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NCT04909346
Adeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson Disease
trial in Ornithine Transcarbamylase Deficiency in 51 participants. Terminated before completion.
17 November 2022
Quick facts
| Lead sponsor | Ultragenyx Pharmaceutical Inc |
|---|---|
| Status | Terminated |
| Study type | OBSERVATIONAL |
| Enrollment | 51 |
| Start date | 23 June 2021 |
| Primary completion | 17 November 2022 |
| Estimated completion | 17 November 2022 |
| Sites | 5 locations across Brazil, United Kingdom, Canada, United States, Spain |
Conditions studied
- Ornithine Transcarbamylase Deficiency — all drugs for Ornithine Transcarbamylase Deficiency →
- Wilson Disease — all drugs for Wilson Disease →
- Glycogen Storage Disease Type IA — all drugs for Glycogen Storage Disease Type IA →
Sponsor
Ultragenyx Pharmaceutical Inc — full company profile →
Who can join
18 and older, any sex, with Ornithine Transcarbamylase Deficiency or Wilson Disease. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
The objective of this observational study is to evaluate the seroprevalence of anti-AAV antibodies in subjects with Ornithine Transcarbamylase (OTC) deficiency, Glycogen Storage Disease Type Ia (GSDIa), and Wilson Disease
Publications & conference data
1 peer-reviewed publication reference this trial (live from Europe PMC):
-
Emerging Perspectives on Prime Editor Delivery to the Brain.
BenDavid E, Ramezanian S, Lu Y, Rousseau J, et al · · 2024 · cited 4× · PMID 38931430 · DOI 10.3390/ph17060763
Verify or expand the search:
- PubMed search for NCT04909346
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
Other recruiting trials for Ornithine Transcarbamylase Deficiency
Currently open trials in the same condition.
- NCT06488313 — A Study to Evaluate the Pharmacodynamics and Safety of ARCT-810 in Participants With OTCD · Phase 2 · recruiting
- NCT05092685 — Halting Ornithine Transcarbamylase Deficiency With Recombinant AAV in ChildrEn · Phase 1, PHASE2 · recruiting
- NCT04908319 — Hepatic Histopathology in Urea Cycle Disorders · recruiting
- NCT04612764 — Liver Disease in Urea Cycle Disorders · recruiting
Other Ultragenyx Pharmaceutical Inc trials
Trials by the same sponsor.
- NCT04812106 — Long-Chain Fatty Acid Oxidation Disorders Online Disease Monitoring Program · terminated
- NCT05196165 — Clinical Survey Study to Assess Physical Function and the Incidence of Hypoglycemia in Participants With Glycogen Storag · terminated
- NCT05312697 — Long-term Extension Study of Setrusumab in Adults With Type I, III, or IV Osteogenesis Imperfecta · Phase 2 · terminated
- NCT04783428 — Tumor-induced Osteomalacia Disease Monitoring Program · active not recruiting
- NCT05139316 — A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen S · Phase 3 · completed
Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT04909346 (US National Library of Medicine, public domain)
- Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by Ultragenyx Pharmaceutical Inc
- Last refreshed: 12 June 2023
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04909346.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing