Ultragenyx Clinical Trial Results

Ultragenyx Pharmaceutical Inc. is committed to transparency in its activities. To that end and in accordance with applicable law and regulations, Ultragenyx shares clinical trial results with trial participants and the public. Learn more about the results from our clinical trials below.

Trial ID Trial Title Condition Study Drug Results Completion  
UX007G-CL301 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) Triheptanoin Study >>
Summary >>
October 2019 10/01/2019
UX007G-CL202 Study to Assess the Long Term Safety and Efficacy of UX007 in Subjects With Glucose Type 1 DS Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) Triheptanoin Study >> October 2019 10/01/2019
UX023-CL205 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 Hyposphosphatemia (XLH) Burosumab Study >> September 2019 09/01/2019
UX023-CL301 Efficacy and Safety of Burosumab (KRN23) Versus Oral Phosphate and Active Vitamin D Treatment in Pediatric Patients With X Linked Hypophosphatemia (XLH) X Linked Hyposphosphatemia (XLH) Burosumab Study >>
Summary >>
July 2019 07/01/2019
UX003-CL203 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 VII (MPS VII) Vestronidase alfa Study >> March 2019 03/01/2019
UX003-CL202 A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Mucopolysaccharidosis VII (MPS VII) Vestronidase alfa Study >>
Summary >>
January 2019 01/01/2019
UX023-CL304 Open Label Study of KRN23 on Osteomalacia in Adults With X-linked Hypophosphatemia (XLH) X Linked Hyposphosphatemia (XLH) Burosumab Study >> December 2018 12/01/2018
UX023-CL303 A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study with Open-Label Extension to Assess the Efficacy and Safety of KRN23 in Adults with X-linked Hypophosphatemia (XLH) X Linked Hyposphosphatemia (XLH) Burosumab Study >> December 2018 12/01/2018
UX023-CL203 Long-Term Extension Study of KRN23 in Adult Subjects With X-Linked Hypophosphatemia (XLH) X Linked Hyposphosphatemia (XLH) Burosumab Study >> November 2018 11/01/2018
UX023-CL201 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 Hyposphosphatemia (XLH) Burosumab Study >>
Summary >>
October 2018 10/01/2018
UX001-CL302 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) GNE Myopathy (GNEM) UX001 Study >> January 2018 01/01/2018
UX001-CL203 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 GNE Myopathy (GNEM) UX001 Study >> January 2018 01/01/2018
101HEMB01 Safety and Dose Finding Study of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B Hemophilia B DTX 101 Study >> October 2017 10/01/2017
UX007G-CL201 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) Triheptanoin Study >>
Summary >>
September 2017 09/01/2017
UX001-CL301 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) GNE Myopathy (GNEM) UX001 Study >> June 2017 06/01/2017
UX001-CL202 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 (GNEM) UX001 Study >> February 2017 02/01/2017
UX007-CL201 An Open-label Phase 2 Study of UX007 (Triheptanoin) in Subjects With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Triheptanoin Study >> August 2016 08/01/2016
UX003-CL201 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 VII (MPS VII) Vestronidase alfa Study >> July 2016 07/01/2016
UX003-CL301 A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7) Mucopolysaccharidosis VII (MPS VII) Vestronidase alfa Study >>
Summary >>
May 2016 05/01/2016
UX023T-CL201 A Phase 2 Open-Label Trial to Assess the Efficacy and Safety of KRN23, an Antibody to FGF23, in Subjects With Tumor-Induced Osteomalacia (TIO) or Epidermal Nevus Syndrome (ENS)-Associated Osteomalacia Tumor-Induced Osteomalacia (TIO) Burosumab Study >> Ongoing 01/01/2030
CONDITIONTumor-Induced Osteomalacia (TIO)
TRIAL TITLEA Phase 2 Open-Label Trial to Assess the Efficacy and Safety of KRN23, an Antibody to FGF23, in Subjects With Tumor-Induced Osteomalacia (TIO) or Epidermal Nevus Syndrome (ENS)-Associated Osteomalacia
STUDY DRUGBurosumab
TRIAL IDUX023T-CL201
COMPLETION Ongoing
RESULTS Study >>
CONDITIONGlucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
TRIAL TITLECrossover 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)
STUDY DRUGTriheptanoin
TRIAL IDUX007G-CL301
COMPLETION October 2019
CONDITIONGlucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
TRIAL TITLEStudy to Assess the Long Term Safety and Efficacy of UX007 in Subjects With Glucose Type 1 DS
STUDY DRUGTriheptanoin
TRIAL IDUX007G-CL202
COMPLETION October 2019
RESULTS Study >>
CONDITIONX Linked Hyposphosphatemia (XLH)
TRIAL TITLEStudy of the Safety, Pharmacodynamics (PD) and Efficacy of KRN23 in Children From 1 to 4 Years Old With X-linked Hypophosphatemia (XLH)
STUDY DRUGBurosumab
TRIAL IDUX023-CL205
COMPLETION September 2019
RESULTS Study >>
CONDITIONX Linked Hyposphosphatemia (XLH)
TRIAL TITLEEfficacy and Safety of Burosumab (KRN23) Versus Oral Phosphate and Active Vitamin D Treatment in Pediatric Patients With X Linked Hypophosphatemia (XLH)
STUDY DRUGBurosumab
TRIAL IDUX023-CL301
COMPLETION July 2019
CONDITIONMucopolysaccharidosis VII (MPS VII)
TRIAL TITLEStudy 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
STUDY DRUGVestronidase alfa
TRIAL IDUX003-CL203
COMPLETION March 2019
RESULTS Study >>
CONDITIONMucopolysaccharidosis VII (MPS VII)
TRIAL TITLEA Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)
STUDY DRUGVestronidase alfa
TRIAL IDUX003-CL202
COMPLETION January 2019
CONDITIONX Linked Hyposphosphatemia (XLH)
TRIAL TITLEOpen Label Study of KRN23 on Osteomalacia in Adults With X-linked Hypophosphatemia (XLH)
STUDY DRUGBurosumab
TRIAL IDUX023-CL304
COMPLETION December 2018
RESULTS Study >>
CONDITIONX Linked Hyposphosphatemia (XLH)
TRIAL TITLEA Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study with Open-Label Extension to Assess the Efficacy and Safety of KRN23 in Adults with X-linked Hypophosphatemia (XLH)
STUDY DRUGBurosumab
TRIAL IDUX023-CL303
COMPLETION December 2018
RESULTS Study >>
CONDITIONX Linked Hyposphosphatemia (XLH)
TRIAL TITLELong-Term Extension Study of KRN23 in Adult Subjects With X-Linked Hypophosphatemia (XLH)
STUDY DRUGBurosumab
TRIAL IDUX023-CL203
COMPLETION November 2018
RESULTS Study >>
CONDITIONX Linked Hyposphosphatemia (XLH)
TRIAL TITLEStudy of KRN23 (Burosumab), a Recombinant Fully Human Monoclonal Antibody Against Fibroblast Growth Factor 23 (FGF23), in Pediatric Subjects With X-linked Hypophosphatemia (XLH)
STUDY DRUGBurosumab
TRIAL IDUX023-CL201
COMPLETION October 2018
CONDITIONGNE Myopathy (GNEM)
TRIAL TITLEStudy 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)
STUDY DRUGUX001
TRIAL IDUX001-CL302
COMPLETION January 2018
RESULTS Study >>
CONDITIONGNE Myopathy (GNEM)
TRIAL TITLEA 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
STUDY DRUGUX001
TRIAL IDUX001-CL203
COMPLETION January 2018
RESULTS Study >>
CONDITIONHemophilia B
TRIAL TITLESafety and Dose Finding Study of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B
STUDY DRUGDTX 101
TRIAL ID101HEMB01
COMPLETION October 2017
RESULTS Study >>
CONDITIONGlucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
TRIAL TITLEPhase 2 Study of Triheptanoin (UX007) for the Treatment of Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
STUDY DRUGTriheptanoin
TRIAL IDUX007G-CL201
COMPLETION September 2017
CONDITIONGNE Myopathy (GNEM)
TRIAL TITLEPhase 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)
STUDY DRUGUX001
TRIAL IDUX001-CL301
COMPLETION June 2017
RESULTS Study >>
CONDITIONGNE Myopathy (GNEM)
TRIAL TITLEAn 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
STUDY DRUGUX001
TRIAL ID UX001-CL202
COMPLETION February 2017
RESULTS Study >>
CONDITIONLong-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
TRIAL TITLEAn Open-label Phase 2 Study of UX007 (Triheptanoin) in Subjects With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
STUDY DRUGTriheptanoin
TRIAL IDUX007-CL201
COMPLETION August 2016
RESULTS Study >>
CONDITIONMucopolysaccharidosis VII (MPS VII)
TRIAL TITLEAn 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)
STUDY DRUGVestronidase alfa
TRIAL IDUX003-CL201
COMPLETION July 2016
RESULTS Study >>
CONDITIONMucopolysaccharidosis VII (MPS VII)
TRIAL TITLEA Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)
STUDY DRUGVestronidase alfa
TRIAL IDUX003-CL301
COMPLETION May 2016