Search clinical trials for: UX003

Total 34 results

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
Joyce Fox
Ultragenyx Pharmaceutical Inc

No longer available

An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient

Mucopolysaccharidosis Type 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
Institut National de la Santé Et de la Recherche...
Rigshospitalet, Denmark

Unknown

Triheptanoin in Mc Ardle

Glycogen Storage Disease Type V
Rigshospitalet, Denmark
University of Texas Southwestern Medical Center; Groupe Hospitalier Pitie-Salpetriere and other collaborators

Completed

The Effect of Triheptanoin in Adults With McArdle Disease (Glycogen Storage Disease Type V)

Glycogen Storage Disease Type V

Denmark
Ultragenyx Pharmaceutical Inc

Available

Expanded Access to Mepsevii

Sly Syndrome | Mucopolysaccharidosis VII | MPS VII
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
Areeg El-Gharbawy
Ultragenyx Pharmaceutical Inc

Completed

Anaplerotic Therapy Using Triheptanoin for Patients With Glycogen Storage Disease Type I

Glycogen Storage Disease Type I

United States
University of Liege

Unknown

A Pilot Trial of Triheptanoin for the Preventive Treatment of Migraine (TRIMI)

Migraine
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
Jerry Vockley, MD, PhD
Ultragenyx Pharmaceutical Inc

No longer available

Treatment With UX007 for a Single Patient With GLUT1 Deficiency Syndrome

Glucose Transporter 1 Deficiency Syndrome

United States
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
Kyowa Kirin Co., Ltd.

Available

Expanded Access to Burosumab

X-linked Hypophosphatemia | Tumor-Induced Osteomalacia
Hadassah Medical Organization

Completed

Efficacy of Sialic Acid GNE Related Thrombocytopenia (SA-thrombo)

Thrombocytopenia
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

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
Kyowa Kirin, Inc.
Kyowa Kirin Co., Ltd.

Completed

Open Label Study of KRN23 on Osteomalacia in Adults With X-linked Hypophosphatemia (XLH)

X-linked Hypophosphatemia

United States, Korea, Republic of, Canada, Japan, France, Denmark
Kyowa Kirin, Inc.

Completed

Study of Burosumab (KRN23) in Adults With Tumor-Induced Osteomalacia (TIO) or Epidermal Nevus Syndrome (ENS)

Tumor Induced Osteomalacia (TIO) | Epidermal Nevus Syndrome (ENS)

United States
Kyowa Kirin, Inc.

Completed

Study of the Safety, Pharmacodynamics (PD) and Efficacy of KRN23 in Children From 1 to 4 Years Old With X-linked Hypophosphatemia (XLH)

X-Linked Hypophosphatemia

United States
Ultragenyx Pharmaceutical Inc

Available

Expanded Access to Triheptanoin

Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
Irina A Anselm

No longer available

Triheptanoin (UX007) to Treat Citrate Transporter Deficiency

Citrate Transporter Deficiency | SLC13A5 Gene Mutation

United States
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
Kyowa Kirin, Inc.
Kyowa Kirin Co., Ltd.

Completed

Study of KRN23 in Adults With X-linked Hypophosphatemia (XLH)

X-linked Hypophosphatemia

United States, Korea, Republic of, Japan, Ireland, United Kingdom, France, Italy
Kyowa Kirin, Inc.
Kyowa Kirin Co., Ltd.

Completed

Study of KRN23 (Burosumab), a Recombinant Fully Human Monoclonal Antibody Against Fibroblast Growth Factor 23 (FGF23), in Pediatric Subjects With X-linked Hypophosphatemia (XLH)

X-linked Hypophosphatemia

Netherlands, United States, United Kingdom, France
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

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

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
Institut National de la Santé Et de la Recherche...

Completed

Pilot Study, Comparative, Single-center, Randomized, Crossover, Double-blind, Against Placebo, Testing the Effectiveness of Triheptanoin Oil in Alternating Hemiplegia of Childhood (HEMIHEP)

Alternating Hemiplegia of Childhood

France
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
Kyowa Kirin, Inc.
Kyowa Kirin Co., Ltd.

Completed

Efficacy and Safety of Burosumab (KRN23) Versus Oral Phosphate and Active Vitamin D Treatment in Pediatric Patients With X Linked Hypophosphatemia (XLH)

X-Linked Hypophosphatemia

Korea, Republic of, Canada, United States, Australia, Japan, United Kingdom, Sweden
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
Zogenix, Inc.

Completed

A Safety and PK Study of Injectable Risperidone-SABER and the DosePro Delivery System in Patients With Schizophrenia

Schizophrenia

United States
Zogenix, Inc.

Completed

Safety and PK Trial With Injectable ZX003 (Risperidone-SABER®) Compared to Risperdal® Consta® in Stable Schizophrenia

Schizophrenia | Schizoaffective Disorder

United States
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

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