- Conditions
- Achondroplasia
- Interventions
- BMN 111, Placebo
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 5 Years to 18 Years
- Enrollment
- 121 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2019
- U.S. locations
- 11
- States / cities
- Oakland, California • Torrance, California • Wilmington, Delaware + 8 more
Search Results
Search by objective public record fields.
Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results are retrieved from ClinicalTrials.gov and synchronized into the directory. Search pages remain noindex by default.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Showing 1–24
of 188
matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy, Hyperinsulinemic Hypoglycemia, Familial 1, Diabetes Mellitus, Adrenoleukodystrophy, Neonatal, Medium-chain Acyl-CoA Dehydrogenase Deficiency, Very Long Chain Acyl Coa Dehydrogenase Deficiency, Beta-ketothiolase Deficiency, Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency, Primary Hyperoxaluria Type 1, Congenital Bile Acid Synthesis Defect Type 2, Pyridoxine-Dependent Epilepsy, Hereditary Fructose Intolerance, Hypophosphatasia, Hyperargininemia, Mucopolysaccharidosis Type 6, Argininosuccinic Aciduria, Citrullinemia, Type I, Wilson Disease, Maple Syrup Urine Disease, Type 1A, Maple Syrup Urine Disease, Type 1B, Biotinidase Deficiency, Neonatal Severe Primary Hyperparathyroidism, Intrinsic Factor Deficiency, Usher Syndrome Type 1D/F Digenic (Diagnosis), Cystic Fibrosis, Stickler Syndrome Type 2, Stickler Syndrome Type 1, Alport Syndrome, Autosomal Recessive, Alport Syndrome, X-Linked, Carbamoyl Phosphate Synthetase I Deficiency Disease, Carnitine Palmitoyl Transferase 1A Deficiency, Carnitine Palmitoyltransferase II Deficiency, Cystinosis, Chronic Granulomatous Disease, Cerebrotendinous Xanthomatoses, Maple Syrup Urine Disease, Type 2, Severe Combined Immunodeficiency Due to DCLRE1C Deficiency, Thyroid Dyshormonogenesis 6, Thyroid Dyshormonogenesis 5, Supravalvar Aortic Stenosis, Factor X Deficiency, Hemophilia A, Hemophilia B, Tyrosinemia, Type I, Fructose 1,6 Bisphosphatase Deficiency, Glycogen Storage Disease Type I, G6PD Deficiency, Glycogen Storage Disease II, Galactokinase Deficiency, Mucopolysaccharidosis Type IV A, Galactosemias, Guanidinoacetate Methyltransferase Deficiency, Agat Deficiency, Glutaryl-CoA Dehydrogenase Deficiency, Gtp Cyclohydrolase I Deficiency, Hyperinsulinism-Hyperammonemia Syndrome, Primary Hyperoxaluria Type 2, 3-Hydroxyacyl-CoA Dehydrogenase Deficiency, Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency, Mitochondrial Trifunctional Protein Deficiency, Sickle Cell Disease, Beta-Thalassemia, Holocarboxylase Synthetase Deficiency, 3-Hydroxy-3-Methylglutaric Aciduria, Primary Hyperoxaluria Type 3, Hermansky-Pudlak Syndrome 1, Hermansky-Pudlak Syndrome 4, Apparent Mineralocorticoid Excess, HSDB, CBAS1, Mucopolysaccharidosis Type 2, Mucopolysaccharidosis Type 1, Severe Combined Immunodeficiency, X Linked, Severe Combined Immunodeficiency Due to IL-7Ralpha Deficiency, Diabetes Mellitus, Permanent Neonatal, Isovaleric Acidemia, Severe Combined Immunodeficiency T-Cell Negative B-Cell Positive Due to Janus Kinase-3 Deficiency (Disorder), Jervell and Lange-Nielsen Syndrome 2, Hyperinsulinemic Hypoglycemia, Familial, 2, Diabetes Mellitus, Permanent Neonatal, With Neurologic Features, Jervell and Lange-Nielsen Syndrome 1, Lysosomal Acid Lipase Deficiency, CblF, 3-Methylcrotonyl CoA Carboxylase 1 Deficiency, 3-Methylcrotonyl CoA Carboxylase 2 Deficiency, Waardenburg Syndrome Type 2A, Methylmalonic Aciduria cblA Type, Methylmalonic Aciduria cblB Type, Methylmalonic Aciduria and Homocystinuria Type cblC, MAHCD, Methylmalonic Aciduria Due to Methylmalonyl-CoA Mutase Deficiency, Congenital Disorder of Glycosylation Type 1B, Mthfr Deficiency, Methylcobalamin Deficiency