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ClinicalTrials.gov public records Last synced Nov 8, 2026, 10:10 AM EST

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Showing 3049–3072 of 10,825 matching trials from the live ClinicalTrials.gov search.

Filters: Condition: Developmental Defect Clear all

Conditions: Tuberous Sclerosis, Autism Disorder, Intellectual Disability
Interventions: Not listed
Lead sponsor: Boston Children's Hospital; Other
Eligibility: 18 Months and older
Enrollment: 205 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2015 – 2026
U.S. locations: 6
States / cities: Birmingham, Alabama • Los Angeles, California • Palo Alto, California + 3 more

Conditions: Congenital Bleeding Disorder, Haemophilia A
Interventions: turoctocog alfa pegol; Drug
Lead sponsor: Novo Nordisk A/S; Industry
Eligibility: 12 Years and older · Male only
Enrollment: 186 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2018
U.S. locations: 31
States / cities: Phoenix, Arizona • Long Beach, California • Sacramento, California + 26 more

Conditions: Neurofibromatosis, MPNST
Interventions: MRI, FDG-PET/CT scans, [18F]-FLT-PET/CT scans; Procedure · Drug
Lead sponsor: National Cancer Institute (NCI); NIH
Eligibility: 10 Years and older
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2019
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Friedreich Ataxia
Interventions: Elamipretide; Drug
Lead sponsor: Children's Hospital of Philadelphia; Other
Eligibility: 16 Years and older
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2024
U.S. locations: 1
States / cities: Philadelphia, Pennsylvania

Conditions: Sarcoma, Neuroblastoma, Wilms Tumor, Osteosarcoma, Brain Tumor
Interventions: Pazopanib (GW786034); Drug
Lead sponsor: National Cancer Institute (NCI); NIH
Eligibility: 1 Year to 25 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2011
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Glycogen Storage Disease Type II
Interventions: Not listed
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 8 Years and older
Enrollment: 61 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2006
U.S. locations: 3
States / cities: Washington D.C., District of Columbia • St Louis, Missouri • Seattle, Washington

Conditions: Retinitis Pigmentosa
Interventions: 400 µg Brimonidine Tartrate Implant, 200 µg Brimonidine Tartrate Implant, 100 µg Brimonidine Tartrate Implant, Sham (no implant); Drug · Other
Lead sponsor: Allergan; Industry
Eligibility: 18 Years and older
Enrollment: 21 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2010
U.S. locations: 1
States / cities: Arlington, Texas

Conditions: Respiratory Distress Syndrome, Newborn, Bronchopulmonary Dysplasia
Interventions: Noninvasive neurally adjusted ventilatory assist (NIV NAVA), Nasal intermittent positive pressure ventilation (NIPPV); Other
Lead sponsor: Arkansas Children's Hospital Research Institute; Other
Eligibility: Up to 12 Months
Enrollment: 15 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2018
U.S. locations: 1
States / cities: Little Rock, Arkansas

Conditions: Rett Syndrome
Interventions: Intravenous infusion; Procedure
Lead sponsor: The Cleveland Clinic; Other
Eligibility: 5 Years to 25 Years · Female only
Enrollment: 4 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2017
U.S. locations: 1
States / cities: Cleveland, Ohio

