Recruiting
Osteopetrosis
RecruitingStudy of gene-modified stem cells (FT024) in children with TCIRG1-related osteopetrosis using treosulfan, thiotepa, and plerixafor for treatment
Registered drugNo placebo
Study of gene-modified stem cells (FT024) in children with TCIRG1-related osteopetrosis using treosulfan, thiotepa, and plerixafor for treatment
Registered drugNo placebo
- Disease:Osteopetrosis
- Substances:PlerixaforThiotepa+2
Countries:
Italy
Italy- Participants:0–17 years
- Substances:Plerixafor
- Sponsor:Fondazione Telethon Ets
