Dwarfism
Dwarfism
Explore linked clinical trials. Standard reference: MESH:M000689778 results · Page 1 of 4
Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| Phase 3 Study of LUM-201 in Children With Growth Hormone Deficiency | Bone Diseases, Developmental, Bone Diseases, Endocrine, Dwarfism, bone disorder, brain disorder, central nervous system disorder, endocrine system disorder, hypopituitarism, hypothalamic disorder, musculoskeletal system disorder, nervous system disorder, pituitary dwarfism, pituitary gland disorder | Recruiting | Phase 3 | 2026-10-09 | NCT06948214 | |
| Evaluate the Efficacy and Safety of KK8398 in Patients With Achondroplasia（AOBA Study） | Bone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, achondroplasia, bone disorder, hereditary disease, musculoskeletal system disorder, osteochondrodysplasia | Active, not recruiting | Phase 3 | 2026-10-08 | NCT06926491 | |
| Ngenla Subcutaneous Injection Special Investigation | Bone Diseases, Developmental, Bone Diseases, Endocrine, Dwarfism, bone disorder, brain disorder, central nervous system disorder, endocrine system disorder, hypopituitarism, hypothalamic disorder, musculoskeletal system disorder, nervous system disorder, pituitary dwarfism, pituitary gland disorder | Active, not recruiting | Not stated | 2026-10-07 | NCT05602766 | |
| Prospective Evaluation of OptiThyDose | Bone Diseases, Developmental, Bone Diseases, Endocrine, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, Graves disease, autoimmune disease, bone disorder, congenital hypothyroidism, disease of orbital part of eye adnexa, endocrine system disorder, exophthalmos, eye disorder, goiter, hereditary disease, hyperthyroidism, hypothyroidism, immune system disorder, musculoskeletal system disorder, thyroid gland disorder | Recruiting | Not applicable | 2026-10-02 | NCT06864351 | |
| A Study of TYRA-300 in Children With Achondroplasia: BEACH301 | Bone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, achondroplasia, bone disorder, hereditary disease, musculoskeletal system disorder, osteochondrodysplasia | Recruiting | Phase 2 | 2026-10-01 | NCT06842355 | |
| Trial Investigating the Efficacy and Safety of Weekly Lonapegsomatropin Compared to Daily Somatropin in Children and Adolescents With Short Stature or Growth Failure Due to Growth Hormone Sufficient Disorders | Bone Diseases, Developmental, Cardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Craniofacial Abnormalities, Dwarfism, Failure to Thrive, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Musculoskeletal Abnormalities, Noonan syndrome, Pathological Conditions, Signs and Symptoms, Sex Chromosome Disorders, Signs and Symptoms, Skin and Connective Tissue Diseases, Turner syndrome, Urogenital Abnormalities, Urogenital Diseases, bone disorder, cardiovascular disorder, chromosomal disorder, congenital heart disease, connective tissue disorder, disorder of sexual differentiation, endocrine system disorder, gonadal disorder, gonadal dysgenesis, heart disorder, hereditary disease, musculoskeletal system disorder, sex chromosome disorder of sex development | Recruiting | Phase 3 | 2026-09-30 | NCT07221851 | |
| The AgRP and GH/IGF-1 Axis in Children | Bone Diseases, Developmental, Bone Diseases, Endocrine, Dwarfism, bone disorder, brain disorder, central nervous system disorder, endocrine system disorder, hypopituitarism, hypothalamic disorder, musculoskeletal system disorder, nervous system disorder, pituitary dwarfism, pituitary gland disorder | Not yet recruiting | Not stated | 2026-09-25 | NCT05645211 | |
| Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia | Bone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, achondroplasia, bone disorder, hereditary disease, musculoskeletal system disorder, osteochondrodysplasia | Recruiting | Phase 2 / Phase 3 | 2026-09-24 | NCT07441876 | |
| A Study to Learn About Somatrogon in Patients With Pediatric Growth Hormone Deficiency (p GHD) in India. | Bone Diseases, Developmental, Bone Diseases, Endocrine, Dwarfism, bone disorder, brain disorder, central nervous system disorder, endocrine system disorder, hypopituitarism, hypothalamic disorder, musculoskeletal system disorder, nervous system disorder, pituitary dwarfism, pituitary gland disorder | Recruiting | Not stated | 2026-09-24 | NCT06587035 | |
| Prospective Longitudinal Multicentric Study to Measure Limb Movement in Patients With FGFR3-related Skeletal Dysplasia | Bone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, achondroplasia, bone disorder, hereditary disease, hypochondroplasia, musculoskeletal system disorder, osteochondrodysplasia | Recruiting | Not stated | 2026-09-23 | NCT07388966 | |
| A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment | Bone Diseases, Developmental, Cardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Craniofacial Abnormalities, Dwarfism, Musculoskeletal Abnormalities, Noonan syndrome, Skin and Connective Tissue Diseases, bone disorder, cardiovascular disorder, congenital heart disease, connective tissue disorder, endocrine system disorder, heart disorder, hereditary disease, musculoskeletal system disorder | Active, not recruiting | Phase 2 | 2026-09-23 | NCT06668805 | |
| BELux Children OutcoME During A(@)Dulthood With GHD | Bone Diseases, Developmental, Bone Diseases, Endocrine, Dwarfism, bone disorder, brain disorder, central nervous system disorder, endocrine system disorder, hypopituitarism, hypothalamic disorder, musculoskeletal system disorder, nervous system disorder, pituitary dwarfism, pituitary gland disorder | Recruiting | Not applicable | 2026-09-21 | NCT06694506 | |
| Charactertizing Growth Hormone Deficiency in Traumatic Brain Injury | Bone Diseases, Developmental, Bone Diseases, Endocrine, Brain Concussion, Brain Injuries, Traumatic, Craniocerebral Trauma, Dwarfism, Head Injuries, Closed, Wounds, Nonpenetrating, bone disorder, brain disorder, brain injury, central nervous system disorder, endocrine system disorder, hypopituitarism, hypothalamic disorder, injury, musculoskeletal system disorder, nervous system disorder, nervous system injury, pituitary dwarfism, pituitary gland disorder | Recruiting | Not stated | 2026-08-31 | NCT07795736 | |
| Open-Label, Long-Term, Extension Study of Infigratinib in Children With Hypochondroplasia | Bone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, bone disorder, connective tissue disorder, cutaneous mucinosis, endocrine system disorder, hereditary disease, hypochondroplasia, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, mucopolysaccharidosis, mucopolysaccharidosis type 4, musculoskeletal system disorder, osteochondrodysplasia | Enrolling by invitation | Phase 2 | 2026-08-26 | NCT07393373 | |
| A Clinical Trial to Investigate Long-term Safety, Tolerability, and Efficacy of Weekly Subcutaneous Doses With TransCon CNP in Children and Adolescents With Achondroplasia | Bone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, achondroplasia, bone disorder, endocrine system disorder, hereditary disease, musculoskeletal system disorder, osteochondrodysplasia | Enrolling by invitation | Phase 2 / Phase 3 | 2026-08-17 | NCT05929807 | |
| Safety, Pharmacokinetic (PK) Profile and Efficacy of Inpegsomatropin Injection in Adults With Growth Hormone Deficiency | Bone Diseases, Developmental, Bone Diseases, Endocrine, Dwarfism, bone disorder, brain disorder, central nervous system disorder, endocrine system disorder, hypopituitarism, hypothalamic disorder, musculoskeletal system disorder, nervous system disorder, pituitary dwarfism, pituitary gland disorder | Recruiting | Phase 2 | 2026-07-22 | NCT07551804 | |
| Long-term Safety and Effectiveness of Growtropin®-II Treatment in Children With Short Stature | Bone Diseases, Developmental, Bone Diseases, Endocrine, Cardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Sex Chromosome Disorders, Turner syndrome, Urogenital Abnormalities, Urogenital Diseases, bone disorder, brain disorder, cardiovascular disorder, central nervous system disorder, chromosomal disorder, congenital heart disease, disorder of sexual differentiation, endocrine system disorder, gonadal disorder, gonadal dysgenesis, heart disorder, hereditary disease, hypopituitarism, hypothalamic disorder, musculoskeletal system disorder, nervous system disorder, pituitary dwarfism, pituitary gland disorder, sex chromosome disorder of sex development | Recruiting | Not stated | 2026-07-21 | NCT04798690 | |
| Study of Skeletal Disorders | Bone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, bone disorder, connective tissue disorder, cutaneous mucinosis, endocrine system disorder, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, mucopolysaccharidosis, mucopolysaccharidosis type 4, musculoskeletal system disorder | Recruiting | Not stated | 2026-07-20 | NCT05031507 | |
| An Interventional Study of Infigratinib in Children With Hypochondroplasia | Bone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, bone disorder, connective tissue disorder, cutaneous mucinosis, endocrine system disorder, hereditary disease, hypochondroplasia, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, mucopolysaccharidosis, mucopolysaccharidosis type 4, musculoskeletal system disorder, osteochondrodysplasia | Enrolling by invitation | Phase 2 / Phase 3 | 2026-07-16 | NCT06873035 | |
| Prospective Clinical Assessment Study in Children With Hypochondroplasia | Bone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, bone disorder, connective tissue disorder, cutaneous mucinosis, endocrine system disorder, hereditary disease, hypochondroplasia, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, mucopolysaccharidosis, mucopolysaccharidosis type 4, musculoskeletal system disorder, osteochondrodysplasia | Recruiting | Not stated | 2026-07-16 | NCT06410976 | |
| Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH) | Bone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Skin and Connective Tissue Diseases, achondroplasia, bone disorder, connective tissue disorder, cutaneous mucinosis, endocrine system disorder, hereditary disease, hereditary endocrine growth disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, mucopolysaccharidosis, mucopolysaccharidosis type 4, musculoskeletal system disorder, osteochondrodysplasia | Recruiting | Phase 2 | 2026-07-13 | NCT07169279 | |
| Growth Hormone Replacement Therapy in Veterans With Mild Traumatic Brain Injury (mTBI) and Adult Growth Hormone Deficiency (AGHD) | Bone Diseases, Developmental, Bone Diseases, Endocrine, Brain Concussion, Brain Injuries, Traumatic, Concussion, Mild, Craniocerebral Trauma, Dwarfism, Head Injuries, Closed, Wounds, Nonpenetrating, bone disorder, brain disorder, brain injury, central nervous system disorder, endocrine system disorder, hypopituitarism, hypothalamic disorder, injury, musculoskeletal system disorder, nervous system disorder, nervous system injury, pituitary dwarfism, pituitary gland disorder | Recruiting | Phase 3 | 2026-07-09 | NCT04867317 | |
| Phase 3 Long Term Safety Extension Study of LUM-201 in Children With Growth Hormone Deficiency | Bone Diseases, Developmental, Bone Diseases, Endocrine, Dwarfism, bone disorder, brain disorder, central nervous system disorder, endocrine system disorder, hypopituitarism, hypothalamic disorder, musculoskeletal system disorder, nervous system disorder, pituitary dwarfism, pituitary gland disorder | Not yet recruiting | Phase 3 | 2026-07-08 | NCT07129759 | |
| Early Check: Expanded Screening in Newborns | 3-hydroxy-3-methylglutaric aciduria, 3-hydroxyacyl-CoA dehydrogenase deficiency, 3-methylcrotonyl-CoA carboxylase 1 deficiency, 3-methylcrotonyl-CoA carboxylase 2 deficiency, 3-phosphoglycerate dehydrogenase deficiency, AGAT deficiency, Abnormalities, Multiple, Adrenal hyperplasia, congenital, type 5, Adrenoleukodystrophy, Neonatal, Angelman syndrome, Aortic Stenosis, Supravalvular, Arrhythmias, Cardiac, Barth syndrome, Bone Diseases, Developmental, Bone Diseases, Endocrine, Bone Diseases, Metabolic, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Canavan disease, Cardiac Conduction System Disease, Cardiovascular Abnormalities, Chronic Disease, Citrullinemia 1, Collagen Diseases, Congenital Abnormalities, Congenital Hyperinsulinism, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Diabetes Mellitus, Permanent Neonatal, With Neurologic Features, Digestive System Diseases, Disease Attributes, Duchenne muscular dystrophy, Dwarfism, Dyslipidemias, Epilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, Eye Diseases, Hereditary, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Fructose Metabolism, Inborn Errors, G6PD deficiency, Glycogen Storage Disease, Type IXA2, Hemic and Lymphatic Diseases, Hereditary Central Nervous System Demyelinating Diseases, Hermansky-Pudlak syndrome, Hermansky-Pudlak syndrome 1, Hermansky-Pudlak syndrome 4, Hyperphenylalaninemia, BH4-Deficient, B, Hypophosphatemia, Familial, Imprinting Disorders, Infant, Newborn, Diseases, Jervell and Lange-Nielsen syndrome, Jervell and Lange-Nielsen syndrome 1, Jervell and Lange-Nielsen syndrome 2, Krabbe disease, Leber congenital amaurosis 2, Leukoencephalopathies, Liddle syndrome, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, MPI-congenital disorder of glycosylation, Male Urogenital Diseases, Maple syrup urine disease, type 1A, Maple syrup urine disease, type 1B, Maple syrup urine disease, type 2, Menkes disease, Methylmalonic aciduria cblB type, Mucopolysaccharidosis I, Mucopolysaccharidosis III, Mucopolysaccharidosis Type IV A, Muscular Disorders, Atrophic, Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Germ Cell and Embryonal, Neoplasms, Multiple Primary, Neoplasms, Nerve Tissue, Neurobehavioral Manifestations, Neurologic Manifestations, Niemann-Pick disease, Niemann-Pick disease type A, Niemann-Pick disease type C, Niemann-Pick disease, type C1, Niemann-Pick disease, type C2, Nutritional and Metabolic Diseases, Overweight, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Peroxisomal Disorders, Pitt-Hopkins syndrome, Prader-Willi syndrome, Primary Immunodeficiency Diseases, Rett syndrome, Schnyder corneal dystrophy, Sex Chromosome Disorders, Skin and Connective Tissue Diseases, Smith-Lemli-Opitz syndrome, Stickler syndrome type 1, Stickler syndrome type 2, Stress Disorders, Traumatic, Sulfatidosis, Tyrosinemia, Type I, Urogenital Abnormalities, Urogenital Diseases, Usher syndrome type 1B, Usher syndrome type 1C, Usher syndrome, type 1C, Usher syndrome, type 1F, Ventricular Outflow Obstruction, Waardenburg Syndrome, Type 2E, Waardenburg syndrome, Waardenburg syndrome type 1, Waardenburg syndrome type 2, Waardenburg syndrome type 2A, Wilson disease, Wolman disease, X-Linked Combined Immunodeficiency Diseases, X-linked Alport syndrome, X-linked disease, X-linked hypophosphatemic rickets, acrodermatitis enteropathica, acute stress disorder, adenine phosphoribosyltransferase deficiency, albinism, anemia, aortic valve disorder, aortic valve stenosis, apparent mineralocorticoid excess, arginase deficiency, argininosuccinic aciduria, autosomal recessive Alport syndrome, basal ganglia disorder, beta thalassemia, beta-ketothiolase deficiency, biotin-responsive basal ganglia disease, biotinidase deficiency, blood coagulation disease, blood platelet disease, bone disorder, brain disorder, calcium metabolic disease, carbamoyl phosphate synthetase I deficiency disease, cardiovascular disorder, carnitine palmitoyl transferase 1A deficiency, carnitine palmitoyltransferase II deficiency, carnitine-acylcarnitine translocase deficiency, central nervous system disorder, cerebrotendinous xanthomatosis, cholesteryl ester storage disease, chromosomal disorder, chronic granulomatous disease, citrullinemia, coagulation protein disease, combined immunodeficiency due to ZAP70 deficiency, congenital bile acid synthesis defect 1, congenital bile acid synthesis defect 2, congenital heart disease, congenital hypothyroidism, congenital isolated adrenocorticotropic hormone deficiency, connective tissue disorder, creatine transporter deficiency, cutaneous mucinosis, cystic fibrosis, cystinosis, demyelinating disease, diabetes mellitus, disease, disorder of glycogen metabolism, disorder of pilosebaceous unit, dopa-responsive dystonia due to sepiapterin reductase deficiency, encephalopathy due to GLUT1 deficiency, endocrine system disorder, epilepsy, epilepsy, early-onset, vitamin B6-dependent, epithelial neoplasm, eye disorder, factor VII deficiency, factor X deficiency, familial hemolytic anemia, familial isolated deficiency of vitamin E, fragile X syndrome, fructose-1,6-bisphosphatase deficiency, galactosemia, glucose metabolism disease, glutaryl-CoA dehydrogenase deficiency, glycogen storage disease I, glycogen storage disease II, glycogen storage disease IXb, glycogen storage disease IXc, glycogen storage disease Ib, guanidinoacetate methyltransferase deficiency, heart disorder, heart valve disorder, hematologic disorder, hemolytic anemia, hemophilia A, hemophilia B, hemorrhagic disease, hereditary disease, hereditary fructose intolerance, hereditary neoplastic syndrome, hereditary nephritis, hereditary skin disorder, histiocytosis, holocarboxylase synthetase deficiency, homocystinuria, homocystinuria due to methylene tetrahydrofolate reductase deficiency, hyperammonemia due to N-acetylglutamate synthase deficiency, hyperhomocysteinemia, hyperinsulinemic hypoglycemia, familial, 2, hyperinsulinism, hyperinsulinism-hyperammonemia syndrome, hypoglycemia, hypophosphatasia, hypophosphatemia, hypophosphatemic rickets, hypopigmentation of the skin, hypothyroidism, hypothyroidism due to TSH receptor mutations, immune system disorder, inborn carbohydrate metabolic disorder, inborn disorder of amino acid metabolism, inborn error of immunity, inborn errors of metabolism, inborn metal metabolism disorder, inborn mitochondrial metabolism disorder, inherited blood coagulation disorder, inherited hemoglobinopathy, inherited neurodegenerative disorder, intellectual disability, isovaleric acidemia, kidney disorder, leukocyte disorder, liver disorder, lung disorder, lymphatic system disorder, lysosomal lipid storage disorder, lysosomal storage disease, malignant endocrine neoplasm, maturity-onset diabetes of the young type 4, medium chain acyl-CoA dehydrogenase deficiency, metabolic disease, metachromatic leukodystrophy, methylcobalamin deficiency type cblE, methylmalonic aciduria and homocystinuria, methylmalonic aciduria and homocystinuria type cblC, methylmalonic aciduria due to methylmalonyl-CoA mutase deficiency, methylmalonic aciduria, cblA type, mitochondrial trifunctional protein deficiency, motor neuron disorder, movement disorder, mucopolysaccharidosis, mucopolysaccharidosis type 1, mucopolysaccharidosis type 2, mucopolysaccharidosis type 3A, mucopolysaccharidosis type 4, mucopolysaccharidosis type 6, mucopolysaccharidosis type 7, multiple carboxylase deficiency, multiple endocrine neoplasia, multiple endocrine neoplasia type 2B, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, neonatal severe primary hyperparathyroidism, neoplasm, nephritis, nervous system disorder, neurodegenerative disease, neuroepithelial neoplasm, neuromuscular disease, non-Langerhans cell histiocytosis, nutritional deficiency disease, nutritional disorder, obesity disorder, ocular cancer, oculocutaneous albinism, optic choroid disorder, ornithine aminotransferase deficiency, ornithine carbamoyltransferase deficiency, ornithine translocase deficiency, overnutrition, pancreas disorder, pancreatic agenesis 1, permanent neonatal diabetes mellitus, phagocyte bactericidal dysfunction, phenylketonuria, phosphorus metabolism disease, pituitary hormone deficiency, combined, 1, platelet storage pool deficiency, post-traumatic stress disorder, primary hyperoxaluria type 1, primary hyperoxaluria type 2, primary hyperoxaluria type 3, primitive neuroectodermal tumor, propionic acidemia, pseudohypoaldosteronism, pseudohypoaldosteronism type 1, psychiatric disorder, pyridoxal phosphate-responsive seizures, pyridoxine-dependent epilepsy, renal tubular transport disease, respiratory system disorder, retinal cancer, retinal disorder, retinoblastoma, riboflavin transporter deficiency, rickets, severe combined immunodeficiency, severe combined immunodeficiency due to DCLRE1C deficiency, severe combined immunodeficiency, autosomal recessive, T cell-negative, B cell-negative, NK cell-negative, due to adenosine deaminase deficiency, sickle cell disease, skin disorder, skin pigmentation disorder, sphingolipidosis, spinal cord disorder, spinal muscular atrophy, steroid inherited metabolic disorder, sulfite oxidase deficiency due to molybdenum cofactor deficiency type A, thalassemia, thyroid dyshormonogenesis 2A, thyroid dyshormonogenesis 3, thyroid dyshormonogenesis 5, thyroid dyshormonogenesis 6, thyroid gland disorder, transcobalamin II deficiency, transient neonatal diabetes mellitus, tuberous sclerosis 1, tuberous sclerosis 2, tyrosine hydroxylase deficiency, tyrosinemia, urea cycle disorder, urinary system disorder, uveal disorder, very long chain acyl-CoA dehydrogenase deficiency, vitamin D deficiency, vitamin deficiency disorder, von Willebrand disease (hereditary or acquired), von Willebrand disease 3, xanthomatosis | Active, not recruiting | Not stated | 2026-07-01 | NCT03655223 | |
| VIrtual STudy in Achondroplasia for the US (VISTA) | Bone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, achondroplasia, bone disorder, hereditary disease, musculoskeletal system disorder, osteochondrodysplasia | Recruiting | Not stated | 2026-06-30 | NCT06168201 |