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ClinicalTrials.gov public records Last synced Oct 8, 2026, 5:52 PM EDT

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Showing 1–24 of 71 matching trials from the live ClinicalTrials.gov search.
Filters: Sponsor: Ultragenyx Pharmaceutical Inc Clear all
Active, not recruiting Phase 3 Interventional

Setrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis Imperfecta

NCT05768854
View directory record Official record
Conditions
Osteogenesis Imperfecta
Interventions
Bisphosphonate, Setrusumab
Drug · Biological
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
2 Years to 6 Years
Enrollment
69 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
13
States / cities
Phoenix, Arizona • Los Angeles, California • Aurora, Colorado + 10 more
Source: ClinicalTrials.gov public record
Updated Feb 25, 2026 · Synced Oct 8, 2026, 5:52 PM EDT
Completed Phase 3 Interventional Results available

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

NCT02230566
View directory record Official record
Conditions
MPS 7, Sly Syndrome, Mucopolysaccharidosis, MPS VII
Interventions
UX003, Placebo
Drug · Other
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
5 Years to 35 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
4
States / cities
Oakland, California • Orange, California • Miami, Florida + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 30, 2020 · Synced Oct 8, 2026, 5:52 PM EDT
Completed Early Phase 1 Interventional

Burosumab for CSHS

NCT03993821
View directory record Official record
Conditions
Cutaneous Skeletal Hypophosphatemia Syndrome (CSHS), Epidermal Nevus Syndrome
Interventions
Burosumab
Drug
Lead sponsor
Laura Tosi
Other
Eligibility
Not listed
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Jul 30, 2026 · Synced Oct 8, 2026, 5:52 PM EDT
Recruiting No phase listed Observational

Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program

NCT06636383
View directory record Official record
Conditions
Glycogen Storage Disease Type Ia
Interventions
No Intervention
Other
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
2 Years and older
Enrollment
140 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2036
U.S. locations
9
States / cities
Orange, California • Denver, Colorado • Hartford, Connecticut + 6 more
Source: ClinicalTrials.gov public record
Updated Aug 26, 2026 · Synced Oct 8, 2026, 5:52 PM EDT
Completed Phase 1 Interventional

Safety and Pharmacokinetics of Sialic Acid Tables in Patients With Hereditary Inclusion Body Myopathy (HIBM)

NCT01359319
View directory record Official record
Conditions
Hereditary Inclusion Body Myopathy (HIBM)
Interventions
Sialic Acid Extended Release (SA-ER) Tablets, Sialic Acid Extended Release (SA-ER) Tables
Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years to 70 Years
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2012
U.S. locations
2
States / cities
Costa Mesa, California • New York, New York
Source: ClinicalTrials.gov public record
Updated May 21, 2012 · Synced Oct 8, 2026, 5:52 PM EDT
Active, not recruiting No phase listed Observational

Tumor-induced Osteomalacia Disease Monitoring Program

NCT04783428
View directory record Official record
Conditions
Tumor-induced Osteomalacia (TIO)
Interventions
No intervention
Other
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
Not listed
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2032
U.S. locations
5
States / cities
New Haven, Connecticut • Bloomington, Indiana • Baltimore, Maryland + 2 more
Source: ClinicalTrials.gov public record
Updated Jun 3, 2026 · Synced Oct 8, 2026, 5:52 PM EDT
Completed Phase 2 Interventional Results available

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

NCT02418455
View directory record Official record
Conditions
Sly Syndrome, MPS VII, Mucopolysaccharidosis, Mucopolysaccharidosis VII
Interventions
UX003
Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
1 Day to 5 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
3
States / cities
Washington D.C., District of Columbia • New York, New York • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Oct 30, 2019 · Synced Oct 8, 2026, 5:52 PM EDT
Active, not recruiting No phase listed Observational

Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency

NCT03636438
View directory record Official record
Conditions
Ornithine Transcarbamylase (OTC) Deficiency
Interventions
No Intervention
Other
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years and older
Enrollment
11 participants
Timeline
2018 – 2029
U.S. locations
4
States / cities
Aurora, Colorado • Boston, Massachusetts • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 1, 2026 · Synced Oct 8, 2026, 5:52 PM EDT
Completed Phase 2 Interventional Results available

