3 results
Approved WMOCompleted
The objective of the study is to assess the safety, tolerability and efficacy of PRO044.
Approved WMORecruiting
The primary aim of the trial is to demonstrate the non-inferiority of CSL222 (formerly AMT-061) (2 × 1013 gc/kg) during the 52 weeks following establishment of stable factor IX expression (months 6 to 18) post-treatment (CSL222 (formerly AMT-061))…
Approved WMOPending
The primary, secondary and exploratory objectives of the study are as follows:Part APrimary:• To evaluate the safety and tolerability of the different GEH200520 Injection mass doses and a fixed dose of GEH200521 (18F) Injection when administered…