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
Study
Completion

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
Study
Completion

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
Study
Completion

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
Study
Completion

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
Study
Completion

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
Study
Completion

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
Study
Completion

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
Study
Completion

July 2016

Trial ID

UX003-CL301

Trial Title

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

Condition

Mucopolysaccharidosis VII (MPS VII)

Study Drug

Vestronidase alfa

Completion

May 2016