3 results
Approved WMOCompleted
The primary objective is to evaluate the safety of ReFacto AF in previously untreated patients (no prior exposure to factor products or any blood products) of less than 6 years of age.
DDAVP treatment combined with FVIII clotting factor concentrates in patients with mild hemophilia A.
Primary objectives:To assess the proportion of non-severe hemophilia A patients within FVIII target levels with the DDAVP and FVIII concentrate combination treatment in the first 72 hours after the start of combination treatment, without adding off-…
Approved WMOCompleted
To evaluate the efficacy of VX-121/TEZ/D-IVA in CF subjects who are homozygous forF508del, heterozygous for F508del and a gating (F/G) or residual function (F/RF) mutation, orhave at least 1 other TCR CFTR mutation and no F508del mutation