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 | |
|---|---|---|---|---|---|---|
| 101HEMB02 | Long-Term Follow-up Study to Evaluate the Safety, Tolerability, and Efficacy of Adeno-Associated Virus (AAV) rh10-Mediated Gene Transfer of Human Factor IX in Adults With Moderate/Severe to Severe Hemophilia B | Hemophilia B | DTX 101 |
Study >>
Summary >> |
December 2021 | 12/01/202101/01/2030 |
| 301OTC01 | Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset OTC Deficiency | Ornithine Transcarbamylase (OTC) Deficiency | DTX 301 |
Study >>
Summary >> |
December 2021 | 12/01/2021 |
| 401GSDIA01 | Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa) | Glycogen Storage Disease Type Ia (GSDIa) | DTX 401 |
Study >>
Summary >> |
November 2021 | 11/01/2021 |
| 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 >>
Summary >> |
January 2021 | 01/01/2021 |
| MBPS205 | A Study in Adult Patients With Type I, III or IV Osteogenesis Imperfecta Treated With BPS804 (ASTEROID) | Osteogenesis Imperfecta (OI) | Setrusumab |
Study >>
Summary >> |
November 2020 | 11/01/2020 |
| UX007-CL202 | An Open-label Long-Term Safety and Efficacy Extension Study in Subjects with Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Previously Enrolled in UX007 | Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) | Triheptanoin |
Study >>
Summary >> |
October 2020 | 10/01/2020 |
| 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 >>
Summary >> |
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 |
CONDITIONHemophilia B
TRIAL TITLELong-Term Follow-up Study to Evaluate the Safety, Tolerability, and Efficacy of Adeno-Associated Virus (AAV) rh10-Mediated Gene Transfer of Human Factor IX in Adults With Moderate/Severe to Severe Hemophilia B
STUDY DRUGDTX 101
TRIAL ID101HEMB02
COMPLETION
December 2021
CONDITIONOrnithine Transcarbamylase (OTC) Deficiency
TRIAL TITLESafety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset OTC Deficiency
STUDY DRUGDTX 301
TRIAL ID301OTC01
COMPLETION
December 2021
CONDITIONGlycogen Storage Disease Type Ia (GSDIa)
TRIAL TITLESafety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa)
STUDY DRUGDTX 401
TRIAL ID401GSDIA01
COMPLETION
November 2021
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
January 2021
CONDITIONOsteogenesis Imperfecta (OI)
TRIAL TITLEA Study in Adult Patients With Type I, III or IV Osteogenesis Imperfecta Treated With BPS804 (ASTEROID)
STUDY DRUGSetrusumab
TRIAL IDMBPS205
COMPLETION
November 2020
RESULTS
Study >>
CONDITIONLong-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
TRIAL TITLEAn Open-label Long-Term Safety and Efficacy Extension Study in Subjects with Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Previously Enrolled in UX007
STUDY DRUGTriheptanoin
TRIAL IDUX007-CL202
COMPLETION
October 2020
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
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