Conditions: Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Non-Hodgkin Lymphoma, Ann Arbor Stage IV Non-Hodgkin Lymphoma, Histiocytic Sarcoma, Juvenile Xanthogranuloma, Langerhans Cell Histiocytosis, Malignant Glioma, Recurrent Childhood Rhabdomyosarcoma, Recurrent Ependymoma, Recurrent Ewing Sarcoma, Recurrent Glioma, Recurrent Hepatoblastoma, Recurrent Langerhans Cell Histiocytosis, Recurrent Malignant Germ Cell Tumor, Recurrent Malignant Solid Neoplasm, Recurrent Medulloblastoma, Recurrent Neuroblastoma, Recurrent Non-Hodgkin Lymphoma, Recurrent Osteosarcoma, Recurrent Peripheral Primitive Neuroectodermal Tumor, Recurrent Primary Central Nervous System Neoplasm, Recurrent Rhabdoid Tumor, Recurrent Soft Tissue Sarcoma, Refractory Ewing Sarcoma, Refractory Glioma, Refractory Hepatoblastoma, Refractory Langerhans Cell Histiocytosis, Refractory Malignant Germ Cell Tumor, Refractory Malignant Solid Neoplasm, Refractory Medulloblastoma, Refractory Neuroblastoma, Refractory Non-Hodgkin Lymphoma, Refractory Osteosarcoma, Refractory Peripheral Primitive Neuroectodermal Tumor, Refractory Primary Central Nervous System Neoplasm, Refractory Rhabdoid Tumor, Refractory Rhabdomyosarcoma, Rhabdoid Tumor, Stage III Osteosarcoma AJCC v7, Stage III Soft Tissue Sarcoma AJCC v7, Stage IV Osteosarcoma AJCC v7, Stage IV Soft Tissue Sarcoma AJCC v7, Stage IVA Osteosarcoma AJCC v7, Stage IVB Osteosarcoma AJCC v7, Wilms Tumor
Interventions: Biopsy Procedure, Biospecimen Collection, Bone Marrow Aspiration and Biopsy, Bone Scan, Computed Tomography, Ensartinib, Erdafitinib, Ivosidenib, Laboratory Biomarker Analysis, Larotrectinib Sulfate, Magnetic Resonance Imaging, Mutation Carrier Screening, Olaparib, Palbociclib, Pharmacological Study, Positron Emission Tomography, Radionuclide Imaging, Samotolisib, Selpercatinib, Selumetinib Sulfate, Tazemetostat, Tipifarnib, Ulixertinib, Vemurafenib, X-Ray Imaging; Procedure · Drug · Other
Lead sponsor: National Cancer Institute (NCI); NIH
Eligibility: 12 Months to 21 Years
Enrollment: 1,377 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2027
U.S. locations: 168
States / cities: Birmingham, Alabama • Anchorage, Alaska • Mesa, Arizona + 126 more

Conditions: Revision Spine Surgery, Fusion of Spine, Cervical Fusion, Lumbar Fusion
Interventions: Ketamine Hydrochloride; Drug
Lead sponsor: AXIS Spine Center- a division of Northwest Specialty Hospital; Other
Eligibility: 18 Years to 75 Years
Enrollment: 40 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2024
U.S. locations: 1
States / cities: Coeur d'Alene, Idaho

Conditions: Nicotine Dependence
Interventions: tobacco abstinent contingent, non-contingent, control; Behavioral
Lead sponsor: Johns Hopkins University; Other
Eligibility: 18 Years to 50 Years · Female only
Enrollment: 210 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2010
U.S. locations: 1
States / cities: Baltimore, Maryland

Conditions: Sickle Cell Disease, Sickle Cell Crisis, Pain, Acute
Interventions: Lidocaine Iv, Placebo; Drug
Lead sponsor: Community Medical Center, Toms River, NJ; Other
Eligibility: 18 Years and older
Enrollment: 100 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2024 – 2025
U.S. locations: 3
States / cities: Long Branch, New Jersey • New Brunswick, New Jersey • Newark, New Jersey

Conditions: Cystic Fibrosis
Interventions: Hyperpolarized Xenon; Drug
Lead sponsor: Children's Hospital Medical Center, Cincinnati; Other
Eligibility: 6 Years to 12 Years
Enrollment: 38 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2025
U.S. locations: 1
States / cities: Cincinnati, Ohio

Conditions: Molar Incisor Hypomineralization
Interventions: Hydroxyapatite toothpaste, Sodium Fluoride Toothpaste; Other
Lead sponsor: The University of Texas Health Science Center at San Antonio; Other
Eligibility: 18 Years to 60 Years
Enrollment: 15 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2022
U.S. locations: 1
States / cities: San Antonio, Texas

Conditions: Mucopolysaccharidosis I, Lysosomal Storage Diseases, Spinal Cord Compression
Interventions: laronidase; Drug
Lead sponsor: Patricia I. Dickson, M.D.; Individual
Eligibility: 8 Years and older
Enrollment: 4 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2011
U.S. locations: 1
States / cities: Torrance, California

Conditions: Chronic Myeloproliferative Disorders, Leukemia, Multiple Myeloma and Malignant Plasma Cell Neoplasms, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms
Interventions: busulfan, Cyclophosphamide, VP-16, Fractionated Total Body Irradiation (FTBI); Drug · Radiation
Lead sponsor: H. Lee Moffitt Cancer Center and Research Institute; Other
Eligibility: 15 Years to 55 Years
Enrollment: 125 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1993 – 2007
U.S. locations: 1
States / cities: Tampa, Florida

