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RARE

Ultragenyx Pharmaceutical Inc

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15Trials in this database
17Recorded changes · last 12 months
Partial coverageOnly the trials and history collected so far

Research sample: selected versions from May 2023 onward plus a preceding baseline where available. Only selected fields were retained; endpoint measures were truncated to 300 characters. This is not complete trial or sponsor history. Daily snapshots begin when collection starts.

Completion dates · Pro

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Pro summarizes how far estimated completion dates moved later and counts trials with edits to their main study measures, using the history available here. View Pro features

Included trials

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TrialSponsorPhaseStatusPrimary completion
Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Extension Study for Subjects Previously Enrolled in Triheptanoin StudiesNCT02214160RAREPhase 2Completed2020-12-03Actual
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSHNCT02716246RAREPhase 2, Phase 3Recruiting2029-03Estimated
A Study in Adult Patients With Type I, III or IV Osteogenesis Imperfecta Treated With BPS804NCT03118570RAREPhase 2Completed2019-10-01Actual
Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIANCT04360265RAREPhase 3Enrolling by invitation2027-08Estimated
A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson DiseaseNCT04884815RAREPhase 1, Phase 2Active not recruiting2029-03Estimated
Setrusumab vs Placebo for Osteogenesis ImperfectaNCT05125809RAREPhase 2, Phase 3Active not recruiting2025-10-20Actual
A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)NCT05139316RAREPhase 3Completed2024-02-20Actual
Long-term Extension Study of Setrusumab in Adults With Type I, III, or IV Osteogenesis ImperfectaNCT05312697RAREPhase 2Terminated2022-07-07Actual
Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) DeficiencyNCT05345171RAREPhase 3Active not recruiting2027-09Estimated
Setrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis ImperfectaNCT05768854RAREPhase 3Active not recruiting2025-10-23Actual
A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAODNCT05933200RAREPhase 3Active not recruiting2027-08Estimated
Long-term Extension of GTX-102 in Angelman SyndromeNCT06415344RAREPhase 3Active not recruiting2029-02Estimated
Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)NCT06617429RAREPhase 3Active not recruiting2026-07-10Actual
Setrusumab in Pediatric Japanese Subjects With Osteogenesis ImperfectaNCT06636071RAREPhase 3Active not recruiting2027-01Estimated
A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)NCT07157254RAREPhase 2Active not recruiting2030-01Estimated

Latest recorded trial changes

Long-term Extension of GTX-102 in Angelman Syndrome

NCT06415344 · Ultragenyx Pharmaceutical Inc

· 1 field changed

Main study measure wording changed

Text changed; whether the study measures changed has not been verified. Saved text may be shortened.

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− Before

Measure: Frequency, Severity, and Relationship to Investigational Drug of Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

+ After

Measure: Incidence of Treatment Emergent Adverse Events (TEAEs)

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