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ULTRAGENYX PHARMACEUTICAL INC

ULTRAGENYX PHARMACEUTICAL INC logo
🇪🇸Spain
Ownership
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Employees
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Market Cap
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Website

Setrusumab in Pediatric Japanese Subjects With Osteogenesis Imperfecta

Phase 3
Active, not recruiting
Conditions
Osteogenesis Imperfecta
Interventions
First Posted Date
2024-10-10
Last Posted Date
2025-05-13
Lead Sponsor
Ultragenyx Pharmaceutical Inc
Target Recruit Count
6
Registration Number
NCT06636071
Locations
🇯🇵

Osaka Metropolitan University Hospital, Osaka, Japan

🇯🇵

Osaka University Hospital, Osaka, Japan

🇯🇵

Keio University Hospital, Tokyo, Japan

Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program

Recruiting
Conditions
Glycogen Storage Disease Type Ia
First Posted Date
2024-10-10
Last Posted Date
2025-07-08
Lead Sponsor
Ultragenyx Pharmaceutical Inc
Target Recruit Count
140
Registration Number
NCT06636383
Locations
🇺🇸

Children's Hospital of Orange County, Orange, California, United States

🇺🇸

Children's Hospital Colorado, Denver, Colorado, United States

🇺🇸

University of Connecticut Health Center, Hartford, Connecticut, United States

and more 16 locations

Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With AS

Phase 3
Recruiting
Conditions
Angelman Syndrome
Interventions
Procedure: Sham-LP
First Posted Date
2024-09-27
Last Posted Date
2025-06-22
Lead Sponsor
Ultragenyx Pharmaceutical Inc
Target Recruit Count
120
Registration Number
NCT06617429
Locations
🇺🇸

Cedars Sinai, Los Angeles, California, United States

🇺🇸

UCSD, Rady Children's Hospital, San Diego, California, United States

🇺🇸

UCSF, San Francisco, California, United States

and more 25 locations

Long-term Extension of GTX-102 in Angelman Syndrome

Phase 3
Conditions
Angelman Syndrome
Interventions
First Posted Date
2024-05-16
Last Posted Date
2025-04-16
Lead Sponsor
Ultragenyx Pharmaceutical Inc
Target Recruit Count
75
Registration Number
NCT06415344
Locations
🇺🇸

University of California, Los Angeles (UCLA), Los Angeles, California, United States

🇺🇸

University of California, San Diego - Rady Children's Hospital, San Diego, California, United States

🇺🇸

Rare Disease Research, LLC, Atlanta, Georgia, United States

and more 20 locations

A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAOD

Phase 3
Recruiting
Conditions
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
Interventions
Dietary Supplement: MCT Oil
First Posted Date
2023-07-06
Last Posted Date
2025-06-12
Lead Sponsor
Ultragenyx Pharmaceutical Inc
Target Recruit Count
60
Registration Number
NCT05933200
Locations
🇨🇿

General University Hospital in Prague-GUH (Všeobecná fakultní nemocnice v Praze- VFN), Prague, Czechia

🇩🇪

Universitätsklinikum Freiburg, Freiburg, Germany

🇯🇵

Juntendo University Hospital, Bunkyo City, Tokyo, Japan

and more 12 locations

Setrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis Imperfecta

Phase 3
Active, not recruiting
Conditions
Osteogenesis Imperfecta
Interventions
Drug: Bisphosphonate
First Posted Date
2023-03-14
Last Posted Date
2025-04-06
Lead Sponsor
Ultragenyx Pharmaceutical Inc
Target Recruit Count
69
Registration Number
NCT05768854
Locations
🇺🇸

Washington University School of Medicine, Saint Louis, Missouri, United States

🇺🇸

Phoenix Children's Hospital, Phoenix, Arizona, United States

🇺🇸

Childrens Hospital LA, Los Angeles, California, United States

and more 18 locations

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

Phase 3
Active, not recruiting
Conditions
OTC Deficiency
Interventions
Genetic: DTX301
Other: Placebo
Drug: Oral Corticosteroids
Drug: Placebo for oral corticosteroids
First Posted Date
2022-04-25
Last Posted Date
2025-06-15
Lead Sponsor
Ultragenyx Pharmaceutical Inc
Target Recruit Count
32
Registration Number
NCT05345171
Locations
🇺🇸

University of California, Los Angeles, California, United States

🇺🇸

University of Colorado, Aurora, Colorado, United States

🇺🇸

Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States

and more 14 locations

Long-term Extension Study of Setrusumab in Adults With Type I, III, or IV Osteogenesis Imperfecta

Phase 2
Terminated
Conditions
Osteogenesis Imperfecta
First Posted Date
2022-04-05
Last Posted Date
2023-06-29
Lead Sponsor
Ultragenyx Pharmaceutical Inc
Target Recruit Count
2
Registration Number
NCT05312697
Locations
🇺🇸

New Mexico Clinical Research & Osteoporosis Center, Albuquerque, New Mexico, United States

Clinical Survey Study to Assess Physical Function and the Incidence of Hypoglycemia in Participants With Glycogen Storage Disease Type III

Terminated
Conditions
Glycogen Storage Disease Type III
First Posted Date
2022-01-19
Last Posted Date
2023-09-28
Lead Sponsor
Ultragenyx Pharmaceutical Inc
Target Recruit Count
14
Registration Number
NCT05196165
Locations
🇺🇸

Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States

🇺🇸

Baylor College of Medicine, Houston, Texas, United States

🇺🇸

University of Texas, Houston, Texas, United States

and more 1 locations

A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)

Phase 3
Active, not recruiting
Conditions
Glycogen Storage Disease Type IA
Interventions
Genetic: DTX401
Other: Placebo
Drug: Oral corticosteroids
Drug: Placebo for oral corticosteroids
First Posted Date
2021-12-01
Last Posted Date
2025-02-21
Lead Sponsor
Ultragenyx Pharmaceutical Inc
Target Recruit Count
49
Registration Number
NCT05139316
Locations
🇺🇸

Children's Hospital of Orange County, Orange, California, United States

🇺🇸

Children's Hospital Colorado, Aurora, Colorado, United States

🇺🇸

University of Connecticut Health Center, Farmington, Connecticut, United States

and more 17 locations
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