CompletedPHASE1, PHASE2NCT03173521
Gene Therapy in Patients With Mucopolysaccharidosis Disease
Studying Mucopolysaccharidosis type 6
Last synced from ClinicalTrials.gov
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Report missing dataKey facts
- Sponsor
- Fondazione Telethon
- Principal Investigator
- Nicola Brunetti-Pierri, MDDepartment of Translational Medicine (DISMET) of "Federico II" University, Naples
- Intervention
- AAV2/8.TBG.hARSB(biological)
- Enrollment
- 9 enrolled
- Eligibility
- 4-65 years · All sexes
- Timeline
- 2017 – 2024
Study locations (2)
- Department of Translational Medicine (DISMET) of "Federico II" University, Naples, Naples, Italy
- Children's Hospital Hacettepe University, Ankara, Turkey (Türkiye)
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 NCT03173521 on ClinicalTrials.gov