Enrolling by invitationPhase 3NCT04360265
Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA
Studying Mucopolysaccharidosis
Last synced from ClinicalTrials.gov
ℹ
Clinical trial records are synced from ClinicalTrials.gov through automated extraction.
Report missing dataKey facts
- Sponsor
- Ultragenyx Pharmaceutical Inc
- Principal Investigator
- Medical DirectorUltragenyx Pharmaceutical Inc
- Intervention
- No Investigational Product(other)
- Enrollment
- 41 enrolled
- Eligibility
- All sexes
- Timeline
- 2020 – 2027
Study locations (4)
- Nationwide Children's Hospital, Columbus, Ohio, United States
- Women's and Children's Hospital, North Adelaide, South Australia, Australia
- Vall d'Hebron Barcelona Campus, Barcelona, Spain
- Hospital Clínico Universitario de Santiago, Santiago de Compostela, Spain
Primary source
Recruitment status, site addresses, contacts, and full eligibility criteria can change between syncs. Always verify with the trial team before planning travel or treatment.
Open NCT04360265 on ClinicalTrials.govOther trials for Mucopolysaccharidosis
Additional recruiting or active studies for the same condition.
- ENROLLING BY INVITATIONPHASE2, PHASE3NCT06075537An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007Denali Therapeutics Inc.
- ENROLLING BY INVITATIONNCT06103487Long Term Follow-Up for RGX-111REGENXBIO Inc.
- RECRUITINGPHASE1NCT05682144ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS IImmusoft of CA, Inc.
- ENROLLING BY INVITATIONPHASE3NCT05594992An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in MPS II (Hunter Syndrome) SubjectsJCR Pharmaceuticals Co., Ltd.
- ACTIVE NOT RECRUITINGPHASE1NCT05422482A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients with MPS ⅡGC Biopharma Corp
- RECRUITINGPHASE2, PHASE3NCT05371613A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)Denali Therapeutics Inc.
- RECRUITINGNANCT05619900Registry of Patients Diagnosed With Lysosomal Storage DiseasesUniversity of California, San Francisco
- ACTIVE NOT RECRUITINGPHASE3NCT04573023A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)JCR Pharmaceuticals Co., Ltd.