Ultragenyx Pharmaceutical
Open enrollment with an early-phase pipeline tilt
According to ClinicalTrials.gov-derived indexes, Ultragenyx Pharmaceutical sits in the top decile of US sponsors by registered trial count (#363 of 15,742 sponsors by registered trial count).
Ultragenyx Pharmaceutical: 52 sponsored US clinical trials, 4 recruiting.
Ultragenyx Pharmaceutical sponsors 52 registered US clinical trials on ClinicalTrials.gov, 4 of them currently recruiting, and 25 completed. 9 of these trials are in Phase 3-4 (later-stage) and 24 in Phase 1-2 (earlier-stage). The most-studied condition in this pipeline is Long-chain Fatty Acid Oxidation Disorders (LC-FAOD), with 3 trials.
Top-decile sponsor footprint
Ultragenyx Pharmaceutical ranks in the top decile of sponsors by registered trial count (#363 of 15,742 sponsors by registered trial count). Volume at this scale usually reflects a long institutional pipeline, read phase tilt and therapeutic areas below before treating it as current-year activity.
Trial completion reliability score
25 of 38 decided trials (Completed vs. Terminated on ClinicalTrials.gov) reached their planned completion (65.8%), vs. national p10 68.4% / p90 100% among 1,234 sponsors with at least 10 decided trials. Still-open trials (Recruiting, Active-not-recruiting, Not-yet-recruiting) are excluded since they have not reached an outcome yet.
Open and recent trials
52 total, page 1 of 2
A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)
NCT07157254
Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program
NCT06636383
Mucopolysaccharidosis VII Disease Monitoring Program
NCT03604835
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
NCT02716246
Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)
NCT06617429
Setrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis Imperfecta
NCT05768854
Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency
NCT05345171
Setrusumab vs Placebo for Osteogenesis Imperfecta
NCT05125809
Tumor-induced Osteomalacia Disease Monitoring Program
NCT04783428
Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program
NCT04632953
A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease
NCT04884815
Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency
NCT03636438
X-linked Hypophosphatemia Disease Monitoring Program
NCT03651505
First-in-human Study of UX016 in GNEM
NCT07511556
Long-Chain Fatty Acid Oxidation Disorders Online Disease Monitoring Program
NCT04812106
Clinical Survey Study to Assess Physical Function and the Incidence of Hypoglycemia in Participants With Glycogen Storage Disease Type III
NCT05196165
Long-term Extension Study of Setrusumab in Adults With Type I, III, or IV Osteogenesis Imperfecta
NCT05312697
A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)
NCT05139316
Safety, Tolerability, and Pharmacokinetics of UX053 in Patients With Glycogen Storage Disease Type III (GSD III)
NCT04990388
Adeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson Disease
NCT04909346
Clinical Evaluation and Assessment of Instruments and Biomarkers in Subjects With Wilson Disease
NCT04531189
Study to Evaluate Biomarkers and Clinical Manifestations in Individuals With Glycogen Storage Disease Type III (GSD III)
NCT04574830
Retrospective Study of Glucose Monitoring for Glycemic Control in Patients With GSDIa
NCT04708015
Study to Characterize Rate of Ureagenesis in Patients With Ornithine Transcarbamylase (OTC) Deficiency
NCT04717453
A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome
NCT04259281
Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease
NCT04088734
A Study to Assess Plasma Ammonia Time-Normalized Area Under the Curve and Rate of Ureagenesis in Healthy Adult Subjects
NCT04269122
Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia
NCT03970278
Clinical Outcome of Triheptanoin Treatment in Patients With Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Treated Under Expanded Access Program
NCT03768817
Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa)
NCT03517085
A Study in Adult Patients With Type I, III or IV Osteogenesis Imperfecta Treated With BPS804
NCT03118570
Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamylase (OTC) Deficiency
NCT02991144
Crossover Study to Assess the Efficacy and Safety of UX007 in the Treatment of Movement Disorders Associated With Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
NCT02960217
Long-Term Safety, Tolerability, and Efficacy of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B
NCT02971969
Observational Study of Males With Creatine Transporter Deficiency
NCT02931682
Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)
NCT02736188
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
NCT02731690
Safety and Dose Finding Study of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B
NCT02618915
A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)
NCT02432144
Study to Assess the Long Term Safety and Efficacy of UX007 in Participants With Glucose Type 1 Deficiency Syndrome (Glut1 DS)
NCT02599961
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
NCT02418455
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)
NCT02377921
Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Extension Study for Subjects Previously Enrolled in Triheptanoin Studies
NCT02214160
A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)
NCT02230566
Phase 2 Study of Triheptanoin (UX007) for the Treatment of Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
NCT01993186
A Study of UX007 (Triheptanoin) in Participants With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
NCT01886378
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
NCT01830972
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)
NCT01784679
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
NCT01517880
Safety and Pharmacokinetics of Sialic Acid Tables in Patients With Hereditary Inclusion Body Myopathy (HIBM)
NCT01359319
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 9 |
| Phase 2 | 15 |
| Phase 3 | 9 |
Therapeutic Areas
What is open in the Ultragenyx Pharmaceutical pipeline
4 recruiting of 52 indexed (8% open); 25 completed (48%).
Phase mix: 9 late (III/IV) vs 24 early (I/II) (early-phase heavy).
Top condition: Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) (3 trials), 9 others listed.
How open-pipeline share is computed
Recruiting share is recruiting_count divided by trial_count for this sponsor in the public registry export. Status can change between pulls; treat the figure as a research-attention snapshot, not product availability.
Ultragenyx Pharmaceutical: open-pipeline peers nationwide
Peers matched on 52 registered trials and 8% open enrollment, not the therapeutic-area sidebar.
Ultragenyx Pharmaceutical sits at or above the median trial volume and at or above the median recruiting share among these 9 nationwide peers -- the two do not always move together.
Sponsors near 52 trials (± recruiting share)
- Albany Medical College · 52 trials · 13% recruiting
- Burzynski Research Institute · 52 trials · 0% recruiting
- Dr. Reddy's Laboratories Limited · 52 trials · 0% recruiting
- PTC Therapeutics · 52 trials · 2% recruiting
Sponsors near 8% open enrollment (± trial volume)
- Alumis · 8% recruiting · 13 trials
- Andrei Iagaru · 8% recruiting · 13 trials
- BioXcel Therapeutics · 8% recruiting · 13 trials
- Corporal Michael J. Crescenz VA Medical Center · 8% recruiting · 13 trials
Related
What to do with this Ultragenyx Pharmaceutical page
52 registered trials is a research-attention snapshot, not an endorsement of Ultragenyx Pharmaceutical or any single study.
- 4 of Ultragenyx Pharmaceutical's trials are currently recruiting, check each one's own eligibility criteria before contacting a site. Browse recruiting trials
- Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) is the condition Ultragenyx Pharmaceutical studies most, compare against other sponsors active in the same condition before evaluating a specific study. See Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) trials
- Bring the specific trial ID (NCT number) to your own doctor before enrolling, this page is a directory, not medical guidance. Read the trial-finding guide
Trial and recruiting counts reflect ClinicalTrials.gov registry status as of the data vintage above; individual trial eligibility and enrollment status can change.
Ultragenyx Pharmaceutical pipeline card: 52 registered trials from official public datasets.Report a data error.