Figure 1.
Study design.aAfter the administration of AMT-060 to each of the first 2 participants in each cohort, the Data Monitoring Committee evaluated available safety data during 24 hours before dosing of the next participant could be initiated. bProphylactic FIX replacement therapy was generally tapered between weeks 6 and 12 if FIX activity was ≥2.0 IU/dL in at least 2 consecutive visits. Investigators could taper prophylaxis later than 12 weeks at their discretion. The decision to continue tapering/withholding of prophylactic FIX replacement therapy was based on the individual assessment by the investigator but included the requirement to document that the participant could maintain a FIX activity level ≥2.0 IU/dL. cCohort 2 dosing was initiated after the completion of cohort 1 dosing and review of initial safety data by the Data Monitoring Committee.