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ClinicalTrials.gov public records Last synced Oct 10, 2026, 2:31 AM EDT

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Showing 49–71 of 71 matching trials from the live ClinicalTrials.gov search.
Filters: Sponsor: Ultragenyx Pharmaceutical Inc Clear all
Completed No phase listed Observational

Retrospective Study of Glucose Monitoring for Glycemic Control in Patients With GSDIa

NCT04708015
View directory record Official record
Conditions
Glycogen Storage Disease Type IA
Interventions
No Intervention
Other
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
8 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated May 29, 2024 · Synced Oct 10, 2026, 2:31 AM 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 10, 2026, 2:31 AM 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 10, 2026, 2:31 AM EDT
No Longer Available No phase listed Expanded access

Treatment With UX007 for a Single Patient With GLUT1 Deficiency Syndrome

NCT02968953
View directory record Official record
Conditions
Glucose Transporter 1 Deficiency Syndrome
Interventions
Triheptanoin
Drug
Lead sponsor
Jerry Vockley, MD, PhD
Other
Eligibility
3 Years and older · Female only
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 8, 2019 · Synced Oct 10, 2026, 2:31 AM EDT
Terminated No phase listed Observational

Long-Chain Fatty Acid Oxidation Disorders Online Disease Monitoring Program

NCT04812106
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
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Nov 18, 2022 · Synced Oct 10, 2026, 2:31 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease

NCT04088734
View directory record Official record
Conditions
MPS IIIA, Sanfilippo Syndrome, Sanfilippo A, Mucopolysaccharidosis III
Interventions
ABO-102
Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
2 Years to 18 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 25, 2023 · Synced Oct 10, 2026, 2:31 AM EDT
Active, not recruiting Phase 2Phase 3 Interventional

Setrusumab vs Placebo for Osteogenesis Imperfecta

NCT05125809
View directory record Official record
Conditions
Osteogenesis Imperfecta
Interventions
Setrusumab, Placebo
Biological · Other
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
5 Years to 25 Years
Enrollment
183 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
22
States / cities
Little Rock, Arkansas • Los Angeles, California • Sacramento, California + 19 more
Source: ClinicalTrials.gov public record
Updated Oct 6, 2026 · Synced Oct 10, 2026, 2:31 AM 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 10, 2026, 2:31 AM EDT
Terminated No phase listed Observational

Adeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson Disease

NCT04909346
View directory record Official record
Conditions
Ornithine Transcarbamylase Deficiency, Wilson Disease, Glycogen Storage Disease Type IA
Interventions
Not listed
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years and older
Enrollment
51 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
1
States / cities
Akron, Ohio
Source: ClinicalTrials.gov public record
Updated Jun 12, 2023 · Synced Oct 10, 2026, 2:31 AM 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 10, 2026, 2:31 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamylase (OTC) Deficiency

NCT02991144
View directory record Official record
Conditions
Ornithine Transcarbamylase (OTC) Deficiency
Interventions
scAAV8OTC, Reactive Corticosteroid Taper Regimen, Prophylactic Corticosteroid Taper Regimen
Genetic · Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
4
States / cities
Aurora, Colorado • Boston, Massachusetts • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Jan 26, 2023 · Synced Oct 10, 2026, 2:31 AM EDT
Completed Phase 2 Interventional Results available

A Study in Adult Patients With Type I, III or IV Osteogenesis Imperfecta Treated With BPS804

NCT03118570
View directory record Official record
Conditions
Osteogenesis Imperfecta, Type I, Osteogenesis Imperfecta Type III, Osteogenesis Imperfecta Type IV
Interventions
setrusumab, Calcium, Vitamin D, zoledronic acid (optional)
Drug · Dietary Supplement
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years to 75 Years
Enrollment
112 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
12
States / cities
Birmingham, Alabama • Jacksonville, Florida • Baltimore, Maryland + 9 more
Source: ClinicalTrials.gov public record
Updated Jul 5, 2023 · Synced Oct 10, 2026, 2:31 AM 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 10, 2026, 2:31 AM EDT
Enrolling by invitation No phase listed Observational Accepts healthy volunteers

ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program

NCT05368038
View directory record Official record
Conditions
Acid Sphingomyelinase Deficiency, Ceroid Lipofuscinosis, Neuronal, 2, Cerebrotendinous Xanthomatosis, Fabry Disease, GM1 Gangliosidosis, Gaucher Disease, Lysosomal Acid Lipase Deficiency, Metachromatic Leukodystrophy, Mucopolysaccharidosis II, Mucopolysaccharidosis III-B, Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI, Mucopolysaccharidosis VII, Niemann-Pick Disease, Type C
Interventions
Confirmatory Testing
Diagnostic Test
Lead sponsor
Albert Einstein College of Medicine
Other
Eligibility
Up to 4 Weeks
Enrollment
100,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2029
U.S. locations
9
States / cities
Brooklyn, New York • Manhasset, New York • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Jul 28, 2026 · Synced Oct 10, 2026, 2:31 AM 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 10, 2026, 2:31 AM EDT
Recruiting Phase 1 Interventional

Triheptanoin for Children With Primary-Specific Pyruvate Dehydrogenase Complex (PDC) Deficiency

NCT06340685
View directory record Official record
Conditions
Pyruvate Dehydrogenase Complex Deficiency
Interventions
Triheptanoin
Drug
Lead sponsor
Jirair Krikor Bedoyan
Other
Eligibility
1 Year to 17 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 17, 2026 · Synced Oct 10, 2026, 2:31 AM 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 10, 2026, 2:31 AM EDT
Active, not recruiting Phase 2 Interventional

A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)

NCT07157254
View directory record Official record
Conditions
Angelman Syndrome
Interventions
No intervention, GTX-102
Other · Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
1 Year to 64 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
8
States / cities
Los Angeles, California • Chicago, Illinois • Baltimore, Maryland + 5 more
Source: ClinicalTrials.gov public record
Updated Sep 9, 2026 · Synced Oct 10, 2026, 2:31 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

A Study to Assess Plasma Ammonia Time-Normalized Area Under the Curve and Rate of Ureagenesis in Healthy Adult Subjects

NCT04269122
View directory record Official record
Conditions
Ornithine Transcarbamylase Deficiency
Interventions
No Intervention
Other
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years to 55 Years
Enrollment
120 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2020
U.S. locations
1
States / cities
Austin, Texas
Source: ClinicalTrials.gov public record
Updated Mar 30, 2020 · Synced Oct 10, 2026, 2:31 AM EDT
Terminated Phase 2 Interventional Results available

Study to Assess the Long Term Safety and Efficacy of UX007 in Participants With Glucose Type 1 Deficiency Syndrome (Glut1 DS)

NCT02599961
View directory record Official record
Conditions
Glucose Transporter Type 1 Deficiency Syndrome
Interventions
UX007
Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
1 Year and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
5
States / cities
Aurora, Colorado • Miami, Florida • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Jun 11, 2020 · Synced Oct 10, 2026, 2:31 AM EDT
Completed Phase 1Phase 2 Interventional Accepts healthy volunteers Results available

A Pilot Trial of Triheptanoin for People With Amyotrophic Lateral Sclerosis (PALS)

NCT03506425
View directory record Official record
Conditions
ALS
Interventions
Triheptanoin
Drug
Lead sponsor
Richard Bedlack, M.D., Ph.D.
Other
Eligibility
18 Years and older
Enrollment
15 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2019
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Dec 24, 2019 · Synced Oct 10, 2026, 2:31 AM EDT
Active, not recruiting Phase 3 Interventional

Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)

NCT06617429
View directory record Official record
Conditions
Angelman Syndrome
Interventions
GTX-102, Sham-LP
Drug · Procedure
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
4 Years to 17 Years
Enrollment
129 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
14
States / cities
Los Angeles, California • San Diego, California • San Francisco, California + 11 more
Source: ClinicalTrials.gov public record
Updated Aug 6, 2026 · Synced Oct 10, 2026, 2:31 AM EDT
Active, not recruiting Phase 3 Interventional

Long-term Extension of GTX-102 in Angelman Syndrome

NCT06415344
View directory record Official record
Conditions
Angelman Syndrome
Interventions
GTX-102
Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
Not listed
Enrollment
255 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
6
States / cities
Los Angeles, California • San Diego, California • Atlanta, Georgia + 3 more
Source: ClinicalTrials.gov public record
Updated Sep 9, 2026 · Synced Oct 10, 2026, 2:31 AM EDT