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Ultragenyx Pharmaceutical Inc.
Sponsor

Ultragenyx Pharmaceutical Inc.

United States 60 Leveroni Court Suite 200, 94949-5746, United States

About the sponsor

Ultragenyx Pharmaceutical Inc. funds clinical research in glycogen storage disease type Ia, ornithine transcarbamylase deficiency, and long-chain fatty acid oxidation disorders, with a strong focus on restoring metabolic control and reducing disease burden in inherited conditions affecting energy and ammonia handling.

  • Glycogen storage disease type Ia
  • Ornithine transcarbamylase deficiency
  • Long-chain fatty acid oxidation disorders

Its research portfolio also includes Wilson disease, reflecting interest in disorders of copper regulation and broader inborn errors of metabolism.

About Specializations

Ultragenyx Pharmaceutical Inc. runs clinical trials in conditions such as Glycogen storage disease type I, Angelman's syndrome, Mucopolysaccharidosis III, Ornithine transcarbamoylase deficiency, among others.

Recruiting now

Ongoing trials

Clinical trials run by this sponsor – those enrolling participants first.

Condition
Country
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Type of trial
Recruitment status
Quick filters

14 clinical trials

Glycogen storage disease type I

Recruiting

Study on Long-Term Safety and Pregnancy Outcomes of Pariglasgene Brecaparvovec in Patients with Glycogen Storage Disease Type Ia (GSDIa)

InvestigationalNo placebo
Countries:DenmarkDenmark
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Pariglasgene Brecaparvovec
  • Sponsor:Ultragenyx Pharmaceutical Inc.

Mucopolysaccharidosis III

Recruiting

Long-term safety study of rebisufligene etisparvovec gene therapy in patients previously treated for Mucopolysaccharidosis type IIIA

InvestigationalNo placebo
Countries:SpainSpain
  • Participants:0–17 years
  • Substances:Rebisufligene Etisparvovec
  • Sponsor:Ultragenyx Pharmaceutical Inc.

Mucopolysaccharidosis III

Recruiting

Study on the Safety and Effectiveness of Rebisufligene Etisparvovec for Patients with Mucopolysaccharidosis Type IIIA

InvestigationalNo placebo
Countries:SpainSpain
  • Participants:0–17 years
  • Substances:Rebisufligene Etisparvovec
  • Sponsor:Ultragenyx Pharmaceutical Inc.

Hepato-lenticular degeneration

Recruiting

Study on the Safety and Effects of UX701 Gene Therapy for Adults with Wilson Disease

Registered drugNo placebo
Countries:DenmarkDenmark
  • Participants:18–64 years · 65+ years
  • Substances:Eculizumab
  • Sponsor:Ultragenyx Pharmaceutical Inc.

Angelman's syndrome

Not recruiting

A Study Testing the Safety and How Well GTX-102 Works in Adults and Children with Angelman Syndrome

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years
  • Substances:Apazunersen
  • Sponsor:Ultragenyx Pharmaceutical Inc.

Angelman's syndrome

Not recruiting

Study on the Effects of GTX-102 for Children with Angelman Syndrome

InvestigationalNo placebo
Countries:GermanyGermany
  • Participants:0–17 years
  • Substances:Apazunersen
  • Sponsor:Ultragenyx Pharmaceutical Inc.

Angelman's syndrome

Not recruiting

Study on the Long-Term Safety of GTX-102 for Patients with Angelman Syndrome

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:0–17 years
  • Substances:Apazunersen
  • Sponsor:Ultragenyx Pharmaceutical Inc.

Ornithine transcarbamoylase deficiency

Not recruiting

Study of Avalotcagene Ontaparvovec for Patients Aged 12 and Older with Late-onset Ornithine Transcarbamylase Deficiency

Registered drug
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Prednisolone
  • Sponsor:Ultragenyx Pharmaceutical Inc.

Glycogen storage disease type I

Not recruiting

Study on the Effects of Pariglasgene Brecaparvovec for Adults with Glycogen Storage Disease Type Ia

InvestigationalNo placebo
Countries:The NetherlandsThe Netherlands
  • Participants:18–64 years · 65+ years
  • Substances:Pariglasgene Brecaparvovec
  • Sponsor:Ultragenyx Pharmaceutical Inc.

Glycogen storage disease type I

Not recruiting

Study of Pariglasgene Brecaparvovec and Prednisolone for Adults with Glycogen Storage Disease Type Ia

Registered drug
Countries:DenmarkDenmark
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Prednisolone
  • Sponsor:Ultragenyx Pharmaceutical Inc.

Fatty acid oxidation disorder

Not recruiting

Study on the Effects of Triheptanoin and Medium-chain Triglycerides in Children with Long-chain Fatty Acid Oxidation Disorders

Registered drugNo placebo
Countries:CzechiaCzechia
  • Participants:0–17 years
  • Substances:Doconexent
  • Sponsor:Ultragenyx Pharmaceutical Inc.

Osteogenesis imperfecta

Not recruiting

Study on Setrusumab for Patients with Osteogenesis Imperfecta (Brittle Bone Disease)

Investigational
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years
  • Substances:Setrusumab
  • Sponsor:Ultragenyx Pharmaceutical Inc.

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