Boston Children's Hospital
Boston Children's Hospital
Boston, Massachusetts, United States20 results · Page 1 of 1
Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| PediRISE Feasibility | childhood malignant neoplasm, neoplasm | Recruiting | Not applicable | 2026-10-05 | NCT06283251 | |
| The iCat2, GAIN (Genomic Assessment Informs Novel Therapy) Consortium Study | Pediatric Solid Tumor | Active, not recruiting | Not stated | 2026-09-21 | NCT02520713 | |
| Buprenorphine for the Management of Pain in Children With Sickle Cell Disease | Acute Pain, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Hemoglobin SC Disease, Neurologic Manifestations, Pain, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Vaso-Occlusive Crises, anemia, familial hemolytic anemia, hematologic disorder, hemolytic anemia, hereditary disease, inherited hemoglobinopathy, sickle cell disease | Not yet recruiting | Phase 1 | 2026-09-02 | NCT07799571 | |
| A Pilot Study to Evaluate the Feasibility of Post-Hematopoietic Stem Cell Transplant Prophylaxis With Decitabine Combined With Filgrastim for Children and Young Adults With AML, MDS and Related Myeloid Malignancies | Bone Marrow Failure Disorders, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Infant, Newborn, Diseases, Neoplasms by Histologic Type, acute myeloid leukemia, bone marrow disorder, hematologic disorder, leukemia, myelodysplastic syndrome, myeloid leukemia, myeloid neoplasm, neoplasm | Active, not recruiting | Phase 2 | 2026-08-20 | NCT05796570 | |
| HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome | Abnormalities, Multiple, Angelman syndrome, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Imprinting Disorders, central nervous system disorder, chromosomal disorder, hereditary disease, movement disorder, nervous system disorder | Active, not recruiting | Phase 1 / Phase 2 | 2026-08-20 | NCT05127226 | |
| COMparison Between Anakinra and Tocilizumab in NORSE - "COMBAT-NORSE" | Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Status Epilepticus, febrile infection-related epilepsy syndrome, nervous system disorder, new-onset refractory status epilepticus, visual epilepsy | Not yet recruiting | Phase 3 | 2026-08-19 | NCT07281027 | |
| A Phase 2 Study Evaluating Safety and Tolerability of RCT2100 (CFTR mRNA) in Healthy Participants and in Participants With CF | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Infant, Newborn, Diseases, cystic fibrosis, hereditary disease, lung disorder, pancreas disorder, respiratory system disorder | Active, not recruiting | Phase 2 | 2026-08-19 | NCT06237335 | |
| Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects With RPS19 Deficient Diamond-Blackfan Anemia | Bone Marrow Failure Disorders, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Diamond-Blackfan anemia, Hemic and Lymphatic Diseases, anemia, aplastic anemia, bone marrow disorder, hematologic disorder, hereditary disease, inherited aplastic anemia, pure red-cell aplasia | Recruiting | Phase 1 | 2026-07-21 | NCT07476183 | |
| Prevention of Bleeding in Patients With Moderate and Severe Hemophilia A Playing Sports: A Comparison Between Factor VIII and Emicizumab Prophylaxis | 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 | Recruiting | Not stated | 2026-07-02 | NCT05022459 | |
| Anxiety Surrounding Supracondylar Pin Removal in Children: A Randomized Controlled Trial | anxiety, anxiety disorder, psychiatric disorder | Enrolling by invitation | Not applicable | 2026-06-30 | NCT05501834 | |
| BElumosudil for Bronchiolitis Obliterans Prevention/Therapy (BEBOP) | Bronchiolitis Obliterans, bronchial disorder, bronchiolitis, bronchiolitis obliterans syndrome, bronchitis, cryptogenic organizing pneumonia, graft versus host disease, immune system disorder, lung disorder, obstructive lung disease, respiratory system disorder | Recruiting | Phase 2 | 2026-06-10 | NCT05922761 | |
| Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program | carnitine-acylcarnitine translocase deficiency, mitochondrial trifunctional protein deficiency, very long chain acyl-CoA dehydrogenase deficiency | Active, not recruiting | Not stated | 2026-06-03 | NCT04632953 | |
| A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace) | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, hereditary disease, inborn disorder of amino acid metabolism, inborn errors of metabolism, metabolic disease, nervous system disorder, phenylketonuria | Recruiting | Not stated | 2026-03-25 | NCT05813678 | |
| A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade) | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, hereditary disease, inborn disorder of amino acid metabolism, inborn errors of metabolism, metabolic disease, nervous system disorder, phenylketonuria | Recruiting | Not stated | 2026-03-24 | NCT06305234 | |
| Spastic Paraplegia - Centers of Excellence Research Network | Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Spastic Paraplegia Type 4, central nervous system malformation, hereditary disease, hereditary motor and sensory neuropathy, hereditary spastic paraplegia, hereditary spastic paraplegia 4, hereditary spastic paraplegia 5A, inherited neurodegenerative disorder, lateral sclerosis, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, peripheral nervous system disorder, polyneuropathy | Recruiting | Not stated | 2026-03-18 | NCT06553976 | |
| Bevacizumab Treatment For Type 1 ROP | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Infant, Newborn, Diseases, Infant, Premature, Diseases, eye disorder, retinal disorder, retinopathy of prematurity | Active, not recruiting | Phase 2 | 2026-02-11 | NCT04634578 | |
| A Long-term Follow-up Study in Patients Who Received BEAM-101 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, anemia, familial hemolytic anemia, hematologic disorder, hemolytic anemia, hereditary disease, inherited hemoglobinopathy, sickle cell disease | Recruiting | Not stated | 2026-01-28 | NCT07373639 | |
| Promoting Resilience in Early Survivorship Among Adolescents and Young Adults With Cancer | anxiety, anxiety disorder, psychiatric disorder | Recruiting | Not applicable | 2025-12-29 | NCT06038318 | |
| BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, anemia, familial hemolytic anemia, hematologic disorder, hemolytic anemia, hereditary disease, inherited hemoglobinopathy, sickle cell disease | Active, not recruiting | Phase 1 / Phase 2 | 2025-12-16 | NCT05456880 | |
| Restarting Triple Therapy With Robust Monitoring for Adverse Events (RETRIAL) | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Infant, Newborn, Diseases, cystic fibrosis, hereditary disease, lung disorder, pancreas disorder, respiratory system disorder | Recruiting | Not stated | 2025-11-03 | NCT06683092 |