Ultragenyx Pharmaceutical Clinical Trials: 62 Led Studies

Archive historyOct 2022 to Apr 2026

70 recorded changes across Ultragenyx Pharmaceutical trials

Every status move, completion date shift and enrollment change ComplyRx has recorded across 16 of these trials. One is shown below.

Status changeStatus moved to completed from active not recruitingNCT05139316 · recorded 1 Apr 2026

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+66 more recorded changes · Track a drug to see every change the night it lands

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Most active programmes

The drugs appearing most often across Ultragenyx Pharmaceutical's recently active trials. Comparator arms are excluded.

UX0075 trials
GTX-1024 trials
BPS8044 trials
UX0034 trials
Sialic Acid Extended Release (SA-ER) Tablets4 trials
Setrusumab3 trials

Upcoming readouts

Next primary completion dates among Ultragenyx Pharmaceutical's active phase 2 and phase 3 trials. Dates are registry estimates and move often, which is exactly what the tracker watches.

Mar 2029Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSHPhase 2
Jan 2030Aurora: A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman S…Phase 2

Top therapeutic areas

Conditions studied most often across Ultragenyx Pharmaceutical's recently active trials.

GNE Myopathy7 trials
Osteogenesis Imperfecta6 trials
MPS VII5 trials
Sly Syndrome5 trials
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)5 trials
Hereditary Inclusion Body Myopathy5 trials

Ultragenyx Pharmaceutical trials

The 50 most significant of Ultragenyx Pharmaceutical's recently active trials, soonest estimated readout first.
Select any row for the full record; the change timeline unlocks with tracking.

