7 results
The primary objectives of this study is:* To determine the safety and tolerability of rhHNS via ascending doses administered via a surgically implanted intrathecal drug device (IDDD) once monthly for 6 months, in patientswith MPS IIIA.The secondary…
The primary objective of this study is to assess the potential clinical efficacy of HGT-1410administered via a surgically implanted IDDD in patients with MPS IIIA. Efficacy will bemeasured as a meaningful amelioration in the progression of cognitive…
Primary Objective(s): To evaluate long-term safety in patients with mucopolysaccharidosis type IIIA disease (MPS IIIA or Sanfilippo Type A) who received HGT-1410Secondary Objective(s):To evaluate:• The long-term cognitive function as measured by the…
The objective of this study is to test the safety, tolerability and anti-tumor activity of the combination of the investigational products epacadostat and pembrolizumab, compared to pembrolizumab as mono therapy, in patients with unresectable or…
The objective of this study is to test the safety, tolerability and anti-tumor activity of the combination of the investigational products epacadostat and pembrolizumab, compared to pembrolizumab as mono therapy, in patients with cisplatin-…
The objective of this study is to test the safety, tolerability and anti-tumor activity of the combination of the investigational products epacadostat and pembrolizumab, compared to pembrolizumab as mono therapy, in patients with advanced/…
Dose Escalation: * To determine the MTD or the RP2D of tazemetostat when administered as an oral suspension BID in pediatric subjects with relapsed/refractory rhabdoid tumors, INI1-negative tumors or synovial sarcoma.* To evaluate the preliminary…