DeepMed Research

ULTRAGENYX PHARMACEUTICAL INC

🇪🇸Spain

Country: 🇪🇸Spain

Ownership: -

Employees: -

Market Cap: -

Website

Clinical Trials

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

Completed

Conditions: Wilson Disease

First Posted Date: 2020-08-28

Last Posted Date: 2022-04-29

Lead Sponsor: Ultragenyx Pharmaceutical Inc

Target Recruit Count: 16

Registration Number: NCT04531189

Locations: 🇺🇸
Northwestern University Feinberg School of Medicine, Chicago, Illinois, United States
🇺🇸
Seattle Children's Hospital, Seattle, Washington, United States
🇺🇸
Yale University School of Medicine, New Haven, Connecticut, United States

and more 3 locations

Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA

Phase 3

Conditions: MPS IIIA
Mucopolysaccharidosis IIIA
Sanfilippo Syndrome
Sanfilippo A

Interventions: Other: No Investigational Product
Drug: Adjuvant Immunomodulatory (IM) Therapy

First Posted Date: 2020-04-24

Last Posted Date: 2025-04-13

Lead Sponsor: Ultragenyx Pharmaceutical Inc

Target Recruit Count: 41

Registration Number: NCT04360265

Locations: 🇪🇸
Vall d'Hebron Barcelona Campus, Barcelona, Spain
🇺🇸
Nationwide Children's Hospital, Columbus, Ohio, United States
🇦🇺
Women's and Children's Hospital, North Adelaide, South Australia, Australia

and more 1 locations

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

Completed

Conditions: Ornithine Transcarbamylase Deficiency

Interventions: Other: No Intervention

First Posted Date: 2020-02-13

Last Posted Date: 2020-03-30

Lead Sponsor: Ultragenyx Pharmaceutical Inc

Target Recruit Count: 120

Registration Number: NCT04269122

Locations: 🇺🇸
PPD Phase 1 Unit, Austin, Texas, United States

A Study of the Safety and Tolerability of GTX-102 in Children with Angelman Syndrome

Phase 1

Completed

Conditions: Angelman Syndrome

Interventions: Drug: GTX-102

First Posted Date: 2020-02-06

Last Posted Date: 2025-01-31

Lead Sponsor: Ultragenyx Pharmaceutical Inc

Target Recruit Count: 74

Registration Number: NCT04259281

Locations: 🇺🇸
Rare Disease Research, Atlanta, Georgia, United States
🇺🇸
Rush University Medical Center, Chicago, Illinois, United States
🇺🇸
Boston Children's Hospital, Boston, Massachusetts, United States

and more 22 locations

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

Phase 1

Terminated

Conditions: MPS IIIA
Sanfilippo A
Mucopolysaccharidosis III
Sanfilippo Syndrome

Interventions: Drug: ABO-102

First Posted Date: 2019-09-13

Last Posted Date: 2023-07-25

Lead Sponsor: Ultragenyx Pharmaceutical Inc

Target Recruit Count: 5

Registration Number: NCT04088734

Locations: 🇺🇸
Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, United States
🇦🇺
Adelaide Women's and Children's Hospital, North Adelaide, South Australia, Australia
🇪🇸
Hospital Clínico Universitario de Santiago, Santiago De Compostela, Spain

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

Completed

Conditions: Von Gierke's Disease (GSD Type Ia)
Glycogen Storage Disease Type IA

Interventions: Other: No intervention

First Posted Date: 2019-05-31

Last Posted Date: 2025-03-06

Lead Sponsor: Ultragenyx Pharmaceutical Inc

Target Recruit Count: 12

Registration Number: NCT03970278

Locations: 🇺🇸
University of Texas Health Science Center at Houston, Houston, Texas, United States
🇳🇱
University Medical Center Groningen, Groningen, Netherlands
🇪🇸
Complejo Hospitalario Universitario de Santiago, Santiago De Compostela, A Coruna, Spain

and more 3 locations

Expanded Access to Mepsevii

Conditions: MPS VII
Mucopolysaccharidosis VII
Sly Syndrome

First Posted Date: 2018-12-13

Last Posted Date: 2025-03-18

Lead Sponsor: Ultragenyx Pharmaceutical Inc

Registration Number: NCT03775174

Expanded Access to Triheptanoin

Conditions: Long Chain Fatty Acid Oxidation Disorders

First Posted Date: 2018-12-12

Last Posted Date: 2025-03-18

Lead Sponsor: Ultragenyx Pharmaceutical Inc

Registration Number: NCT03773770

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

Completed

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

Interventions: Other: No Intervention

First Posted Date: 2018-12-07

Last Posted Date: 2020-08-28

Lead Sponsor: Ultragenyx Pharmaceutical Inc

Target Recruit Count: 20

Registration Number: NCT03768817

Locations: 🇺🇸
Kaiser Permanente, Los Angeles, California, United States
🇺🇸
Boston Children's Hospital, Boston, Massachusetts, United States
🇺🇸
UPMC Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, United States

and more 12 locations

X-linked Hypophosphatemia Disease Monitoring Program

Active, not recruiting

Conditions: Hypophosphatemic Rickets
X-linked Hypophosphatemia

Interventions: Other: No intervention

First Posted Date: 2018-08-29

Last Posted Date: 2025-04-18

Lead Sponsor: Ultragenyx Pharmaceutical Inc

Target Recruit Count: 780

Registration Number: NCT03651505

Locations: 🇺🇸
University of Virgina School of Medicine, Charlottesville, Virginia, United States
🇺🇸
Children's Hospital of Alabama, Birmingham, Alabama, United States
🇺🇸
Children's Hospital Los Angeles, Los Angeles, California, United States

and more 36 locations

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