Our clinical trial transparency commitment
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 | |
|---|---|---|---|---|---|---|
| UX053-CL101 | A Phase 1/2 First-In-Human, Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Single Ascending Doses and Repeat Doses of UX053 in Patients With GSD III | Glycogen Storage Disease Type III (GSD III) | UX053 | March 2023 | 03/01/2023 | |
| 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 | December 2021 | 12/01/2021 | |
| 301OTC01 | Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset OTC Deficiency | Ornithine Transcarbamylase (OTC) Deficiency | DTX 301 | December 2021 | 12/01/2021 | |
| 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 | February 2017 | 02/01/2017 | |
| 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 | November 2021 | 11/01/2021 | |
| MBPS205 | A Study in Adult Patients With Type I, III or IV Osteogenesis Imperfecta Treated With BPS804 (ASTEROID) | Osteogenesis Imperfecta (OI) | Setrusumab | 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 | 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 | 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 | October 2019 | 10/01/2019 | |
| 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 | 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 | January 2019 | 01/01/2019 | |
| 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 | 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 | 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 | 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 | 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 | June 2017 | 06/01/2017 | |
| 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 | 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 | 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 | May 2016 | 05/01/2016 |
Trial ID
UX053-CL101
Trial Title
A Phase 1/2 First-In-Human, Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Single Ascending Doses and Repeat Doses of UX053 in Patients With GSD III
Condition
Glycogen Storage Disease Type III (GSD III)
Study Drug
UX053
Completion
March 2023
Trial ID
101HEMB02
Trial Title
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
Condition
Hemophilia B
Study Drug
DTX 101
Completion
December 2021
Trial ID
301OTC01
Trial Title
Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset OTC Deficiency
Condition
Ornithine Transcarbamylase (OTC) Deficiency
Study Drug
DTX 301
Completion
December 2021
Trial ID
UX001-CL202
Trial Title
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
Condition
GNE Myopathy (GNEM)
Study Drug
UX001
Results
StudyCompletion
February 2017
Trial ID
401GSDIA01
Trial Title
Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa)
Condition
Glycogen Storage Disease Type Ia (GSDIa)
Study Drug
DTX 401
Completion
November 2021
Trial ID
MBPS205
Trial Title
A Study in Adult Patients With Type I, III or IV Osteogenesis Imperfecta Treated With BPS804 (ASTEROID)
Condition
Osteogenesis Imperfecta (OI)
Study Drug
Setrusumab
Completion
November 2020
Trial ID
UX007-CL202
Trial Title
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
Condition
Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
Study Drug
Triheptanoin
Completion
October 2020
Trial ID
UX007G-CL301
Trial Title
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)
Condition
Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
Study Drug
Triheptanoin
Completion
October 2019
Trial ID
UX007G-CL202
Trial Title
Study to Assess the Long Term Safety and Efficacy of UX007 in Subjects With Glucose Type 1 DS
Condition
Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
Study Drug
Triheptanoin
Results
StudyCompletion
October 2019
Trial ID
UX003-CL203
Trial Title
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
Condition
Mucopolysaccharidosis VII (MPS VII)
Study Drug
Vestronidase alfa
Results
StudyCompletion
March 2019
Trial ID
UX003-CL202
Trial Title
A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)
Condition
Mucopolysaccharidosis VII (MPS VII)
Study Drug
Vestronidase alfa
Completion
January 2019
Trial ID
UX001-CL302
Trial Title
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)
Condition
GNE Myopathy (GNEM)
Study Drug
UX001
Results
StudyCompletion
January 2018
Trial ID
UX001-CL203
Trial Title
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
Condition
GNE Myopathy (GNEM)
Study Drug
UX001
Results
StudyCompletion
January 2018
Trial ID
101HEMB01
Trial Title
Safety and Dose Finding Study of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B
Condition
Hemophilia B
Study Drug
DTX 101
Completion
October 2017
Trial ID
UX007G-CL201
Trial Title
Phase 2 Study of Triheptanoin (UX007) for the Treatment of Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
Condition
Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
Study Drug
Triheptanoin
Completion
September 2017
Trial ID
UX001-CL301
Trial Title
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)
Condition
GNE Myopathy (GNEM)
Study Drug
UX001
Results
StudyCompletion
June 2017
Trial ID
UX007-CL201
Trial Title
An Open-label Phase 2 Study of UX007 (Triheptanoin) in Subjects With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
Condition
Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
Study Drug
Triheptanoin
Results
StudyCompletion
August 2016
Trial ID
UX003-CL201
Trial Title
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)
Condition
Mucopolysaccharidosis VII (MPS VII)
Study Drug
Vestronidase alfa
Results
StudyCompletion
July 2016