Type Cbl G (Disorder), Methylcobalamin Deficiency Type cblE, Usher Syndrome, Type 1B, N-acetylglutamate Synthase Deficiency, Ornithine Transcarbamylase Deficiency, Phenylketonurias, Waardenburg Syndrome Type 1, Congenital Hypothyroidism, Propionic Acidemia, Usher Syndrome, Type 1F, Pancreatic Agenesis 1, Hereditary Hypophosphatemic Rickets, Glycogen Storage Disease IXB, Glycogen Storage Disease IXC, MOWS, Epilepsy, Early-Onset, Vitamin B6-Dependent, Pyridoxal Phosphate-Responsive Seizures, Pituitary Hormone Deficiency, Combined, 1, Ptsd, Dihydropteridine Reductase Deficiency, Severe Combined Immunodeficiency Due to RAG1 Deficiency, Severe Combined Immunodeficiency Due to RAG2 Deficiency, Retinoblastoma, Multiple Endocrine Neoplasia Type 2B, Pseudohypoaldosteronism, Type I, Liddle Syndrome, Biotin-Responsive Basal Ganglia Disease, SCD, DIAR1, GSD1C, Acrodermatitis Enteropathica, Thyroid Dyshormonogenesis 1, Riboflavin Transporter Deficiency, Waardenburg Syndrome, Type 2E, SRD, Congenital Lipoid Adrenal Hyperplasia Due to STAR Deficiency, Barth Syndrome, Adrenocorticotropic Hormone Deficiency, Transcobalamin II Deficiency, Thyroid Dyshormonogenesis 3, Segawa Syndrome, Autosomal Recessive, Autosomal Recessive Nonsyndromic Hearing Loss, Thyroid Dyshormonogenesis 2A, Congenital Isolated Thyroid Stimulating Hormone Deficiency, Hypothyroidism Due to TSH Receptor Mutations, Usher Syndrome Type 1C, Usher Syndrome Type 1G (Diagnosis), Von Willebrand Disease, Type 3, Combined Immunodeficiency Due to ZAP70 Deficiency, Adenine Phosphoribosyltransferase Deficiency, Metachromatic Leukodystrophy, Canavan Disease, Menkes Disease, Carbonic Anhydrase VA Deficiency, Developmental and Epileptic Encephalopathy 2, 17 Alpha-Hydroxylase Deficiency, Smith-Lemli-Opitz Syndrome, Krabbe Disease, Glutathione Synthetase Deficiency, Mucopolysaccharidosis Type 7, Rett Syndrome, Molybdenum Cofactor Deficiency, Type A, Niemann-Pick Disease, Type C1, Niemann-Pick Disease Type C2, Ornithine Aminotransferase Deficiency, 3-Phosphoglycerate Dehydrogenase Deficiency, Leber Congenital Amaurosis 2, Dravet Syndrome, Mucopolysaccharidosis Type 3 A, Ornithine Translocase Deficiency, Carnitine-acylcarnitine Translocase Deficiency, Glucose Transporter Type 1 Deficiency Syndrome, Creatine Transporter Deficiency, Niemann-Pick Disease Type A, Pitt Hopkins Syndrome, Tuberous Sclerosis 1, Tuberous Sclerosis 2, Ataxia With Isolated Vitamin E Deficiency, Angelman Syndrome, Prader-Willi Syndrome, Homocystinuria, Permanent Neonatal Diabetes Mellitus, Transient Neonatal Diabetes Mellitus, Factor VII Deficiency, Glycogen Storage Disease Type IXA1, Glycogen Storage Disease, Type IXA2, Glycogen Storage Disease IC, Glycogen Storage Disease Type IB, Central Hypoventilation Syndrome With or Without Hirschsprung Disease
- Interventions
- Confirmatory Testing
- Diagnostic Test
- Lead sponsor
- RTI International
- Other
- Eligibility
- 1 Day to 31 Days
- Enrollment
- 30,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2018 – 2026
- U.S. locations
- 1
- States / cities
- Research Triangle Park, North Carolina
- Conditions
- Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Chronic Myeloid Leukemia, Myelodysplastic Syndrome, Lymphomas, Bone Marrow Failure, Hemoglobinopathy, Immune Deficiency, Osteopetrosis, Cytopenias, Leukocyte Disorders, Anemia Due to Intrinsic Red Cell Abnormality
- Interventions
- CD34+ enriched, T Cell Depleted donor stem cell product
- Biological
- Lead sponsor
- Christopher Dvorak
- Other
- Eligibility
- 2 Months to 30 Years
- Healthy volunteers
- Healthy volunteers not accepted
- U.S. locations
- 1
- States / cities
- San Francisco, California
- Conditions
- Exostoses, Multiple Hereditary
- Interventions
- Palovarotene 2.5 mg, Palovarotene 5.0 mg, Placebo
- Drug · Other
- Lead sponsor
- Clementia Pharmaceuticals Inc.
