2 results
Approved WMOCompleted
The primary objectives of this study are: • To determine the longterm safety and tolerability of rhHNS administered via an intrathecal (IT) route once monthly for 8 years in patients with MPS IIIA, who have received and tolerated 6 months of…
Approved WMOCompleted
Primary- Phase 1: To determine the recommended dose (RD) of cabiralizumab in patients with pigmented villonodular synovitis (PVNS)/diffuse type tenosynovial giant cell tumor (dt-TGCT)- Phase 2: To estimate the objective response rate (ORR = CR+PR)…