Ultragenyx Pharmaceutical Inc
Ultragenyx Pharmaceutical Inc
Explore linked clinical trials.24 results · Page 1 of 1
Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| Setrusumab vs Placebo for Osteogenesis Imperfecta | Bone Diseases, Developmental, Collagen Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Skin and Connective Tissue Diseases, bone disorder, connective tissue disorder, hereditary disease, musculoskeletal system disorder, osteochondrodysplasia, osteogenesis imperfecta | Active, not recruiting | Phase 2 / Phase 3 | 2026-10-06 | NCT05125809 | |
| MPS IIIA Disease Monitoring Program | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Mucopolysaccharidosis III, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, connective tissue disorder, cutaneous mucinosis, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, mucopolysaccharidosis, mucopolysaccharidosis type 3A | Not yet recruiting | Not stated | 2026-10-02 | NCT07855562 | |
| Setrusumab in Pediatric Japanese Subjects With Osteogenesis Imperfecta | Bone Diseases, Developmental, Collagen Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Skin and Connective Tissue Diseases, bone disorder, connective tissue disorder, hereditary disease, musculoskeletal system disorder, osteochondrodysplasia, osteogenesis imperfecta | Active, not recruiting | Phase 3 | 2026-09-28 | NCT06636071 | |
| A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Nutritional and Metabolic Diseases, Wilson disease, basal ganglia disorder, brain disorder, central nervous system disorder, hereditary disease, inborn errors of metabolism, inborn metal metabolism disorder, inherited neurodegenerative disorder, liver disorder, metabolic disease, movement disorder, nervous system disorder, neurodegenerative disease | Active, not recruiting | Phase 1 / Phase 2 | 2026-09-25 | NCT04884815 | |
| Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Mucopolysaccharidosis III, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, connective tissue disorder, cutaneous mucinosis, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, mucopolysaccharidosis, mucopolysaccharidosis type 3A | Recruiting | Phase 2 / Phase 3 | 2026-09-22 | NCT02716246 | |
| A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAOD | very long chain acyl-CoA dehydrogenase deficiency | Active, not recruiting | Phase 3 | 2026-09-17 | NCT05933200 | |
| A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS) | Abnormalities, Multiple, Angelman syndrome, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Imprinting Disorders, central nervous system disorder, chromosomal disorder, hereditary disease, movement disorder, nervous system disorder | Active, not recruiting | Phase 2 | 2026-09-09 | NCT07157254 | |
| Long-term Extension of GTX-102 in Angelman Syndrome | Abnormalities, Multiple, Angelman syndrome, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Imprinting Disorders, central nervous system disorder, chromosomal disorder, hereditary disease, movement disorder, nervous system disorder | Active, not recruiting | Phase 3 | 2026-09-09 | NCT06415344 | |
| Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, X-linked disease, brain disorder, central nervous system disorder, hereditary disease, inborn disorder of amino acid metabolism, inborn errors of metabolism, metabolic disease, nervous system disorder, ornithine carbamoyltransferase deficiency, urea cycle disorder | Active, not recruiting | Phase 3 | 2026-09-09 | NCT05345171 | |
| First-in-human Study of UX016 in GNEM | GNE myopathy | Recruiting | Phase 1 / Phase 2 | 2026-09-03 | NCT07511556 | |
| X-linked Hypophosphatemia Disease Monitoring Program | Bone Diseases, Metabolic, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Hypophosphatemia, Familial, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Rickets, X-Linked Hypophosphatemic, Urogenital Diseases, X-linked hypophosphatemic rickets, bone disorder, calcium metabolic disease, hereditary disease, hypophosphatemia, hypophosphatemic rickets, inborn errors of metabolism, inborn metal metabolism disorder, kidney disorder, metabolic disease, musculoskeletal system disorder, nutritional deficiency disease, nutritional disorder, phosphorus metabolism disease, renal tubular transport disease, rickets, urinary system disorder, vitamin D deficiency, vitamin deficiency disorder | Active, not recruiting | Not stated | 2026-08-28 | NCT03651505 | |
| Mucopolysaccharidosis VII Disease Monitoring Program | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, connective tissue disorder, cutaneous mucinosis, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, mucopolysaccharidosis, mucopolysaccharidosis type 7 | Recruiting | Not stated | 2026-08-27 | NCT03604835 | |
| Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, disorder of glycogen metabolism, glucose metabolism disease, glycogen storage disease I, glycogen storage disease due to glucose-6-phosphatase deficiency type IA, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, metabolic disease | Recruiting | Not stated | 2026-08-26 | NCT06636383 | |
| Assessing the Impact of Intensification of Lipid Lowering Therapy With Guidelines-based Evinacumab Administration on Coronary Plaque Volumes Measured by Coronary Computed Tomography Angiography (CCTA) in Patients With Homozygous Familial Hypercholesterolemia (HoFH) | Arterial Occlusive Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Nutritional and Metabolic Diseases, arteriosclerosis disorder, atherosclerosis, cardiovascular disorder, familial hypercholesterolemia, hereditary disease, homozygous familial hypercholesterolemia, hyperlipidemia, hyperlipoproteinemia, inborn errors of metabolism, metabolic disease, vascular disorder | Recruiting | Not stated | 2026-08-21 | NCT07447648 | |
| Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS) | Abnormalities, Multiple, Angelman syndrome, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Imprinting Disorders, central nervous system disorder, chromosomal disorder, hereditary disease, movement disorder, nervous system disorder | Active, not recruiting | Phase 3 | 2026-08-06 | NCT06617429 | |
| ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cerebral Small Vessel Diseases, Ceroid Lipofuscinosis, Neuronal, 2, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Fabry disease, GM1 gangliosidosis, Gangliosidoses, Gaucher disease, Hemic and Lymphatic Diseases, Hereditary Central Nervous System Demyelinating Diseases, Infant, Newborn, Diseases, Leukoencephalopathies, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Mucopolysaccharidosis III, Neurobehavioral Manifestations, Neurologic Manifestations, Niemann-Pick disease, Niemann-Pick disease type C, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Rare Diseases, Skin and Connective Tissue Diseases, Sulfatidosis, Wolman disease, X-linked disease, brain disorder, cardiovascular disorder, central nervous system disorder, cerebrotendinous xanthomatosis, cerebrovascular disorder, cholesteryl ester storage disease, connective tissue disorder, cutaneous mucinosis, demyelinating disease, hereditary disease, histiocytosis, inborn carbohydrate metabolic disorder, inborn errors of metabolism, inherited neurodegenerative disorder, intellectual disability, lymphatic system disorder, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, metachromatic leukodystrophy, mucopolysaccharidosis, mucopolysaccharidosis type 2, mucopolysaccharidosis type 4, mucopolysaccharidosis type 6, mucopolysaccharidosis type 7, nervous system disorder, non-Langerhans cell histiocytosis, sphingolipidosis, vascular disorder, xanthomatosis | Enrolling by invitation | Not stated | 2026-07-28 | NCT05368038 | |
| Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Mucopolysaccharidosis III, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, connective tissue disorder, cutaneous mucinosis, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, mucopolysaccharidosis, mucopolysaccharidosis type 3A | Enrolling by invitation | Phase 3 | 2026-07-17 | NCT04360265 | |
| Tumor-induced Osteomalacia Disease Monitoring Program | Oncogenic osteomalacia | Active, not recruiting | Not stated | 2026-06-03 | NCT04783428 | |
| Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program | carnitine-acylcarnitine translocase deficiency, mitochondrial trifunctional protein deficiency, very long chain acyl-CoA dehydrogenase deficiency | Active, not recruiting | Not stated | 2026-06-03 | NCT04632953 | |
| Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, X-linked disease, brain disorder, central nervous system disorder, hereditary disease, inborn disorder of amino acid metabolism, inborn errors of metabolism, metabolic disease, nervous system disorder, ornithine carbamoyltransferase deficiency, urea cycle disorder | Active, not recruiting | Not stated | 2026-06-01 | NCT03636438 | |
| Setrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis Imperfecta | Bone Diseases, Developmental, Collagen Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Skin and Connective Tissue Diseases, bone disorder, connective tissue disorder, hereditary disease, musculoskeletal system disorder, osteochondrodysplasia, osteogenesis imperfecta | Active, not recruiting | Phase 3 | 2026-02-25 | NCT05768854 | |
| Triheptanoin for Children With Primary-Specific Pyruvate Dehydrogenase Complex (PDC) Deficiency | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pyruvate Dehydrogenase Complex Deficiency Disease, Pyruvate Metabolism, Inborn Errors, X-linked disease, brain disorder, central nervous system disorder, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, inborn mitochondrial metabolism disorder, inherited neurodegenerative disorder, intellectual disability, metabolic disease, nervous system disorder | Recruiting | Phase 1 | 2026-02-17 | NCT06340685 | |
| Cardiovascular Structure and Function in the Mucopolysaccharidoses | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Inflammation, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Skin and Connective Tissue Diseases, cardiovascular disorder, connective tissue disorder, cutaneous mucinosis, heart disorder, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, mucopolysaccharidosis | Active, not recruiting | Not stated | 2026-01-07 | NCT05063435 | |
| Gene Therapy for Wilson Disease Evaluated by 64Cu PET/CT | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Nutritional and Metabolic Diseases, Wilson disease, basal ganglia disorder, brain disorder, central nervous system disorder, hereditary disease, inborn errors of metabolism, inborn metal metabolism disorder, inherited neurodegenerative disorder, liver disorder, metabolic disease, movement disorder, nervous system disorder, neurodegenerative disease | Enrolling by invitation | Not stated | 2025-09-08 | NCT07159581 |