DeepMed Research

ULTRAGENYX PHARMACEUTICAL INC

🇪🇸Spain

Country: 🇪🇸Spain

Ownership: -

Employees: -

Market Cap: -

Website

Clinical Trials

A Study to Evaluate the Safety of Aceneuramic Acid Extended Release (Ace-ER; UX001) Tablets in Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy (GNEM) (Also Known as Hereditary Inclusion Body Myopathy [HIBM]) Patients With Severe Ambulatory Impairment

Phase 2

Terminated

Conditions: Hereditary Inclusion Body Myopathy
Distal Myopathy With Rimmed Vacuoles
Inclusion Body Myopathy 2
Distal Myopathy, Nonaka Type
GNE Myopathy
Quadriceps Sparing Myopathy

Interventions: Drug: Aceneuramic Acid Extended-Release

First Posted Date: 2016-04-07

Last Posted Date: 2019-02-19

Lead Sponsor: Ultragenyx Pharmaceutical Inc

Target Recruit Count: 42

Registration Number: NCT02731690

Locations: 🇺🇸
NYU Langone Medical Center, New York, New York, United States
🇨🇦
McMaster University, Hamilton, Ontario, Canada
🇺🇸
University of California, Irvine, Orange, California, United States

and more 2 locations

Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH

Phase 2

Recruiting

Conditions: Sanfilippo Syndrome
Sanfilippo A
MPS IIIA
Mucopolysaccharidosis III

Interventions: Biological: UX111
Drug: Prophylactic Immunomodulatory (IM) Therapy
Drug: Optimized Prophylactic IM Therapy
Drug: Adjuvant IM Therapy

First Posted Date: 2016-03-23

Last Posted Date: 2025-04-15

Lead Sponsor: Ultragenyx Pharmaceutical Inc

Target Recruit Count: 36

Registration Number: NCT02716246

Locations: 🇺🇸
Nationwide Children's Hospital, Columbus, Ohio, United States
🇺🇸
Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, United States
🇦🇺
Women's and Children's Hospital, North Adelaide, South Australia, Australia

and more 2 locations

Safety and Dose Finding Study of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B

Phase 1

Terminated

Conditions: Hemophilia B

Interventions: Genetic: DTX101

First Posted Date: 2015-12-02

Last Posted Date: 2018-11-14

Lead Sponsor: Ultragenyx Pharmaceutical Inc

Target Recruit Count: 6

Registration Number: NCT02618915

Locations: 🇺🇸
Orthopaedic Institute for Children, Los Angeles, California, United States
🇺🇸
Boston Children's Hospital, Boston, Massachusetts, United States
🇺🇸
Vanderbilt Hemostasis-Thrombosis Clinic, Nashville, Tennessee, United States

and more 6 locations

Study to Assess the Long Term Safety and Efficacy of UX007 in Participants With Glucose Type 1 Deficiency Syndrome (Glut1 DS)

Phase 2

Terminated

Conditions: Glucose Transporter Type 1 Deficiency Syndrome

Interventions: Drug: UX007

First Posted Date: 2015-11-09

Last Posted Date: 2020-06-11

Lead Sponsor: Ultragenyx Pharmaceutical Inc

Target Recruit Count: 15

Registration Number: NCT02599961

Locations: 🇺🇸
Miami Children's Hospital, Miami, Florida, United States
🇺🇸
Seattle Children's Hospital, Seattle, Washington, United States
🇩🇰
Copenhagen University Hospital, Copenhagen, Denmark

and more 6 locations

A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)