- Industry
- Eligibility
- 2 Years to 14 Years
- Enrollment
- 193 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2020
- U.S. locations
- 13
- States / cities
- Los Angeles, California • Sacramento, California • San Francisco, California + 9 more
- Conditions
- Gastrointestinal, Colic
- Interventions
- Lactobacillus reuteri, Placebo
- Biological
- Lead sponsor
- The University of Texas Health Science Center, Houston
- Other
- Eligibility
- 3 Weeks to 3 Months
- Enrollment
- 21 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2013 – 2016
- U.S. locations
- 1
- States / cities
- Houston, Texas
- Conditions
- Osteogenesis Imperfecta
- Interventions
- SAR439459, Placebo
- Drug
- Lead sponsor
- Sanofi
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 16 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2024
- U.S. locations
- 7
- States / cities
- Los Angeles, California • New Haven, Connecticut • Indianapolis, Indiana + 4 more
- Conditions
- McCune Albright Syndrome, Polyostotic Fibrous Dysplasia, Precocious Puberty
- Interventions
- Letrozole
- Drug
- Lead sponsor
- National Institute of Dental and Craniofacial Research (NIDCR)
- NIH
- Eligibility
- 1 Year to 8 Years · Female only
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2000 – 2009
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Mucopolysaccharidosis Disorders, Hurler Syndrome, Hunter Syndrome, Maroteaux Lamy Syndrome, Sly Syndrome, Alpha-Mannosidosis, Fucosidosis, Aspartylglucosaminuria, Glycoprotein Metabolic Disorders, Sphingolipidoses, Recessive Leukodystrophies, Globoid Cell Leukodystrophy, Metachromatic Leukodystrophy, Niemann-Pick B, Niemann-Pick C Subtype 2, Sphingomyelin Deficiency, Peroxisomal Disorders, Adrenoleukodystrophy With Cerebral Involvement, Zellweger Syndrome, Neonatal Adrenoleukodystrophy, Infantile Refsum Disease, Acyl-CoA Oxidase Deficiency, D-Bifunctional Enzyme Deficiency, Multifunctional Enzyme Deficiency, Alpha-methylacyl-CoA Racmase Deficiency, Mitochondrial Neurogastrointestingal Encephalopathy, Severe Osteopetrosis, Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation), Inherited Metabolic Disorders
- Interventions
- Stem Cell Transplantation, IMD Preparative Regimen, Osteopetrosis Only Preparative Regimen, Osteopetrosis Haploidentical Only Preparative Regimen, cALD SR-A (Standard-Risk, Regimen A), cALD SR-B (Standard-Risk, Regimen B), cALD HR-D (High-Risk, Regimen C), cALD HR-D (High-Risk, Regimen D)
- Biological · Drug
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- Up to 55 Years
- Enrollment
- 149 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2029
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Connective Tissue Disorders, Osteogenesis Imperfecta, Bone Diseases, Metabolic, Melorheostosis
- Interventions
- Not listed
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- Up to 70 Years
- Enrollment
- 180 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- Started 2004
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Breast Neoplasms, Prostate Neoplasms, Neoplasms, Squamous Cell, Melanoma, Mesothelioma, Pancreatic Neoplasms, Colorectal Neoplasms, Carcinoma, Renal Cell, Liver Neoplasms
- Interventions
- PF-06952229, Enzalutamide
- Drug
- Lead sponsor
- Pfizer
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 49 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2022
- U.S. locations
- 15
- States / cities
- Scottsdale, Arizona • Santa Monica, California • Boston, Massachusetts + 6 more
- Conditions
- Polyostotic Fibrous Dysplasia
- Interventions
- Not listed
- Lead sponsor
- National Institute of Dental and Craniofacial Research (NIDCR)
- NIH
- Eligibility
- Not listed
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1996 – 2008
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Autosomal Dominant Osteopetrosis Type 2
- Interventions
- ACTIMMUNE
- Drug
- Lead sponsor
- Indiana University
- Other
- Eligibility
- 3 Years to 65 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2019
- U.S. locations
- 1
- States / cities
- Indianapolis, Indiana
- Conditions
- Achondroplasia
- Interventions
- BMN 333, Vosoritide Injection [Voxzogo]
