder, congenital heart disease, heart disorder | Recruiting | Not applicable | 2026-09-10 | NCT07582861 | |
| BOND: Direct Breastfeeding to Enhance Maternal and Infant Health in Congenital Heart Disease | Cardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, cardiovascular disorder, congenital heart disease, heart disorder | Recruiting | Not applicable | 2026-09-10 | NCT07582848 | |
| I-DECIDE After Bronchiolitis Hospitalization | Bronchiolitis Acute | Recruiting | Not applicable | 2026-09-10 | NCT07243652 | |
| Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA) | central nervous system disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophy | Active, not recruiting | Phase 4 | 2026-09-09 | NCT05232929 | |
| Safety and Efficacy Study of Cenobamate in Pediatric Subjects 2-17 Years of Age With Partial-onset (Focal) Seizures | Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, brain disorder, central nervous system disorder, epilepsy, focal epilepsy, nervous system disorder, visual epilepsy | Active, not recruiting | Phase 3 | 2026-09-09 | NCT05067634 | |
| A Study to Assess the Efficacy and Safety of Daily OM-85 in Young Children With Recurrent Wheezing | Pathological Conditions, Signs and Symptoms, Respiratory Sounds, Signs and Symptoms, Signs and Symptoms, Respiratory | Active, not recruiting | Phase 2 | 2026-09-08 | NCT05857930 | |
| Pediatric Influence of Cooling Duration on Efficacy in Cardiac Arrest Patients (P-ICECAP) | Body Temperature Changes, Coma, Consciousness Disorders, Hypothermia, Hypoxia, Hypoxia, Brain, Neurobehavioral Manifestations, Neurologic Manifestations, Out-of-Hospital Cardiac Arrest, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Signs and Symptoms, Respiratory, Unconsciousness, brain disorder, brain hypoxia - ischemia, brain ischemia, cardiac arrest, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, heart disorder, nervous system disorder, vascular disorder | Recruiting | Not applicable | 2026-09-08 | NCT05376267 | |
| A Phase I/II Study of Trametinib and Azacitidine for Patients With Newly Diagnosed Juvenile Myelomonocytic Leukemia | CBL-related disorder, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Neoplasms by Histologic Type, Neoplasms, Nerve Tissue, bone marrow disorder, hematologic disorder, hereditary disease, hereditary neoplastic syndrome, inherited neurodegenerative disorder, juvenile myelomonocytic leukemia, leukemia, myelodysplastic/myeloproliferative disease, myeloid leukemia, neoplasm, nerve sheath neoplasm, nervous system disorder, neurocutaneous syndrome, neurodegenerative disease, neurofibroma, neurofibromatosis, neurofibromatosis type 1, neuromuscular disease, peripheral nervous system disorder | Recruiting | Phase 1 / Phase 2 | 2026-09-04 | NCT05849662 | |
| BBD Longitudinal Study of Osteogenesis Imperfecta | Bone Diseases, Developmental, Collagen Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Skin and Connective Tissue Diseases, bone disorder, brittle bone disorder, connective tissue disorder, hereditary disease, musculoskeletal system disorder, osteochondrodysplasia, osteogenesis imperfecta, osteogenesis imperfecta type 5 | Recruiting | Not stated | 2026-09-01 | NCT02432625 | |
| A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne muscular dystrophy, Muscular Disorders, Atrophic, X-linked disease, hereditary disease, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neuromuscular disease | Active, not recruiting | Phase 3 | 2026-08-28 | NCT05126758 |