Phase 3

Completed

Conditions: Sly Syndrome
MPS VII
Mucopolysaccharidosis
Mucopolysaccharidosis VII

Interventions: Drug: UX003

First Posted Date: 2015-05-01

Last Posted Date: 2020-07-30

Lead Sponsor: Ultragenyx Pharmaceutical Inc

Target Recruit Count: 12

Registration Number: NCT02432144

Locations: 🇺🇸
Baylor College of Medicine, Houston, Texas, United States
🇧🇷
Hospital Infantil Candido Fontoura Sao Paulo, Sao Paulo, Brazil
🇺🇸
Children's Hospital of Orange County, Orange, California, United States

and more 4 locations

Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age

Phase 2

Completed

Conditions: Mucopolysaccharidosis VII
Mucopolysaccharidosis
Sly Syndrome
MPS VII

Interventions: Drug: UX003

First Posted Date: 2015-04-16

Last Posted Date: 2019-10-30

Lead Sponsor: Ultragenyx Pharmaceutical Inc

Target Recruit Count: 8

Registration Number: NCT02418455

Locations: 🇺🇸
New York University Langone Medical Center, New York, New York, United States
🇺🇸
Children's National Health System, Washington, District of Columbia, United States
🇵🇹
Centro Hospitalar do Porto, Porto, Portugal

and more 2 locations

Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)

Phase 3

Completed

Conditions: Hereditary Inclusion Body Myopathy
Distal Myopathy With Rimmed Vacuoles
Distal Myopathy, Nonaka Type
GNE Myopathy

Interventions: Drug: aceneuramic acid extended-release (Ace-ER)
Drug: Placebo

First Posted Date: 2015-03-04

Last Posted Date: 2019-06-27

Lead Sponsor: Ultragenyx Pharmaceutical Inc

Target Recruit Count: 89

Registration Number: NCT02377921

Locations: 🇧🇬
UMHAT "Alexandrovska", Sofia, Bulgaria
🇮🇹
Università Cattolica, Rome, Italy
🇬🇧
The Newcastle upon Tyne Hospitals, Newcastle Upon Tyne, Tyne And Wear, United Kingdom

and more 10 locations

A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)

Phase 3

Completed

Conditions: MPS 7
Sly Syndrome
Mucopolysaccharidosis
MPS VII

Interventions: Drug: UX003
Other: Placebo

First Posted Date: 2014-09-03

Last Posted Date: 2020-07-30

Lead Sponsor: Ultragenyx Pharmaceutical Inc

Target Recruit Count: 12

Registration Number: NCT02230566

Locations: 🇺🇸
Children's Hospital Oakland, Oakland, California, United States
🇺🇸
Miami Children's Hospital, Miami, Florida, United States
🇺🇸
Children's Hospital of Orange County, Orange, California, United States

and more 1 locations

Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Extension Study for Subjects Previously Enrolled in Triheptanoin Studies

Phase 2

Completed

Conditions: Carnitine Palmitoyltransferase (CPT I or CPT II) Deficiency
Very Long Chain Acyl-CoA Dehydrogenase (VLCAD) Deficiency
Trifunctional Protein (TFP) Deficiency
Long-chain 3-hydroxy-acyl-CoA Dehydrogenase (LCHAD) Deficiency
Carnitine-acylcarnitine Translocase (CACT) Deficiency

Interventions: Drug: UX007

First Posted Date: 2014-08-12

Last Posted Date: 2023-08-01

Lead Sponsor: Ultragenyx Pharmaceutical Inc

Target Recruit Count: 94

Registration Number: NCT02214160

Locations: 🇺🇸
Children's National Health System, Washington, District of Columbia, United States
🇺🇸
University of South Florida, Tampa, Florida, United States
🇺🇸
Vanderbilt Medical Center, Nashville, Tennessee, United States

and more 8 locations

Phase 2 Study of Triheptanoin (UX007) for the Treatment of Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

Phase 2

Completed

Conditions: Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

Interventions: Drug: Placebo
Drug: UX007

First Posted Date: 2013-11-25

Last Posted Date: 2020-06-19

Lead Sponsor: Ultragenyx Pharmaceutical Inc

Target Recruit Count: 36

Registration Number: NCT01993186

Locations: 🇦🇺
Melbourne Brain Centre, Heidelberg, Victoria, Australia
🇺🇸
Children's Hospital Colorado - University of Colorado, Denver, School of Medicine, Aurora, Colorado, United States
🇺🇸
Neurology & Epilepsy Research Center, Orlando, Florida, United States

and more 11 locations

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