Trial	Phase	Stability	Status	Enrollment	Est. readout
Setrusumab in Pediatric Japanese Subjects With Osteogenesis ImperfectaNCT06636071setrusumab	Phase 3	Locked · track to reveal	OngoingActive, not recruiting	6 est.	Jan 2027
Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIANCT04360265No Investigational Product	Phase 3	Locked · track to reveal	OngoingEnrolling by invitation	41 est.	Aug 2027
A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAODNCT05933200Triheptanoin	Phase 3	Locked · track to reveal	OngoingActive, not recruiting	69 est.	Aug 2027
Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) DeficiencyNCT05345171DTX301	Phase 3	Locked · track to reveal	OngoingActive, not recruiting	37 est.	Sept 2027
First-in-human Study of UX016 in GNEMNCT07511556UX016	Phase 1	Locked · track to reveal	OngoingNot yet recruiting	24 est.	Dec 2028
Long-term Extension of GTX-102 in Angelman SyndromeNCT06415344GTX-102	Phase 3	Locked · track to reveal	OngoingEnrolling by invitation	255 est.	Feb 2029
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSHNCT02716246UX111	Phase 2	Locked · track to reveal	OngoingRecruiting	36 est.	Mar 2029
A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson DiseaseNCT04884815UX701	Phase 1	Locked · track to reveal	OngoingActive, not recruiting	82 est.	Mar 2029
CAPtivateLong Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC DeficiencyNCT03636438	—	Not yet scored	OngoingActive, not recruiting	11 est.	Dec 2029
AuroraA Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)NCT07157254GTX-102	Phase 2	Locked · track to reveal	OngoingRecruiting	60 est.	Jan 2030
Tumor-induced Osteomalacia Disease Monitoring ProgramNCT04783428	—	Not yet scored	OngoingActive, not recruiting	23 est.	28 Feb 2032
Mucopolysaccharidosis VII Disease Monitoring ProgramNCT03604835	—	Not yet scored	OngoingRecruiting	50 est.	Apr 2032
X-linked Hypophosphatemia Disease Monitoring ProgramNCT03651505	—	Not yet scored	OngoingActive, not recruiting	782 est.	Dec 2032
Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring ProgramNCT04632953	—	Not yet scored	OngoingActive, not recruiting	150 est.	Dec 2035
Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring ProgramNCT06636383	—	Not yet scored	OngoingRecruiting	140 est.	Dec 2036
AspirePhase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)NCT06617429GTX-102	Phase 3	Locked · track to reveal	OngoingActive, not recruiting	129 est.	Jul 2026
CosmicSetrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis ImperfectaNCT05768854Bisphosphonate	Phase 3	Locked · track to reveal	OngoingActive, not recruiting	69 est.	23 Oct 2025
OrbitSetrusumab vs Placebo for Osteogenesis ImperfectaNCT05125809Setrusumab	Phase 2	Locked · track to reveal	OngoingActive, not recruiting	183 est.	20 Oct 2025
Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type IaNCT03970278	—	Not yet scored	Completed	12 est.	25 Feb 2025
A Study of the Safety and Tolerability of GTX-102 in Children With Angelman SyndromeNCT04259281GTX-102	Phase 1	Locked · track to reveal	Completed	74 est.	8 Jan 2025
A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)NCT05139316DTX401	Phase 3	Locked · track to reveal	Completed	49 est.	20 Feb 2024
Safety, Tolerability, and Pharmacokinetics of UX053 in Patients With Glycogen Storage Disease Type III (GSD III)NCT04990388UX053	Phase 1	Locked · track to reveal	StoppedTerminated	9 est.	20 Mar 2023
Clinical Survey Study to Assess Physical Function and the Incidence of Hypoglycemia in Participants With Glycogen Storage Disease Type IIINCT05196165	—	Not yet scored	StoppedTerminated	14 est.	2 Mar 2023
Adeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson DiseaseNCT04909346	—	Not yet scored	StoppedTerminated	51 est.	17 Nov 2022
Long-Chain Fatty Acid Oxidation Disorders Online Disease Monitoring ProgramNCT04812106	—	Not yet scored	StoppedTerminated	8 est.	27 Oct 2022
VigilanObservational Study of Males With Creatine Transporter DeficiencyNCT02931682	—	Not yet scored	StoppedTerminated	50 est.	24 Oct 2022
Long-term Extension Study of Setrusumab in Adults With Type I, III, or IV Osteogenesis ImperfectaNCT05312697Setrusumab	Phase 2	Locked · track to reveal	StoppedTerminated	2 est.	7 Jul 2022
Study to Evaluate Biomarkers and Clinical Manifestations in Individuals With Glycogen Storage Disease Type III (GSD III)NCT04574830	—	Not yet scored	Completed	18 est.	30 Jun 2022
Clinical Evaluation and Assessment of Instruments and Biomarkers in Subjects With Wilson DiseaseNCT04531189	—	Not yet scored	Completed	16 est.	25 Mar 2022
Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA DiseaseNCT04088734ABO-102	Phase 1	Locked · track to reveal	StoppedTerminated	5 est.	10 Mar 2022
Retrospective Study of Glucose Monitoring for Glycemic Control in Patients With GSDIaNCT04708015	—	Not yet scored	Completed	15 est.	4 Mar 2022
CAPtivateSafety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamylase (OTC) DeficiencyNCT02991144scAAV8OTC	Phase 1	Locked · track to reveal	Completed	16 est.	16 Dec 2021
Study to Characterize Rate of Ureagenesis in Patients With Ornithine Transcarbamylase (OTC) DeficiencyNCT04717453	—	Not yet scored	StoppedTerminated	1 est.	15 Dec 2021
Long-Term Safety, Tolerability, and Efficacy of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia BNCT02971969	—	Not yet scored	Completed	6 est.	6 Nov 2021
Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa)NCT03517085DTX401	Phase 1	Locked · track to reveal	Completed	12 est.	2 Nov 2021
Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Extension Study for Subjects Previously Enrolled in Triheptanoin StudiesNCT02214160UX007	Phase 2	Locked · track to reveal	Completed	94 est.	3 Dec 2020
Clinical Outcome of Triheptanoin Treatment in Patients With Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) Treated Under Expanded Access ProgramNCT03768817	—	Not yet scored	Completed	20 est.	8 Jun 2020
A Study to Assess Plasma Ammonia Time-Normalized Area Under the Curve and Rate of Ureagenesis in Healthy Adult SubjectsNCT04269122	—	Not yet scored	Completed	120 est.	20 Feb 2020
Study to Assess the Long Term Safety and Efficacy of UX007 in Participants With Glucose Type 1 Deficiency Syndrome (Glut1 DS)NCT02599961UX007	Phase 2	Locked · track to reveal	StoppedTerminated	15 est.	22 Oct 2019
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)NCT02960217UX007	Phase 3	Locked · track to reveal	StoppedTerminated	44 est.	9 Oct 2019
AsteroidA Study in Adult Patients With Type I, III or IV Osteogenesis Imperfecta Treated With BPS804NCT03118570setrusumab	Phase 2	Locked · track to reveal	Completed	112 est.	1 Oct 2019
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 AgeNCT02418455UX003	Phase 2	Locked · track to reveal	Completed	8 est.	26 Mar 2019
A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)NCT02432144UX003	Phase 3	Locked · track to reveal	Completed	12 est.	14 Jan 2019
MeteoroidAn Exploratory Study of BPS804 Treatment in Adult Patients With Type I, III or IV Osteogenesis ImperfectaNCT03216486BPS804	Phase 2	Locked · track to reveal	StoppedWithdrawn	0 est.	1 Nov 2018
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)NCT02736188Aceneuramic Acid Extended-Release Tablets	Phase 3	Locked · track to reveal	StoppedTerminated	143 est.	10 Jan 2018
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 ImpairmentNCT02731690Aceneuramic Acid Extended-Release	Phase 2	Locked · track to reveal	StoppedTerminated	42 est.	10 Jan 2018
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	—	Not yet scored	Completed	319 est.	30 Nov 2017
Safety and Dose Finding Study of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia BNCT02618915DTX101	Phase 1	Locked · track to reveal	StoppedTerminated	6 est.	18 Oct 2017
Phase 2 Study of Triheptanoin (UX007) for the Treatment of Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)NCT01993186UX007	Phase 2	Locked · track to reveal	Completed	36 est.	20 Sept 2017
GNEMPhase 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)NCT02377921aceneuramic acid extended-release (Ace-ER)	Phase 3	Locked · track to reveal	Completed	89 est.	9 Jun 2017

