Ultragenyx Pharmaceutical

52 total trials 4 currently recruiting 25 completed

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.

Trial Pipeline

52 total, page 1 of 2

RECRUITING Phase 2

A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)

NCT07157254

RECRUITING

Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program

NCT06636383

RECRUITING

Mucopolysaccharidosis VII Disease Monitoring Program

NCT03604835

RECRUITING Phase 2

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

NCT02716246

ACTIVE NOT RECRUITING Phase 3

Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)

NCT06617429

ACTIVE NOT RECRUITING Phase 3

Setrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis Imperfecta

NCT05768854

ACTIVE NOT RECRUITING Phase 3

Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency

NCT05345171

ACTIVE NOT RECRUITING Phase 2

Setrusumab vs Placebo for Osteogenesis Imperfecta

NCT05125809

ACTIVE NOT RECRUITING

Tumor-induced Osteomalacia Disease Monitoring Program

NCT04783428

ACTIVE NOT RECRUITING

Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program

NCT04632953

ACTIVE NOT RECRUITING Phase 1

A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease

NCT04884815

ACTIVE NOT RECRUITING

Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency

NCT03636438

ACTIVE NOT RECRUITING

X-linked Hypophosphatemia Disease Monitoring Program

NCT03651505

NOT YET RECRUITING Phase 1

First-in-human Study of UX016 in GNEM

NCT07511556

TERMINATED

Long-Chain Fatty Acid Oxidation Disorders Online Disease Monitoring Program

NCT04812106

TERMINATED

Clinical Survey Study to Assess Physical Function and the Incidence of Hypoglycemia in Participants With Glycogen Storage Disease Type III

NCT05196165

TERMINATED Phase 2

Long-term Extension Study of Setrusumab in Adults With Type I, III, or IV Osteogenesis Imperfecta

NCT05312697

COMPLETED Phase 3

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

TERMINATED Phase 1

Safety, Tolerability, and Pharmacokinetics of UX053 in Patients With Glycogen Storage Disease Type III (GSD III)

NCT04990388

TERMINATED

Adeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson Disease

NCT04909346

COMPLETED

Clinical Evaluation and Assessment of Instruments and Biomarkers in Subjects With Wilson Disease

NCT04531189

COMPLETED

Study to Evaluate Biomarkers and Clinical Manifestations in Individuals With Glycogen Storage Disease Type III (GSD III)

NCT04574830

COMPLETED

Retrospective Study of Glucose Monitoring for Glycemic Control in Patients With GSDIa

NCT04708015

TERMINATED

Study to Characterize Rate of Ureagenesis in Patients With Ornithine Transcarbamylase (OTC) Deficiency

NCT04717453

COMPLETED Phase 1

A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome

NCT04259281

TERMINATED Phase 1

Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease

NCT04088734

COMPLETED

A Study to Assess Plasma Ammonia Time-Normalized Area Under the Curve and Rate of Ureagenesis in Healthy Adult Subjects

NCT04269122

COMPLETED

Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia

NCT03970278

COMPLETED

Clinical Outcome of Triheptanoin Treatment in Patients With Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Treated Under Expanded Access Program

NCT03768817

COMPLETED Phase 1

Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa)

NCT03517085

COMPLETED Phase 2

A Study in Adult Patients With Type I, III or IV Osteogenesis Imperfecta Treated With BPS804

NCT03118570

COMPLETED Phase 1

Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamylase (OTC) Deficiency

NCT02991144

TERMINATED Phase 3

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

COMPLETED

Long-Term Safety, Tolerability, and Efficacy of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B

NCT02971969

TERMINATED

Observational Study of Males With Creatine Transporter Deficiency

NCT02931682

TERMINATED Phase 3

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

TERMINATED Phase 2

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

TERMINATED Phase 1

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

NCT02618915

COMPLETED Phase 3

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

NCT02432144

TERMINATED Phase 2

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

NCT02599961

COMPLETED Phase 2

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

COMPLETED Phase 3

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

COMPLETED Phase 2

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

NCT02214160

COMPLETED Phase 3

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

NCT02230566

COMPLETED Phase 2

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

NCT01993186

COMPLETED Phase 2

A Study of UX007 (Triheptanoin) in Participants With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)

NCT01886378

COMPLETED Phase 2

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

COMPLETED

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

COMPLETED Phase 2

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

COMPLETED Phase 1

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

NCT01359319

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What the Pipeline for Ultragenyx Pharmaceutical Shows

According to the ClinicalTrials.gov registry, Ultragenyx Pharmaceutical is linked to 52 US clinical trials across every stage of research activity. Of those, 4 studies are currently recruiting, about 8% of the sponsor's indexed portfolio, and 25 are already marked complete, representing roughly 48% of the total.

The phase mix for Ultragenyx Pharmaceutical reports 9 late-stage studies (Phase 3 and Phase 4 combined) and 24 earlier-phase studies (Phase 1 and Phase 2).

The top therapeutic focus area indexed for Ultragenyx Pharmaceutical is Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) with 3 linked trials, and 9 other condition areas appear in the top list above.

How to read these pipeline numbers

Recruiting share is one of the more practical signals here: it reflects how much of a sponsor's research is presently open to new participants, while the completed share indicates the depth of finished work that has already contributed registry results. Both counts come directly from the public ClinicalTrials.gov dataset and are refreshed on the registry side; this page mirrors the latest data pull without altering it.

A portfolio weighted toward Phase 3 usually reflects an organization advancing candidates toward regulatory review, where the research centers on comparative efficacy and broader safety across larger populations. A heavier Phase 1 and Phase 2 tilt generally indicates exploratory work, safety, dosing, and early signal detection, and is common among research-forward sponsors that seed many early programs. Phase 4 entries, when present, track interventions already in real-world use and typically focus on long-term safety, effectiveness across subgroups, or formulation comparisons.

That distribution is a quick read of where the organization concentrates its research attention; it does not imply product availability, market share, or any clinical endorsement. All numbers on this page come from ClinicalTrials.gov maintained by the National Library of Medicine, and counts can shift as new studies are registered or existing ones update their status. This information is provided for reference and educational purposes only, not as medical, investment, or regulatory advice, verify current details directly with ClinicalTrials.gov before relying on any figure here.

Data sourced from official public datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial

Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page aggregates every registered trial from this sponsor, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.