- 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
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 57
matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
- 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
- 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
- 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
- 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
- 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
- Achondroplasia
- Interventions
- Not listed
- Lead sponsor
- QED Therapeutics, a BridgeBio company
- Industry
- Eligibility
- 30 Months to 17 Years
- Enrollment
- 271 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2025
- U.S. locations
- 8
- States / cities
- Oakland, California • Aurora, Colorado • Wilmington, Delaware + 5 more
- Conditions
- Digestive Disease, Gut Health
- Interventions
- Probiotic, Placebo
- Dietary Supplement · Other
- Lead sponsor
- Synbiotic Health
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 92 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2026
- U.S. locations
- 1
- States / cities
- Los Angeles, California
- Conditions
- Syringomyelia, Type I Arnold Chiari Malformation
- Interventions
- Not listed
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- 1 Year and older
- Enrollment
- 152 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2001
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Morquio Syndrome A, Maroteaux Lamy Syndrome, MPS IVA, MPS VI
- Interventions
- Not listed
- Lead sponsor
- Greenwood Genetic Center
- Other
- Eligibility
- Up to 18 Years
- Enrollment
- 17 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2015
- U.S. locations
- 1
- States / cities
- Greenville, South Carolina
- Conditions
- Epilepsy; Seizure, Neuromuscular Diseases, Brain Malformation, Intellectual Disability, Autism Spectrum Disorder, Hypotonia, Inborn Errors of Metabolism, Movement Disorders, Genetic Disease, Development Delay, Chromosome Abnormality, Hearing Loss, Dysmorphic Features, Skeletal Dysplasia, Congenital Abnormality, Microcephaly, Macrocephaly
- Interventions
- Pre-visit prep, usual care + exome seq
- Behavioral · Diagnostic Test
- Lead sponsor
- University of North Carolina, Chapel Hill
- Other
- Eligibility
- 0 Years and older
- Enrollment
- 548 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2024
- U.S. locations
- 3
- States / cities
- Asheville, North Carolina • Chapel Hill, North Carolina • Greenville, North Carolina
- Conditions
- Mucopolysaccharidosis IV Type A, Morquio A Syndrome, MPS IVA
- Interventions
- Vimizim® (elosulfase alfa)
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- Not listed
- Enrollment
- 418 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2024
- U.S. locations
- 26
- States / cities
- Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 22 more
- Conditions
- Hypochondroplasia
- Interventions
- Not listed
- Lead sponsor
- QED Therapeutics, a BridgeBio company
- Industry
- Eligibility
- 30 Months to 16 Years
- Enrollment
- 150 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2026
- U.S. locations
- 8
- States / cities
- Oakland, California • Aurora, Colorado • Washington D.C., District of Columbia + 5 more
- Conditions
- Achondroplasia
- Interventions
- Infigratinib 0.25 mg/kg/day, Placebo Comparator 0.25 mg/kg/day
- Drug
- Lead sponsor
- QED Therapeutics, a BridgeBio company
- Industry
- Eligibility
- 3 Years to 17 Years
- Enrollment
- 114 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2025
- U.S. locations
- 7
- States / cities
- San Francisco, California • Aurora, Colorado • Baltimore, Maryland + 4 more
- Conditions
- Achondroplasia
- Interventions
- Infigratinib
- Drug
- Lead sponsor
- QED Therapeutics, a BridgeBio company
- Industry
- Eligibility
- 3 Years to 18 Years
- Enrollment
- 300 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2032
- U.S. locations
- 8
- States / cities
- Oakland, California • Aurora, Colorado • Wilmington, Delaware + 5 more
- Conditions
- Gas, Bloating, Distension, Constipation, Diarrhea
- Interventions
- APP Diary, Paper Diary
- Other
- Lead sponsor
- Augusta University
- Other
- Eligibility
- 18 Years to 80 Years
- Enrollment
- 40 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2020 – 2024
- U.S. locations
- 1
- States / cities
- Augusta, Georgia
- Conditions
- Mucopolysaccharidosis Type IVA
- Interventions
- Not listed
- Lead sponsor
- Emory University
- Other
- Eligibility
- Not listed
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2013
- U.S. locations
- 1
- States / cities
- Decatur, Georgia
- Conditions
- Developmental Bone Disease, Dwarfism, Skeletal Dysplasias
- Interventions
- Not listed
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- Not listed
- Enrollment
- 600 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1998 – 2001
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Gut Health
- Interventions
- Butyrate Formulation, Butyrate + Polyphenol Formulation, Placebo
- Dietary Supplement
- Lead sponsor
- Supplement Formulators, Inc.
- Industry
- Eligibility
- 21 Years to 70 Years
- Enrollment
- 143 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2023 – 2025
- U.S. locations
- 1
- States / cities
- Fort Lauderdale, Florida
- Conditions
- Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA
- Interventions
- BMN 110
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- Up to 5 Years
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2016
- U.S. locations
- 2
- States / cities
- Oakland, California • Manhasset, New York
- Conditions
- Morquio Disease, Mucopolysaccharidosis IV
- Interventions
- Not listed
- Lead sponsor
- Nadia Ali, PhD
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2013
- U.S. locations
- 1
- States / cities
- Decatur, Georgia
- Conditions
- Mucopolysaccharidosis IV Type A, Morquio A Syndrome, MPS IVA
- Interventions
- Imaging, gait analysis, growth, joint test, hearing test, questionnaire, etc.
- Diagnostic Test
- Lead sponsor
- Nemours Children's Clinic
- Other
- Eligibility
- Not listed
- Enrollment
- 60 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2027
- U.S. locations
- 1
- States / cities
- Wilmington, Delaware
- Conditions
- MPS IVA, MPS VI
- Interventions
- Vosoritide Injection [Voxzogo]
- Drug
- Lead sponsor
- University of California, San Francisco
- Other
- Eligibility
- 5 Years to 10 Years
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2029
- U.S. locations
- 1
- States / cities
- Oakland, California
- Conditions
- MPS IVA, Morquio Syndrome
- Interventions
- Not listed
- Lead sponsor
- Oregon Health and Science University
- Other
- Eligibility
- 1 Year and older
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2017
- U.S. locations
- 1
- States / cities
- Portland, Oregon