These trials completed more than 12 months ago and should have reported results.

These trials are not yet due to report results: some are ongoing, some completed within the past 12 months.

These trials have problematic data on the registry. Details why »

Status Trial ID Title Completion date Category
Reported results 2013-001152-35 An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of UX003 rhGUS Enzyme Replacement Therapy in Patients with MPS 7 2016-07-13 due-trials
Listed as ongoing, but also has a completion date and reported results 2013-003771-35 A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Adaptive Study to Assess the Safety and Efficacy of UX007 in Subjects with Glucose Transporter Type 1 Deficiency Syndrome 2017-09-20 bad-data
Reported results 2013-004830-14 An Open-Label Phase 2 Study to Assess Safety and Clinical Effects of UX007 in Subjects with Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) 2016-08-25 due-trials
Listed as ongoing, but also has a completion date 2014-000406-35 A Randomized, open Label, Dose Finding, Phase 2 Study to Assess the Pharmacodynamics and Safety of the anti-FGF23 antibody, KRN23, in Pediatric Patients with X-linked Hypophosphatemia (XLH) 2018-10-30 bad-data
Reported results 2014-005432-33 A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Sialic Acid Extended-Release Tablets in Patients with GNE Myopathy (GNEM) or Hereditary Inclusion Bo... 2017-06-09 due-trials
Listed as ongoing, but also has a completion date 2014-005529-11 A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study to Assess the Efficacy and Safety of KRN23 in Adults with X-linked Hypophosphatemia (XLH) 2018-12-06 bad-data
Trial is outside EEC, and reported results 2014-005638-71 A Randomized, Placebo-Controlled, Blind-Start, Single-Crossover Phase 3 Study to Assess the Efficacy and Safety of UX003 rhGUS Enzyme Replacement Therapy in Patients with MPS 7 bad-data
Trial is partly outside EEC 2015-000104-26 An Open-label Study of UX003 rhGUS Enzyme Replacement Therapy in MPS 7 Patients Less than 5 years old bad-data
Listed as ongoing, but also has a completion date 2015-000389-69 An Open-label Extension Study to Assess the Long-term Safety and Efficacy of UX007 in Subjects with Glucose Transporter Type 1 Deficiency Syndrome 2019-04-15 bad-data
Completed, report not yet due 2015-001775-41 An Open-Label, Single-Arm, Phase 3 Study to Evaluate the Effects of KRN23 on Osteomalacia in Adults with X-linked Hypophosphatemia (XLH) 2018-08-22 not-yet-due
Trial is partly outside EEC 2015-001875-32 A Long-Term Open-Label Treatment and Extension Study of UX003 rhGUS Enzyme Replacement Therapy in Subjects with MPS 7 bad-data
Reported results Terminated 2015-004553-41 A Phase 2 Open-label study to Evaluate the Safety of Aceneuramic Acid Extended Release (Ace-ER) Tablets in GNE Myopathy (GNEM) (also known as Hereditary Inclusion Body Myopathy (HIBM)) patients with S... 2018-01-10 due-trials
Ongoing 2015-005536-17 A Phase 3, randomized, double-blind, placebo-controlled, crossover study to assess the efficacy and safety of UX007 in the treatment of movement disorders associated with Glucose Transporter Type 1 De... not-yet-due
Ongoing 2016-000322-19 An Open-label Long-Term Safety and Efficacy Extension Study in Subjects with Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Previously Enrolled in UX007 or Triheptanoin Studies not-yet-due
Listed as ongoing, but also has a completion date and reported results 2016-000360-42 A Phase 3b Open-label Extension Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients with GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopat... 2018-01-10 bad-data
Listed as ongoing, but also has a completion date 2016-000600-29 A Randomized, Open-Label, Phase 3 Study to Assess the Efficacy and Safety of KRN23 Versus Oral Phosphate and Active Vitamin D Treatment in Pediatric Patients with X-linked Hypophosphatemia (XLH) 2017-04-07 bad-data
Exempt 2016-003023-30 A Phase 1/2, Open-Label Safety and Dose-Finding Study of Adeno-Associated Virus (AAV)Serotype 8 (AAV8)-Mediated Gene Transfer of Glucose-6-Phosphatase (G6Pase) in Adults with Glycogen Storage Disease ... not-yet-due
Exempt 2018-000156-18 A Long-Term Follow-up Study to Evaluate the Safety and Efficacy of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Adults with Late-O... not-yet-due