DeepMed Research

ULTRAGENYX PHARMACEUTICAL INC

🇪🇸Spain

Country: 🇪🇸Spain

Ownership: -

Employees: -

Market Cap: -

Website

Clinical Trials

Setrusumab vs Placebo for Osteogenesis Imperfecta

Phase 2

Active, not recruiting

Conditions: Osteogenesis Imperfecta

Interventions: Biological: Setrusumab
Other: Placebo

First Posted Date: 2021-11-18

Last Posted Date: 2025-04-25

Lead Sponsor: Ultragenyx Pharmaceutical Inc

Target Recruit Count: 182

Registration Number: NCT05125809

Locations: 🇺🇸
Nationwide Children's Hospital- Ohio State University College of Medicine, Columbus, Ohio, United States
🇺🇸
Arkansas Children's Hospital, Little Rock, Arkansas, United States
🇺🇸
Nemours/ Alfred i. duPoint Hospital for Children, Wilmington, Delaware, United States

and more 88 locations

Safety, Tolerability, and Pharmacokinetics of UX053 in Patients With Glycogen Storage Disease Type III (GSD III)

Phase 1

Terminated

Conditions: Glycogen Storage Disease Type III

Interventions: Biological: UX053
Drug: Antipyretic
Other: Placebo
Drug: H2 Blocker
Drug: H1 Blocker

First Posted Date: 2021-08-04

Last Posted Date: 2024-04-16

Lead Sponsor: Ultragenyx Pharmaceutical Inc

Target Recruit Count: 9

Registration Number: NCT04990388

Locations: 🇺🇸
University of Texas, Health Science Center of Houston, Houston, Texas, United States
🇺🇸
University of California, Irvine, Orange, California, United States
🇺🇸
Rare Disease Research, Atlanta, Georgia, United States

and more 3 locations

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

Terminated

Conditions: Ornithine Transcarbamylase Deficiency
Glycogen Storage Disease Type IA
Wilson Disease

First Posted Date: 2021-06-01

Last Posted Date: 2023-06-12

Lead Sponsor: Ultragenyx Pharmaceutical Inc

Target Recruit Count: 51

Registration Number: NCT04909346

Locations: 🇪🇸
Hospital Clínico Universitario de Santiago de Compostela, Santiago De Compostela, Spain
🇧🇷
Hospital de Clinicas de Porto Alegre, Porto Alegre, Rio Grande Do Sul, Brazil
🇨🇦
McGill University Health Center, Montréal, Quebec, Canada

and more 2 locations

A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease

Phase 1

Recruiting

Conditions: Wilson Disease

Interventions: Genetic: UX701
Other: Placebo
Drug: Standard of Care (SOC)

First Posted Date: 2021-05-13

Last Posted Date: 2025-05-20

Lead Sponsor: Ultragenyx Pharmaceutical Inc

Target Recruit Count: 82

Registration Number: NCT04884815

Locations: 🇺🇸
University of California Los Angeles, Los Angeles, California, United States
🇺🇸
University of California Davis, Sacramento, California, United States
🇺🇸
Massachusetts General Hospital, Boston, Massachusetts, United States

and more 14 locations

Long-Chain Fatty Acid Oxidation Disorders Online Disease Monitoring Program

Terminated

Conditions: Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)

Interventions: Other: No Intervention

First Posted Date: 2021-03-23

Last Posted Date: 2022-11-18

Lead Sponsor: Ultragenyx Pharmaceutical Inc

Target Recruit Count: 8

Registration Number: NCT04812106

Locations: 🇺🇸
Children's Hospital of Pittsburgh of UPMC, Pittsburgh, Pennsylvania, United States

Tumor-induced Osteomalacia Disease Monitoring Program

Active, not recruiting

Conditions: Tumor-induced Osteomalacia (TIO)

Interventions: Other: No intervention

First Posted Date: 2021-03-05

Last Posted Date: 2025-02-03

Lead Sponsor: Ultragenyx Pharmaceutical Inc

Target Recruit Count: 21

Registration Number: NCT04783428

Locations: 🇺🇸
Vanderbilt University Medical Center, Nashville, Tennessee, United States
🇺🇸
Johns Hopkins University, Baltimore, Maryland, United States
🇺🇸
Indiana University, Bloomington, Indiana, United States

and more 2 locations

Study to Characterize Rate of Ureagenesis in Patients With Ornithine Transcarbamylase (OTC) Deficiency

Terminated

Conditions: Ornithine Transcarbamylase Deficiency

Interventions: Other: No Intervention

First Posted Date: 2021-01-22

Last Posted Date: 2022-02-18

Lead Sponsor: Ultragenyx Pharmaceutical Inc

Target Recruit Count: 1

Registration Number: NCT04717453

Locations: 🇺🇸
PPD Phase 1 Clinic - Orlando, Orlando, Florida, United States

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

Completed

Conditions: Glycogen Storage Disease Type IA

First Posted Date: 2021-01-13

Last Posted Date: 2024-05-29

Lead Sponsor: Ultragenyx Pharmaceutical Inc

Target Recruit Count: 15

Registration Number: NCT04708015

Locations: 🇺🇸
UT Health - McGovern Medical School, Houston, Texas, United States
🇳🇱
University Medical Center Groningen, Groningen, Netherlands

Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program

Recruiting

Conditions: Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)

Interventions: Other: No Intervention

First Posted Date: 2020-11-17

Last Posted Date: 2025-04-30

Lead Sponsor: Ultragenyx Pharmaceutical Inc

Target Recruit Count: 150

Registration Number: NCT04632953

Locations: 🇺🇸
Phoenix Children's Hospital, Phoenix, Arizona, United States
🇺🇸
University of California San Francisco, San Francisco, California, United States
🇺🇸
Children's Hospital of Colorado, Aurora, Colorado, United States

and more 13 locations

Study to Evaluate Biomarkers and Clinical Manifestations in Individuals With Glycogen Storage Disease Type III (GSD III)

Completed

Conditions: Glycogen Storage Disease Type III

First Posted Date: 2020-10-05

Last Posted Date: 2022-09-13

Lead Sponsor: Ultragenyx Pharmaceutical Inc

Target Recruit Count: 18

Registration Number: NCT04574830

Locations: 🇺🇸
Children's Hospital Orange County, Orange, California, United States
🇺🇸
Colorado Children's Hospital, Aurora, Colorado, United States
🇺🇸
University of Texas Medical School, Houston, Texas, United States

and more 1 locations

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