A Study of UX007 (Triheptanoin) in Participants With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)

NCT01886378
View directory record Official record
Conditions
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD), Carnitine Palmitoyltransferase (CPT II) Deficiency, Very Long Chain Acyl-CoA Dehydrogenase (VLCAD) Deficiency, Longchain 3-hydroxy-acyl-CoA Dehydrogenase (LCHAD) Deficiency, Trifunctional Protein (TFP) Deficiency
Interventions
UX007
Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
6 Months and older
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
7
States / cities
Washington D.C., District of Columbia • Tampa, Florida • Chicago, Illinois + 4 more
Source: ClinicalTrials.gov public record
Updated Feb 11, 2021 · Synced Oct 8, 2026, 5:52 PM EDT
Completed Phase 3 Interventional

Open Label Trial Assessing Safety and Efficacy of Burosumab (KRN23), in a Patient With ENS and Hypophosphatemic Rickets

NCT03581591
View directory record Official record
Conditions
Hypophosphatemia, Hypophosphatemic Rickets, Pain, Chronic
Interventions
Burosumab
Biological
Lead sponsor
Redwood Dermatology Sciences
Other
Eligibility
Up to 18 Years · Female only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
1
States / cities
Clinton, Utah
Source: ClinicalTrials.gov public record
Updated Jan 18, 2020 · Synced Oct 8, 2026, 5:52 PM EDT
Completed No phase listed Observational Results available

Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia

NCT03970278
View directory record Official record
Conditions
Glycogen Storage Disease Type IA, Von Gierke's Disease (GSD Type Ia)
Interventions
No intervention
Other
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
3
States / cities
Farmington, Connecticut • Ann Arbor, Michigan • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Mar 18, 2026 · Synced Oct 8, 2026, 5:52 PM EDT
Active, not recruiting Phase 3 Interventional

Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency

NCT05345171
View directory record Official record
Conditions
OTC Deficiency
Interventions
DTX301, Placebo, Oral Corticosteroids, Placebo for oral corticosteroids, Sodium Acetate
Genetic · Other · Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
12 Years and older
Enrollment
37 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2031
U.S. locations
4
States / cities
Los Angeles, California • Aurora, Colorado • Chicago, Illinois + 1 more
Source: ClinicalTrials.gov public record
Updated Sep 9, 2026 · Synced Oct 8, 2026, 5:52 PM EDT
Completed No phase listed Observational

Clinical Evaluation and Assessment of Instruments and Biomarkers in Subjects With Wilson Disease

NCT04531189
View directory record Official record
Conditions
Wilson Disease
Interventions
Not listed
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
12 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
5
States / cities
New Haven, Connecticut • Miami, Florida • Chicago, Illinois + 2 more
Source: ClinicalTrials.gov public record
Updated Apr 29, 2022 · Synced Oct 8, 2026, 5:52 PM EDT
Withdrawn Phase 2 Interventional

An Exploratory Study of BPS804 Treatment in Adult Patients With Type I, III or IV Osteogenesis Imperfecta

NCT03216486
View directory record Official record
Conditions
Osteogenesis Imperfecta
Interventions
BPS804
Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
2
States / cities
Albuquerque, New Mexico • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jul 3, 2023 · Synced Oct 8, 2026, 5:52 PM EDT
Completed No phase listed Observational

Clinical Outcome of Triheptanoin Treatment in Patients With Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Treated Under Expanded Access Program

NCT03768817
View directory record Official record
Conditions
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
Interventions
No Intervention
Other
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
Not listed
Enrollment
20 participants
Timeline
2019 – 2020
U.S. locations
15
States / cities
Los Angeles, California • Orange, California • Palo Alto, California + 11 more
Source: ClinicalTrials.gov public record
Updated Aug 28, 2020 · Synced Oct 8, 2026, 5:52 PM EDT
Completed No phase listed Observational

Long-Term Safety, Tolerability, and Efficacy of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B