View all trials62

Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA

Phase 3Enrolling by invitationNCT04360265 ↗

Primary completion: Aug 2027; estimated
Enrollment: 41; estimated
Start: Sept 2020; Ultragenyx Pharmaceutical Inc

Conditions

Mucopolysaccharidosis IIIA
MPS IIIA
Sanfilippo Syndrome
Sanfilippo A

Interventions

No Investigational Product
Adjuvant Immunomodulatory (IM) Therapy

Brief summary

The main objective of this study is to evaluate the safety/tolerability and efficacy of UX111 (previously known as ABO-102) in participants with Mucopolysaccharidosis IIIA (MPS IIIA).

Full record on ClinicalTrials.gov ↗

Change timeline

2 recorded · latest 1 Apr 2025

Completion date move · 1

Enrollment change · 1

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

Phase 1Active, not recruitingNCT04884815 ↗

Primary completion: Mar 2029; estimated
Enrollment: 82; estimated
Start: Sept 2021; Ultragenyx Pharmaceutical Inc

Conditions

Wilson Disease

Interventions

UX701
Standard of Care (SOC)

Brief summary

The primary objectives of this study are to evaluate the safety of single IV doses of UX701 in patients with Wilson disease, to select the UX701 dose with the best benefit/risk profile based on the totality of safety and efficacy data and to evaluate the effect of UX701 on copper regulation.

Full record on ClinicalTrials.gov ↗

Change timeline

5 recorded · latest 1 Oct 2025

Status change · 3

Completion date move · 2

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A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)

Phase 3CompletedNCT05139316 ↗

Primary completion: Feb 2024; actual
Enrollment: 49; estimated
Start: Nov 2021; Ultragenyx Pharmaceutical Inc

Conditions

Glycogen Storage Disease Type IA

Interventions

DTX401
Placebo
Oral prednisolone
Placebo for oral prednisolone

Brief summary

The primary objectives of this study are to evaluate the efficacy of DTX401 to reduce or eliminate dependence on exogenous glucose replacement therapy to maintain euglycemia and to maintain or improve the quality of glucose control.

