DeepMed Research

ULTRAGENYX PHARMACEUTICAL INC

🇪🇸Spain

Country: 🇪🇸Spain

Ownership: -

Employees: -

Market Cap: -

Website

Clinical Trials

A Study of UX007 (Triheptanoin) in Participants With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)

Phase 2

Completed

Conditions: Carnitine Palmitoyltransferase (CPT II) Deficiency
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
Very Long Chain Acyl-CoA Dehydrogenase (VLCAD) Deficiency
Longchain 3-hydroxy-acyl-CoA Dehydrogenase (LCHAD) Deficiency
Trifunctional Protein (TFP) Deficiency

Interventions: Drug: UX007

First Posted Date: 2013-06-25

Last Posted Date: 2021-02-11

Lead Sponsor: Ultragenyx Pharmaceutical Inc

Target Recruit Count: 29

Registration Number: NCT01886378

Locations: 🇺🇸
Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States
🇺🇸
University of Southern Florida, Tampa, Florida, United States
🇺🇸
Boston Children's Hospital, Boston, Massachusetts, United States

and more 7 locations

An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of Study Drug UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)

Phase 1

Completed

Conditions: Mucopolysaccharidosis Type 7

Interventions: Drug: UX003

First Posted Date: 2013-05-17

Last Posted Date: 2019-01-04

Lead Sponsor: Ultragenyx Pharmaceutical Inc

Target Recruit Count: 3

Registration Number: NCT01856218

Locations: 🇬🇧
Manchester Academic Health Science Centre, Manchester, United Kingdom

An Open Label Phase 2 Extension Study of Higher Dose Sialic Acid-Extended Release (SA-ER) Tablets and Sialic Acid-Immediate Release (SA-IR) Capsules in Patients With Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy

Phase 2

Completed

Conditions: Hereditary Inclusion Body Myopathy (HIBM)
GNE Myopathy

Interventions: Drug: SA-ER 500 mg
Drug: SA-IR 500 mg

First Posted Date: 2013-04-12

Last Posted Date: 2018-04-11

Lead Sponsor: Ultragenyx Pharmaceutical Inc

Target Recruit Count: 59

Registration Number: NCT01830972

Locations: 🇺🇸
UCLA Medical Center, Los Angeles, California, United States
🇺🇸
NYU Medical Center, New York, New York, United States
🇮🇱
Hadassah University Hospital, Jerusalem, Israel

and more 1 locations

Study of Safety and Tolerability of BPS804 in Patients With Late-stage Chronic Kidney Disease

Phase 2

Withdrawn

Conditions: Chronic-kidney Disease Stage 5D on Stable Hemodialysis

Interventions: Drug: BPS804
Drug: Placebo

First Posted Date: 2013-03-07

Last Posted Date: 2022-09-14

Lead Sponsor: Ultragenyx Pharmaceutical Inc

Registration Number: NCT01806610

GNE-Myopathy Disease Monitoring Program (GNEM-DMP): A Registry and Prospective Observational Natural History Study to Assess GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)

Completed

Conditions: Hereditary Inclusion Body Myopathy
GNE Myopathy
Quadriceps Sparing Myopathy (QSM)
Nonaka Disease
Distal Myopathy With Rimmed Vacuoles (DMRV)

First Posted Date: 2013-02-06

Last Posted Date: 2018-04-27

Lead Sponsor: Ultragenyx Pharmaceutical Inc

Target Recruit Count: 319

Registration Number: NCT01784679

Locations: 🇧🇬
Bulgarian Neuromuscular Disease Association, Sofia, Bulgaria
🇫🇷
Association Institut de Myologie, Paris, France
🇨🇦
McMaster University, Hamilton, Ontario, Canada

and more 2 locations

A Phase 2 Study to Evaluate the Dose and Pharmacodynamic Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy

Phase 2

Completed

Conditions: GNE Myopathy
Hereditary Inclusion Body Myopathy

Interventions: Drug: Sialic Acid Extended Release (SA-ER)
Drug: Placebo

First Posted Date: 2012-01-25

Last Posted Date: 2016-06-16

Lead Sponsor: Ultragenyx Pharmaceutical Inc

Target Recruit Count: 46

Registration Number: NCT01517880

Locations: 🇺🇸
UCLA Medical Center, Los Angeles, California, United States
🇺🇸
Washington University School of Medicine, St. Louis, Missouri, United States
🇺🇸
New York University School of Medicine, New York, New York, United States

and more 1 locations

Safety, Pharmacokinetics and Pharmacodynamics of BPS804 in Osteogenesis Imperfecta

Phase 2

Completed

Conditions: Osteogenesis Imperfecta

Interventions: Drug: BPS804

First Posted Date: 2011-08-16

Last Posted Date: 2021-05-11

Lead Sponsor: Ultragenyx Pharmaceutical Inc

Target Recruit Count: 10

Registration Number: NCT01417091

Locations: 🇩🇪
Novartis Investigative Site, Wuerzburg, Germany

Dose Escalation Study to Evaluate the Safety and Tolerability of Multiple Infusions of BPS804 in Adults With Hypophosphatasia (HPP)

Phase 2

Completed

Conditions: Hypophosphatasia

Interventions: Drug: BPS804

First Posted Date: 2011-08-01

Last Posted Date: 2022-09-16

Lead Sponsor: Ultragenyx Pharmaceutical Inc

Target Recruit Count: 8

Registration Number: NCT01406977

Locations: 🇩🇪
Mereo BioPharma 3 Ltd Investigative Site, Wuerzburg, Germany

Safety and Efficacy of Multiple Dosing Regimens of BPS804 in Post Menopausal Women With Low Bone Mineral Density

Phase 2

Completed

Conditions: Osteoporosis
Osteopenia

Interventions: Drug: BPS804 20mg/Kg
Drug: Placebo to 20mg/Kg BPS804

First Posted Date: 2011-08-01

Last Posted Date: 2022-09-15

Lead Sponsor: Ultragenyx Pharmaceutical Inc

Target Recruit Count: 44

Registration Number: NCT01406548

Locations: 🇺🇸
Novartis Investigative Site, Duncansville, Pennsylvania, United States

Safety and Pharmacokinetics of Sialic Acid Tables in Patients With Hereditary Inclusion Body Myopathy (HIBM)

Phase 1

Completed

Conditions: Hereditary Inclusion Body Myopathy (HIBM)

Interventions: Drug: Sialic Acid Extended Release (SA-ER) Tablets
Drug: Sialic Acid Extended Release (SA-ER) Tables

First Posted Date: 2011-05-24

Last Posted Date: 2012-05-21

Lead Sponsor: Ultragenyx Pharmaceutical Inc

Target Recruit Count: 26

Registration Number: NCT01359319

Locations: 🇺🇸
Clinilabs, New York City, New York, United States
🇺🇸
West Coast Clinical Trials, Costa Mesa, California, United States

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