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 2 Years to 17 Years
- Enrollment
- 160 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2029
- U.S. locations
- 9
- States / cities
- Oakland, California • Wilmington, Delaware • Chicago, Illinois + 6 more
- Conditions
- Healthy
- Interventions
- Pregabalin, Placebo, Bowel preparation
- Drug · Other
- Lead sponsor
- Mayo Clinic
- Other
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 62 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2010 – 2011
- U.S. locations
- 1
- States / cities
- Rochester, Minnesota
- Conditions
- Osteogenesis Imperfecta
- Interventions
- Zoledronic Acid
- Drug
- Lead sponsor
- Novartis Pharmaceuticals
- Industry
- Eligibility
- 1 Year to 17 Years
- Enrollment
- 127 participants
- Timeline
- 2004 – 2007
- U.S. locations
- 10
- States / cities
- Los Angeles, California • Wilmington, Delaware • Boise, Idaho + 7 more
- Conditions
- Advanced Solid Tumor, Refractory Hodgkin Lymphoma
- Interventions
- HCB301
- Drug
- Lead sponsor
- FBD Biologics Limited
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2027
- U.S. locations
- 1
- States / cities
- Greenville, South Carolina
- Conditions
- Achondroplasia
- Interventions
- Active BMN 111: Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily.
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 15 Months and older
- Enrollment
- 73 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2038
- U.S. locations
- 9
- States / cities
- Oakland, California • Torrance, California • Wilmington, Delaware + 6 more
- Conditions
- Osteopetrosis
- Interventions
- Stem Cell or Umbilical Cord Blood Transplantation, Campath, Busulfan, Clofarabine, Total Lymphoid Irradiation
- Procedure · Drug
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- Up to 45 Years
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2008
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI, Mucopolysaccharidosis VII, Pompe Disease Infantile-Onset, Neuronopathic Gaucher Disease, Wolman Disease
- Interventions
- There is no intervention
- Other
- Lead sponsor
- University of California, San Francisco
- Other
- Eligibility
- Up to 64 Years
- Enrollment
- 250 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2050
- U.S. locations
- 1
- States / cities
- San Francisco, California
- Conditions
- Locally Advanced or Metastatic Cancers, Metastatic Human Papillomavirus-Related Malignant Neoplasm
- Interventions
- TST005
- Drug
- Lead sponsor
- Suzhou Transcenta Therapeutics Co., Ltd.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 19 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2023
- U.S. locations
- 3
- States / cities
- Canton, Ohio • Dallas, Texas • San Antonio, Texas
- Conditions
- MPS I, MPS II, MPS IVA, MPS VI, Mps VII, Gaucher Disease, Type 2, Gaucher Disease, Type 3, Pompe Disease Infantile-Onset, Wolman Disease
- Interventions
- Aldurazyme (laronidase)
- Drug
- Lead sponsor
- University of California, San Francisco
- Other
- Eligibility
- 18 Years to 50 Years · Female only
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2032
- U.S. locations
- 1
- States / cities
- San Francisco, California
- Conditions
- Osteogenesis Imperfecta
- Interventions
- Not listed
- Lead sponsor
- Baylor College of Medicine
- Other
- Eligibility
- 10 Years to 100 Years
- Enrollment
- 75 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2027
- U.S. locations
- 2
- States / cities
- Los Angeles, California • Houston, Texas
- Conditions
- Achondroplasia
- Interventions
- Recifercept
- Biological
- Lead sponsor
- Pfizer
- Industry
- Eligibility
- 3 Months to 10 Years
- Enrollment
- 60 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2023
- U.S. locations
- 6
- States / cities
- Aliso Viejo, California • Irvine, California • Long Beach, California + 3 more
- Conditions
- Osteogenesis Imperfecta (OI)
- Interventions
- AGA2115, Placebo
- Drug · Other
- Lead sponsor
- Angitia Incorporated Limited
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 80 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2029
- U.S. locations
- 9
- States / cities
- Phoenix, Arizona • New Haven, Connecticut • Wilmington, Delaware + 6 more