Full record on ClinicalTrials.gov ↗

Change timeline

8 recorded · latest 1 Apr 2026

Status change · 4

Enrollment change · 2

Completion date move · 2

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

Phase 1CompletedNCT02991144 ↗

Primary completion: Dec 2021; actual
Enrollment: 16; estimated
Start: Jul 2017; Ultragenyx Pharmaceutical Inc

Conditions

Ornithine Transcarbamylase (OTC) Deficiency

Interventions

scAAV8OTC
Reactive Corticosteroid Taper Regimen
Prophylactic Corticosteroid Taper Regimen

Brief summary

This is a Phase 1/2, open-label, single arm, multicenter, safety and dose finding study of DTX301 in adults with late-onset OTC deficiency. The primary objective of the study is to determine the safety of single intravenous (IV) doses of DTX301.

Full record on ClinicalTrials.gov ↗

Change timeline

1 recorded · latest 2 Jan 2023

Enrollment change · 1

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

Phase 2CompletedNCT02214160 ↗

Primary completion: Dec 2020; actual
Enrollment: 94; estimated
Start: Dec 2014; Ultragenyx Pharmaceutical Inc

Conditions

Carnitine Palmitoyltransferase (CPT I or CPT II) Deficiency
Very Long Chain Acyl-CoA Dehydrogenase (VLCAD) Deficiency
Long-chain 3-hydroxy-acyl-CoA Dehydrogenase (LCHAD) Deficiency
Trifunctional Protein (TFP) Deficiency
Carnitine-acylcarnitine Translocase (CACT) Deficiency

Interventions

UX007

Brief summary

The primary objective of this study is to evaluate the long-term safety and efficacy of UX007 in participants with LC-FAOD. The secondary objectives of this study are to evaluate the effect of UX007 on energy metabolism in LC-FAOD and evaluate the impact of UX007 on clinical events associated with LC-FAOD.

Full record on ClinicalTrials.gov ↗

Change timeline

1 recorded · latest 1 Oct 2022

Completion date move · 1

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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

Phase 2TerminatedNCT02731690 ↗

Primary completion: Jan 2018; actual
Enrollment: 42; estimated
Start: Apr 2016; Ultragenyx Pharmaceutical Inc

Conditions

Hereditary Inclusion Body Myopathy
Distal Myopathy With Rimmed Vacuoles
Distal Myopathy, Nonaka Type
GNE Myopathy
Quadriceps Sparing Myopathy
Inclusion Body Myopathy 2

Interventions

Aceneuramic Acid Extended-Release

Brief summary

The primary objective of this Phase 2 study is to evaluate the safety of open-label 6 g/day Ace-ER in GNEM participants with severe ambulatory impairment.

Full record on ClinicalTrials.gov ↗

Change timeline

No changes recorded on this trial in the observed window.

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)

–CompletedNCT01784679 ↗

Primary completion: Nov 2017; actual
Enrollment: 319; estimated
Start: Apr 2013; Ultragenyx Pharmaceutical Inc

Conditions

Hereditary Inclusion Body Myopathy
GNE Myopathy
Nonaka Disease
Quadriceps Sparing Myopathy (QSM)
Distal Myopathy With Rimmed Vacuoles (DMRV)

Brief summary

HIBM is a severe progressive myopathy that typically presents in early adulthood as weakness in the distal muscles of the lower extremities and progresses proximally, leading to a loss of muscle strength and function, and ultimately a wheelchair-bound state. The rate of progression is gradual and variable over the…

Full record on ClinicalTrials.gov ↗

Change timeline

No changes recorded on this trial in the observed window.

GNEM: 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)

Phase 3CompletedNCT02377921 ↗

Primary completion: Jun 2017; actual
Enrollment: 89; estimated
Start: May 2015; Ultragenyx Pharmaceutical Inc

Conditions

Hereditary Inclusion Body Myopathy
Distal Myopathy With Rimmed Vacuoles
Distal Myopathy, Nonaka Type
GNE Myopathy

Interventions

aceneuramic acid extended-release (Ace-ER)
Placebo

Brief summary

The primary objective of this study is to evaluate the effect of 6 g/day aceneuramic acid extended-release (Ace-ER) treatment of participants with GNEM on upper extremity muscle strength (upper extremity composite \[UEC\] score) as measured by dynamometry.

Full record on ClinicalTrials.gov ↗

Change timeline

No changes recorded on this trial in the observed window.