Conditions: Emphysema, Alpha 1-Antitrypsin Deficiency
Interventions: Hyaluronic Acid Inhalation Solution, Placebo Inhalation Solution; Drug
Lead sponsor: Gerard Turino; Individual
Eligibility: 18 Years to 80 Years
Enrollment: 27 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2019
U.S. locations: 5
States / cities: Miami, Florida • Fayetteville, New York • Charleston, South Carolina + 2 more

Conditions: Fanconi Anemia
Interventions: metformin HCl; Drug
Lead sponsor: Boston Children's Hospital; Other
Eligibility: 6 Years to 35 Years
Enrollment: 15 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2020
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: ARG1 Deficiency
Interventions: Loargys Arginine Test System; Device
Lead sponsor: Immedica Pharma US Inc; Industry
Eligibility: 0 Years to 100 Years
Enrollment: 100 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2027
U.S. locations: 1
States / cities: Atlanta, Georgia

Conditions: Fragile X Syndrome
Interventions: Arbaclofen; Drug
Lead sponsor: Seaside Therapeutics, Inc.; Industry
Eligibility: 6 Years to 40 Years
Enrollment: 45 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2012
U.S. locations: 9
States / cities: Los Angeles, California • Sacramento, California • Chicago, Illinois + 6 more

Conditions: Hereditary Angioedema (HAE)
Interventions: navenibart; Drug
Lead sponsor: Astria Therapeutics, Inc.; Industry
Eligibility: 12 Years and older
Enrollment: 145 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2031
U.S. locations: 10
States / cities: Scottsdale, Arizona • Little Rock, Arkansas • San Diego, California + 7 more

Conditions: Epidermolysis Bullosa Dystrophica
Interventions: Not listed
Lead sponsor: Stanford University; Other
Eligibility: Not listed
Enrollment: 70 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2029
U.S. locations: 1
States / cities: Stanford, California

Conditions: Sickle Cell Disease, Chronic Kidney Disease, Pulmonary Hypertension
Interventions: Erythropoietin and Hydroxyurea; Drug
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: 18 Years and older
Enrollment: 7 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2009
U.S. locations: 1
States / cities: Bethesda, Maryland

Search by objective public record fields.

Autism Spectrum Disorder (ASD) and Intellectual Disability (ID) Determinants in Tuberous Sclerosis Complex (TSC)

Evaluation of Safety and Efficacy, Including Pharmacokinetics, of NNC 0129-0000-1003 When Administered for Treatment and Prophylaxis of Bleeding in Subjects With Haemophilia A

Transformation of Plexiform Neurofibromas to Malignant Peripheral Nerve Sheath Tumors in Neurofibromatosis Type 1

FRDA Investigator Initiated Study (IIS) With Elamipretide

A Phase I Study of Pazopanib as a Single Agent for Children With Refractory Solid Tumors

A Prospective, Observational Study in Patients With Late-Onset Pompe Disease

An Exploratory Study to Evaluate the Safety of Brimonidine Intravitreal Implant in Patients With Retinitis Pigmentosa

Work of Breathing During Non-invasive Ventilation in Premature Neonates

An Exploratory Trial of Ketamine for the Treatment of Rett Syndrome

Targeted Therapy Directed by Genetic Testing in Treating Pediatric Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphomas, or Histiocytic Disorders (The Pediatric MATCH Screening Trial)

Pre-Operative Ketamine Infusion for Post Operative Pain Control After Revision Spinal Surgery

Tobacco Use in Opioid Agonist Treated Pregnant Women

Lidocaine Intravenous in the Emergency Department For Sickle Cell Crisis

Monitoring Response to Orkambi in Cystic Fibrosis Lung Disease by Inhaled Xenon MRI

Remineralization of Molar Incisor Hypomineralization (MIH) With a Hydroxyapatite Toothpaste

Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I

Bone Marrow Transplant in Treating Patients With Hematologic Cancers

Efficacy/Safety of HA Inhalation Solution for Hereditary Emphysema in Patients With Alpha-1 Antitrypsin Deficiency

Pilot Study of Metformin for Patients With Fanconi Anemia

Evaluation of the Safety of Loargys Arginine Test System in Loargys-treated Patients

An Open Label Extension Study of STX209 in Subjects With Fragile X Syndrome

A Long-Term Study of Navenibart in Participants With Hereditary Angioedema

Characteristics of Patients With Recessive Dystrophic Epidermolysis Bullosa

Hydroxyurea and Erythropoietin to Treat Sickle Cell Anemia