2 results
Approved WMOCompleted
Primary objectives of the study are:• Arm 1 and Arm 3 (PTPs): to characterize the long-term safety profile of rVIII SingleChain with respect to inhibitor development in PTPs.• Arm 2 (PUPs): • To characterize the safety with respect to inhibitor…
Not approvedWill not start
The primary objective of this study is:* To assess the safety and tolerability of TPN-101 in patients with C9ORF72 ALS/FTDThe exploratory objectives of this study are:* To assess the concentrations of TPN-101 in plasma and cerebrospinal fluid (CSF…