3 results
Approved WMOCompleted
Parts D and E: Primary Objectives:• To evaluate the safety and tolerability of VX-445 in TC with TEZ and IVA in subjects with cystic fibrosis (CF)• To evaluate the efficacy of VX-445 in TC with TEZ and IVA in subjects with CFSecondary Objectives• To…
Approved WMOWill not start
The main objective of this treatment protocol is to study the efficacy on the treatment response after adding a single gift of Mylotarg. Furthermore, monitoring of toxicity and long term efficacy is also important.
Approved WMORecruiting
The objective of the study is to assess the long-term safety and efficacy of intravenous ATB200 co-administration with oral AT2221 in adult subjects with late-onset Pompe disease.