- Conditions
- Cockayne Syndrome, Skin Neoplasms, Xeroderma Pigmentosum, Trichothiodystrophy Syndromes, Genodermatosis
- Interventions
- Not listed
- Lead sponsor
- National Cancer Institute (NCI)
- NIH
- Eligibility
- 6 Weeks to 100 Years
- Enrollment
- 709 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 1999
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
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Showing 1–24
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- Conditions
- Idiopathic Short Stature
- Interventions
- No treatment is given
- Other
- Lead sponsor
- Novo Nordisk A/S
- Industry
- Eligibility
- 3 Years to 11 Years
- Enrollment
- 43 participants
- Timeline
- 2024 – 2025
- U.S. locations
- 2
- States / cities
- Orange, California • Columbia, Maryland
- Conditions
- Leukodystrophy, White Matter Disease, Leukoencephalopathies, 4H Syndrome, Adrenoleukodystrophy, AMN, ALD, ALD Gene Mutation, ALD (Adrenoleukodystrophy), X-linked Adrenoleukodystrophy, X-ALD, Adrenomyeloneuropathy, Aicardi Goutieres Syndrome, AGS, Alexander Disease, Alexanders Leukodystrophy, AxD, ADLD, Canavan Disease, CTX, Cerebrotendinous Xanthomatoses, Krabbe Disease, GALC Deficiency, Globoid Leukodystrophy, TUBB4A-Related Leukodystrophy, H-ABC - Hypomyelination, Atrophy of Basal Ganglia and Cerebellum, HBSL, HBSL - Hypomyelination, Brain Stem, Spinal Cord, Leg Spasticity, LBSL, Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder), Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation, ALSP, CSF1R Gene Mutation, HCC - Hypomyelination and Congenital Cataract, MLC1, Megalencephalic Leukoencephalopathy With Subcortical Cysts, MLD, Metachromatic Leukodystrophy, PMD, Pelizaeus-Merzbacher Disease, PLP1 Null Syndrome, PLP1 Gene Duplication | Blood or Tissue | Mutations, Pelizaeus Merzbacher Like Disease, Peroxisomal Biogenesis Disorder, Zellweger Syndrome, Refsum Disease, Salla Disease, Sialic Storage Disease, Sjögren, Sjogren-Larsson Syndrome, Van Der Knapp Disease, Vanishing White Matter Disease, Charcot-Marie-Tooth, CMT, Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency, Allan-Herndon-Dudley Syndrome, Cadasil, Cockayne Syndrome, Multiple Sulfatase Deficiency, Gangliosidoses, GM2 Gangliosidosis, BPAN, Labrune Syndrome, LCC, Mucopolysaccharidoses, TBCK-Related Intellectual Disability Syndrome
- Interventions
- Not listed
- Lead sponsor
- Children's Hospital of Philadelphia
- Other
- Eligibility
- Not listed
- Enrollment
- 12,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2030
- U.S. locations
- 23
- States / cities
- Los Angeles, California • Orange, California • Palo Alto, California + 18 more
- Conditions
- Arthritis, Juvenile Rheumatoid, Crohn Disease
- Interventions
- somatropin [rDNA origin] for injection
- Drug
- Lead sponsor
- Nationwide Children's Hospital
- Other
- Eligibility
- 5 Years to 17 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2010
- U.S. locations
- 1
- States / cities
- Columbus, Ohio
- Conditions
- Growth Hormone Deficiency, Endocrine System Diseases, Hormone Deficiency
- Interventions
- Lonapegsomatropin, Placebo, Somatropin
- Drug · Other
- Lead sponsor
- Ascendis Pharma Endocrinology Division A/S
- Industry
- Eligibility
- 23 Years to 80 Years
- Enrollment
- 264 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2023
- U.S. locations
- 23
- States / cities
- Birmingham, Alabama • Phoenix, Arizona • Fresno, California + 18 more
- Conditions
- Achondroplasia
- Interventions
- Not listed
- Lead sponsor
- Johns Hopkins University
- Other
- Eligibility
- Not listed
- Enrollment
- 1,500 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2030
- U.S. locations
- 1
- States / cities
- Baltimore, Maryland
- Conditions
- Achondroplasia
- Interventions
- Not listed
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 0 Months to 18 Years
- Enrollment
- 170 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2028
- U.S. locations
- 8
- States / cities
- Oakland, California • San Francisco, California • Baltimore, Maryland + 5 more
- Conditions
- Genetic Disorder, Prader-Willi Syndrome, Growth Disorder, Idiopathic Short Stature, Healthy
- Interventions
- somatropin
- Drug
- Lead sponsor
- Novo Nordisk A/S
- Industry
- Eligibility
- 18 Years to 40 Years
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011
- U.S. locations
- 1
- States / cities
- Evansville, Indiana
- Conditions
- Short Stature
- Interventions
- Vosoritide
- Drug
- Lead sponsor
- Andrew Dauber
- Other
- Eligibility
- 3 Years to 10 Years
- Enrollment
- 56 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2035
- U.S. locations
- 1
- States / cities
- Washington D.C., District of Columbia
- Conditions
- Growth Hormone Deficiency, Pituitary Tumor
- Interventions
- Not listed
- Lead sponsor
- Columbia University
- Other
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 60 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2010
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Growth Disorder
- Interventions
- somatropin, rDNA origin, for injection
- Drug
- Lead sponsor
- Eli Lilly and Company
- Industry
- Eligibility
- 9 Years to 16 Years
- Enrollment
- 11 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2001 – 2006
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Failure to Thrive
- Interventions
- Somatropin (rDNA origin) for injection
- Drug
- Lead sponsor
- Eli Lilly and Company
- Industry
- Eligibility
- 3 Years and older
- Enrollment
- 75 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2000 – 2010
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Hypopituitarism, Hypogonadism, Growth Hormone Deficiency
- Interventions
- Testosterone plus somatropin, testosterone
- Drug
- Lead sponsor
- University of Pennsylvania
- Other
- Eligibility
- 18 Years and older · Male only
- Enrollment
- 35 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2004 – 2010
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Growth Hormone Deficiency
- Interventions
- LUM-201, rhGH Norditropin® pen (34 µg/kg)
- Drug
- Lead sponsor
- Lumos Pharma
- Industry
- Eligibility
- 3 Years to 12 Years
- Enrollment
- 104 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2024
- U.S. locations
- 27
- States / cities
- Sacramento, California • San Diego, California • Greenwood Village, Colorado + 24 more
- Conditions
- Growth Hormone Deficiency, Growth Hormone Treatment
- Interventions
- Long-Acting Growth Hormone (LAGH)
- Drug
- Lead sponsor
- University of Minnesota
- Other
- Eligibility
- 2 Years to 13 Years
- Enrollment
- 19 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2024
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- 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
- Dwarfism, Turner's Syndrome
- Interventions
- Humatrope, Oxandrolone
- Drug
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 10 Years to 14 Years · Female only
- Enrollment
- 80 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1992 – 2007
- U.S. locations
- 2
- States / cities
- Bethesda, Maryland • Philadelphia, Pennsylvania
- 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
- Growth Hormone Deficiency, Pediatric, Endocrine System Diseases, Hormone Deficiency, Pituitary Diseases
- Interventions
- TransCon hGH
- Drug
- Lead sponsor
- Ascendis Pharma A/S
- Industry
- Eligibility
- 1 Year to 18 Years
- Enrollment
- 298 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2023
- U.S. locations
- 22
- States / cities
- Birmingham, Alabama • Los Angeles, California • Orange, California + 18 more
- 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
- Growth Hormone Deficiency
- Interventions
- VRS-317
- Drug
- Lead sponsor
- Versartis Inc.
- Industry
- Eligibility
- 25 Years to 65 Years
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2012
- U.S. locations
- 1
- States / cities
- San Antonio, Texas
- Conditions
- Growth Hormone Disorder, Growth Hormone Deficiency in Children, Growth Disorder, Idiopathic Short Stature
- Interventions
- somatropin
- Drug
- Lead sponsor
- Novo Nordisk A/S
- Industry
- Eligibility
- 3 Years to 15 Years
- Enrollment
- 173 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2000 – 2004
- U.S. locations
- 40
- States / cities
- Los Angeles, California • Sacramento, California • San Diego, California + 34 more
- Conditions
- Growth Hormone Deficiency, Pediatric, Endocrine System Diseases, Hormone Deficiency, Pituitary Diseases
- Interventions
- TransCon hGH
- Drug
- Lead sponsor
- Ascendis Pharma Endocrinology Division A/S
- Industry
- Eligibility
- 6 Months to 17 Years
- Enrollment
- 146 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2019
- U.S. locations
- 21
- States / cities
- Birmingham, Alabama • Los Angeles, California • Sacramento, California + 17 more
- Conditions
- Abdominal Obesity, Growth Hormone Deficiency
- Interventions
- Growth hormone releasing hormone (GHRH) 1-44, Placebo
- Drug
- Lead sponsor
- Massachusetts General Hospital
- Other
- Eligibility
- 18 Years to 55 Years
- Enrollment
- 60 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2012
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts