Clinical Trials Sponsored by Ultragenix pharmaceutical

Total 19784 results

Bayer
Ultragenix pharmaceutical

Active, not recruiting

Study to Test the Safety and How Well Patients With Severe Hemophilia A Respond to Treatment With BAY 2599023 (DTX 201), a Drug Therapy That Delivers a Healthy Version of the Defective Factor VIII Gene Into the Nucleus of Liver Cells Using an Altered, Non-infectious Virus (AAV) as a "Shuttle".

Hemophilia A

Netherlands, United States, Germany, United Kingdom, France, Bulgaria
Ultragenyx Pharmaceutical Inc

Active, not recruiting

Setrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis Imperfecta (Cosmic)

Osteogenesis Imperfecta

United States, Italy, Brazil, France, Canada, Germany, Netherlands, Poland
Ultragenyx Pharmaceutical Inc

Active, not recruiting

Study of UX701 Gene Transfer for the Treatment of Wilson Disease

Wilson Disease

United States, Canada, Spain, Portugal, United Kingdom
Ultragenyx Pharmaceutical Inc

Active, not recruiting

Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia

Glycogen Storage Disease Type IA | Von Gierke's Disease (GSD Type Ia)

Netherlands, United States, Canada, Spain
Ultragenyx Pharmaceutical Inc

Completed

Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Extension Study for Subjects Previously Enrolled in Triheptanoin Studies

Carnitine Palmitoyltransferase (CPT I or CPT II) Deficiency | Very Long Chain Acyl-CoA Dehydrogenase (VLCAD) Deficiency | Long-chain 3-hydroxy-acyl-CoA Dehydrogenase (LCHAD) Deficiency | Trifunctional Protein (TFP) Deficiency | Carnitine-acylcarnitine Translocase (CACT) Deficiency

United States, United Kingdom
Ultragenyx Pharmaceutical Inc

Terminated

Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)

Hereditary Inclusion Body Myopathy | GNE Myopathy | Distal Myopathy With Rimmed Vacuoles | Distal Myopathy, Nonaka Type | Quadriceps Sparing Myopathy

United States, Canada, United Kingdom, Israel, Italy, Bulgaria, France
Ultragenyx Pharmaceutical Inc

Terminated

Long-Chain Fatty Acid Oxidation Disorders Online Disease Monitoring Program

Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)

United States
Ultragenyx Pharmaceutical Inc

Completed

Clinical Evaluation and Assessment of Instruments and Biomarkers in Subjects With Wilson Disease

Wilson Disease

United States, Germany
Ultragenyx Pharmaceutical Inc

Completed

Safety, Pharmacokinetics and Pharmacodynamics of BPS804 in Osteogenesis Imperfecta

Osteogenesis Imperfecta

Belgium, United States, Canada, Germany
Ultragenyx Pharmaceutical Inc

Completed

A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)

Mucopolysaccharidosis | Sly Syndrome | Mucopolysaccharidosis VII | MPS VII

United States, Brazil, Mexico, Portugal
Ultragenyx Pharmaceutical Inc

Completed

A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)

Mucopolysaccharidosis | Sly Syndrome | MPS VII | MPS 7

United States
Ultragenyx Pharmaceutical Inc

Terminated

Study to Assess the Long Term Safety and Efficacy of UX007 in Participants With Glucose Type 1 Deficiency Syndrome (Glut1 DS)

Glucose Transporter Type 1 Deficiency Syndrome

United States, Spain, Denmark, United Kingdom, Australia
Ultragenyx Pharmaceutical Inc

Completed

A Study to Assess Plasma Ammonia Time-Normalized Area Under the Curve and Rate of Ureagenesis in Healthy Adult Subjects

Ornithine Transcarbamylase Deficiency

United States
Ultragenyx Pharmaceutical Inc

Terminated

A Study to Evaluate the Safety of Aceneuramic Acid Extended Release (Ace-ER; UX001) Tablets in Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy (GNEM) (Also Known as Hereditary Inclusion Body Myopathy [HIBM]) Patients With Severe Ambulatory Impairment

Hereditary Inclusion Body Myopathy | GNE Myopathy | Inclusion Body Myopathy 2 | Distal Myopathy With Rimmed Vacuoles | Distal Myopathy, Nonaka Type | Quadriceps Sparing Myopathy

Canada, United States, Bulgaria
Ultragenyx Pharmaceutical Inc

Not yet recruiting

Long-term Extension of GTX-102 in Angelman Syndrome

Angelman Syndrome

United States, Australia, Canada, Germany, United Kingdom, Israel, France, Spain
Ultragenyx Pharmaceutical Inc

Available

Expanded Access to Mepsevii

Sly Syndrome | Mucopolysaccharidosis VII | MPS VII
Ultragenyx Pharmaceutical Inc

Terminated

Adeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson Disease

Wilson Disease | Ornithine Transcarbamylase Deficiency | Glycogen Storage Disease Type IA

Spain, United Kingdom, Canada, United States, Brazil
Ultragenyx Pharmaceutical Inc

Completed

Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamylase (OTC) Deficiency (CAPtivate)

Ornithine Transcarbamylase (OTC) Deficiency

United Kingdom, United States, Canada, Spain
Ultragenyx Pharmaceutical Inc

Terminated

Observational Study of Males With Creatine Transporter Deficiency (Vigilan)

Creatine Deficiency, X-linked

United States, Canada
Ultragenyx Pharmaceutical Inc

Completed

A Study of UX007 (Triheptanoin) in Participants With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)

Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) | Very Long Chain Acyl-CoA Dehydrogenase (VLCAD) Deficiency | Trifunctional Protein (TFP) Deficiency | Carnitine Palmitoyltransferase (CPT II) Deficiency | Longchain 3-hydroxy-acyl-CoA Dehydrogenase (LCHAD) Deficiency

United States, United Kingdom
Ultragenyx Pharmaceutical Inc

Completed

Phase 2 Study of Triheptanoin (UX007) for the Treatment of Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

United States, Spain, Australia, France, Israel, Italy, United Kingdom
Ultragenyx Pharmaceutical Inc

Completed

Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM) (GNEM)

Hereditary Inclusion Body Myopathy | GNE Myopathy | Distal Myopathy With Rimmed Vacuoles | Distal Myopathy, Nonaka Type

United States, Canada, United Kingdom, Israel, Italy, Bulgaria, France
Ultragenyx Pharmaceutical Inc

Completed

An Open Label Phase 2 Extension Study of Higher Dose Sialic Acid-Extended Release (SA-ER) Tablets and Sialic Acid-Immediate Release (SA-IR) Capsules in Patients With Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy

GNE Myopathy | Hereditary Inclusion Body Myopathy (HIBM)

United States, Israel
Ultragenyx Pharmaceutical Inc

Completed

Safety and Pharmacokinetics of Sialic Acid Tables in Patients With Hereditary Inclusion Body Myopathy (HIBM)

Hereditary Inclusion Body Myopathy (HIBM)

United States
Ultragenyx Pharmaceutical Inc

Recruiting

A Study to Determine the Effect of Triheptanoin Compared With Even-chain, MCT on MCEs in Pediatric Patients With LC-FAOD

Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)

Turkey, United Kingdom, Spain, Saudi Arabia, Poland, Czechia, Germany
Ultragenyx Pharmaceutical Inc

Recruiting

Clinical Study of DTX301 AAV- Mediated Gene Transfer for Ornithine Transcarbamylase(OTC) Deficiency

OTC Deficiency

United States, Portugal, Italy, Spain, United Kingdom, Japan, Canada, Germany, France, Argentina, Netherlands, Brazil
Ultragenyx Pharmaceutical Inc

Recruiting

Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program

Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)

United States, Canada
Ultragenyx Pharmaceutical Inc

Available

Expanded Access to Triheptanoin

Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
Ultragenyx Pharmaceutical Inc

Completed

Retrospective Study of Glucose Monitoring for Glycemic Control in Patients With GSDIa

Glycogen Storage Disease Type IA

Netherlands, United States
Ultragenyx Pharmaceutical Inc

Active, not recruiting

Tumor-induced Osteomalacia Disease Monitoring Program

Tumor-induced Osteomalacia (TIO)

United States, Argentina
Ultragenyx Pharmaceutical Inc

Active, not recruiting

A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome

Angelman Syndrome

United States, United Kingdom, Canada, Australia, France, Germany, Spain, Israel
Ultragenyx Pharmaceutical Inc

Active, not recruiting

Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency (CAPtivate)

Ornithine Transcarbamylase (OTC) Deficiency

Canada, United States, France, Spain, United Kingdom
Ultragenyx Pharmaceutical Inc

Terminated

Safety, Tolerability, and Pharmacokinetics of UX053 in Patients With Glycogen Storage Disease Type III (GSD III)

Glycogen Storage Disease Type III

United States, Spain, Italy
Ultragenyx Pharmaceutical Inc

Active, not recruiting

A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)

Glycogen Storage Disease Type IA

United States, Brazil, Canada, Denmark, Germany, Italy, Netherlands, Spain
Ultragenyx Pharmaceutical Inc

Terminated

Clinical Survey Study to Assess Physical Function and the Incidence of Hypoglycemia in Participants With Glycogen Storage Disease Type III

Glycogen Storage Disease Type III

United States, Netherlands
Ultragenyx Pharmaceutical Inc

Completed

Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa)

GSD1

Netherlands, United States, Canada, Spain
Ultragenyx Pharmaceutical Inc

Completed

Study to Evaluate Biomarkers and Clinical Manifestations in Individuals With Glycogen Storage Disease Type III (GSD III)

Glycogen Storage Disease Type III

United States
Ultragenyx Pharmaceutical Inc

Terminated

Study to Characterize Rate of Ureagenesis in Patients With Ornithine Transcarbamylase (OTC) Deficiency

Ornithine Transcarbamylase Deficiency

United States
Ultragenyx Pharmaceutical Inc

Completed

Long-Term Safety, Tolerability, and Efficacy of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B

Hemophilia B

United States, United Kingdom
Ultragenyx Pharmaceutical Inc

Completed

Clinical Outcome of Triheptanoin Treatment in Patients With Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Treated Under Expanded Access Program

Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)

United States
Ultragenyx Pharmaceutical Inc

Terminated

Crossover Study to Assess the Efficacy and Safety of UX007 in the Treatment of Movement Disorders Associated With Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

United States, Spain, France, Germany, Italy, United Kingdom
Ultragenyx Pharmaceutical Inc

Completed

Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age

Mucopolysaccharidosis | Sly Syndrome | Mucopolysaccharidosis VII | MPS VII

Spain, United States, Portugal
Ultragenyx Pharmaceutical Inc

Completed

An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of Study Drug UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)

Mucopolysaccharidosis Type 7

United Kingdom
Ultragenyx Pharmaceutical Inc

Terminated

Safety and Dose Finding Study of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B

Hemophilia B

United States, United Kingdom, Bulgaria
Ultragenyx Pharmaceutical Inc

Completed

A Phase 2 Study to Evaluate the Dose and Pharmacodynamic Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy (GNE Myopathy)

Hereditary Inclusion Body Myopathy | GNE Myopathy

United States, Israel
Ultragenyx Pharmaceutical Inc
Mereo BioPharma

Completed

A Study in Adult Patients With Type I, III or IV Osteogenesis Imperfecta Treated With BPS804 (Asteroid)

Osteogenesis Imperfecta Type III | Osteogenesis Imperfecta Type IV | Osteogenesis Imperfecta, Type I

United States, Canada, Denmark, France, United Kingdom
Ultragenyx Pharmaceutical Inc
Newcastle University

Completed

GNE-Myopathy Disease Monitoring Program (GNEM-DMP): A Registry and Prospective Observational Natural History Study to Assess GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)

Hereditary Inclusion Body Myopathy | GNE Myopathy | Nonaka Disease | Quadriceps Sparing Myopathy (QSM) | Distal Myopathy With Rimmed Vacuoles (DMRV)

United States, Bulgaria, Canada, France, United Kingdom
Ultragenyx Pharmaceutical Inc
Mereo BioPharma

Active, not recruiting

Study to Assess Dose, Efficacy and Safety of Setrusumab in Participants With Osteogenesis Imperfecta (Orbit)

Osteogenesis Imperfecta

United States, Turkey, Germany, United Kingdom, Italy, Portugal, Canada, Australia, Argentina, France, Netherlands, Poland
Ultragenyx Pharmaceutical Inc
Mereo BioPharma

Terminated

Long-term Extension Study of Setrusumab in Adults With Type I, III, or IV Osteogenesis Imperfecta

Osteogenesis Imperfecta

United States
Rigshospitalet, Denmark
Ultragenyx Pharmaceutical Inc

Completed

The Effect of Triheptanoin on Fatty Acid Oxidation and Exercise Tolerance in Patients With Glycogenoses

Tarui Disease | Debrancher Deficiency | GYG1 DEFICIENCY

Denmark

Clinical Trials Sponsored by Ultragenix pharmaceutical

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Benin

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations