DeepMed Research

ULTRAGENYX PHARMACEUTICAL INC

🇪🇸Spain

Country: 🇪🇸Spain

Ownership: -

Employees: -

Market Cap: -

Website

Clinical Trials

Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program

Recruiting

Conditions: Glycogen Storage Disease Type Ia

Interventions: Other: No Intervention

First Posted Date: 2024-10-10

Last Posted Date: 2025-05-11

Lead Sponsor: Ultragenyx Pharmaceutical Inc

Target Recruit Count: 140

Registration Number: NCT06636383

Locations: 🇩🇪
Universitätsklinikum Hamburg Eppendorf, Hamburg, Germany
🇺🇸
Children's Hospital of Orange County, Orange, California, United States
🇺🇸
Children's Hospital Colorado, Denver, Colorado, United States

and more 16 locations

Setrusumab in Pediatric Japanese Subjects With Osteogenesis Imperfecta

Phase 3

Active, not recruiting

Conditions: Osteogenesis Imperfecta

Interventions: Biological: setrusumab

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

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

Phase 3

Recruiting

Conditions: Angelman Syndrome

Interventions: Drug: GTX-102
Procedure: Sham-LP

First Posted Date: 2024-09-27

Last Posted Date: 2025-05-14

Lead Sponsor: Ultragenyx Pharmaceutical Inc

Target Recruit Count: 120

Registration Number: NCT06617429

Locations: 🇪🇸
Clinical Trial Site, Seville, Spain
🇩🇪
University of Leipzig, Leipzig, Germany
🇯🇵
Nagoya City University Graduate School of Medical Sciences, Nagoya, Aichi, Japan

and more 20 locations

Long-term Extension of GTX-102 in Angelman Syndrome

Phase 3

Conditions: Angelman Syndrome

Interventions: Drug: GTX-102

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: 🇪🇸
Corporacio Sanitaria Parc Tauli - Hospital de Sabadell, Sabadell, Spain
🇺🇸
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

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: Drug: Triheptanoin
Dietary Supplement: MCT Oil

First Posted Date: 2023-07-06

Last Posted Date: 2025-02-25

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 13 locations

Setrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis Imperfecta

Phase 3

Active, not recruiting

Conditions: Osteogenesis Imperfecta

Interventions: Drug: Bisphosphonate
Biological: Setrusumab

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
🇨🇦
Childrens Hospital Of Eastern Ontario Research Institute, University Of Ottawa, Ottawa, Canada
🇮🇹
Azienda Ospedaliera Universitaria Policlinico Umberto I, Roma, Italy

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
Drug: Sodium Acetate

First Posted Date: 2022-04-25

Last Posted Date: 2025-03-11

Lead Sponsor: Ultragenyx Pharmaceutical Inc

Target Recruit Count: 50

Registration Number: NCT05345171

Locations: 🇺🇸
University of Arkansas for Medical Sciences, Little Rock, Arkansas, United States
🇺🇸
University of California, Los Angeles, California, United States
🇺🇸
University of Colorado, Aurora, Colorado, United States

and more 22 locations

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

Phase 2

Terminated

Conditions: Osteogenesis Imperfecta

Interventions: Biological: Setrusumab

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: 🇺🇸
Baylor College of Medicine, Houston, Texas, United States
🇺🇸
University of Texas, Houston, Texas, United States
🇺🇸
Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 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: 🇺🇸
Mount Sinai, Bronx, New York, United States
🇯🇵
Osaka City General Hospital, Osaka, Japan
🇯🇵
Kumamoto University Hospital, Kumamoto, Japan

and more 17 locations

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