NCT02971969
View directory record Official record
Conditions
Hemophilia B
Interventions
Not listed
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years and older · Male only
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
3
States / cities
Little Rock, Arkansas • Gainesville, Florida • Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Jan 6, 2022 · Synced Oct 8, 2026, 5:52 PM EDT
Completed Phase 2 Interventional

Safety, Pharmacokinetics and Pharmacodynamics of BPS804 in Osteogenesis Imperfecta

NCT01417091
View directory record Official record
Conditions
Osteogenesis Imperfecta
Interventions
BPS804
Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years to 75 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2012
U.S. locations
2
States / cities
Anaheim, California • Miramar, Florida
Source: ClinicalTrials.gov public record
Updated May 11, 2021 · Synced Oct 8, 2026, 5:52 PM EDT
No Longer Available No phase listed Expanded access

Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism

NCT01461304
View directory record Official record
Conditions
Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency, Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2), Mitochondrial Trifunctional Protein Deficiency, Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency, Glycogen Storage Disorders, Pyruvate Carboxylase Deficiency Disease, ACYL-CoA DEHYDROGENASE FAMILY, MEMBER 9, DEFICIENCY of, Barth Syndrome
Interventions
triheptanoin
Drug
Lead sponsor
Jerry Vockley, MD, PhD
Other
Eligibility
1 Month and older
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 10, 2021 · Synced Oct 8, 2026, 5:52 PM EDT
Terminated Phase 2 Interventional Results available

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

NCT02731690
View directory record Official record
Conditions
Hereditary Inclusion Body Myopathy, Distal Myopathy With Rimmed Vacuoles, Distal Myopathy, Nonaka Type, GNE Myopathy, Quadriceps Sparing Myopathy, Inclusion Body Myopathy 2
Interventions
Aceneuramic Acid Extended-Release
Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years and older
Enrollment
42 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
3
States / cities
Orange, California • St Louis, Missouri • New York, New York
Source: ClinicalTrials.gov public record
Updated Feb 19, 2019 · Synced Oct 8, 2026, 5:52 PM EDT
Completed Phase 3 Interventional Results available

A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)

NCT02432144
View directory record Official record
Conditions
Sly Syndrome, MPS VII, Mucopolysaccharidosis, Mucopolysaccharidosis VII
Interventions
UX003
Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
5 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
4
States / cities
Oakland, California • Orange, California • Minneapolis, Minnesota + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 30, 2020 · Synced Oct 8, 2026, 5:52 PM EDT
Recruiting Phase 1Phase 2 Interventional

First-in-human Study of UX016 in GNEM

NCT07511556
View directory record Official record
Conditions
GNE Myopathy
Interventions
UX016, Placebo
Drug · Other
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years to 55 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
2
States / cities
Orange, California • Iselin, New Jersey
Source: ClinicalTrials.gov public record
Updated Sep 3, 2026 · Synced Oct 8, 2026, 5:52 PM EDT
Terminated Phase 3 Interventional Results available

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)

NCT02736188
View directory record Official record
Conditions
Hereditary Inclusion Body Myopathy, Distal Myopathy With Rimmed Vacuoles, Distal Myopathy, Nonaka Type, GNE Myopathy, Quadriceps Sparing Myopathy
Interventions
Aceneuramic Acid Extended-Release Tablets
Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years and older
Enrollment
143 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
5
States / cities
Los Angeles, California • Orange, California • St Louis, Missouri + 1 more
Source: ClinicalTrials.gov public record
Updated Mar 24, 2023 · Synced Oct 8, 2026, 5:52 PM EDT
Completed Phase 2 Interventional Results available

An Open-Label Trial of Triheptanoin in Patients With Glucose Transporter Type-1 Deficiency Syndrome

NCT02036853
View directory record Official record
Conditions
Glucose Transporter Type-1 Deficiency Syndrome (Glut1 DS)
Interventions
Triheptanoin
Drug
Lead sponsor
Adrian Lacy
Other
Eligibility
1 Year to 50 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
1
States / cities
Fort Worth, Texas
Source: ClinicalTrials.gov public record
Updated Jan 28, 2021 · Synced Oct 8, 2026, 5:52 PM EDT