Clinical Trials Related to Ultragenyx Pharmaceutical Inc

Related studies linked through entity extraction aliases.

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

Status: COMPLETED
Phase: PHASE2

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

Status: WITHDRAWN
Phase: PHASE2

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)

Status: COMPLETED
Phase: N/A

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

Status: COMPLETED
Phase: PHASE2

Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism

Status: NO_LONGER_AVAILABLE
Phase: N/A

Safety, Pharmacokinetics and Pharmacodynamics of BPS804 in Osteogenesis Imperfecta

Status: COMPLETED
Phase: PHASE2

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

Status: COMPLETED
Phase: PHASE2

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

Status: COMPLETED
Phase: PHASE2

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

Status: COMPLETED
Phase: PHASE1

Triheptanoin Treatment Trial for Patients With Adult Polyglucosan Body Disease

Status: COMPLETED
Phase: PHASE2