The BMN 270 clinical development program consists of multiple interventional studies designed to assess the safety and efficacy of a single infusion of BMN 270 for at least 5 years post-infusion. This long-term follow-up study is needed to help further understand the long-term safety of BMN 270 beyond 5 years and to assess the durability of efficacy.
Study 270-401 will collect additional follow-up data in a single study for approximately 10 years among all subjects who consent to participate and have completed their primary treatment study (ie, for any study in which they received BMN 270).
Study Type
OBSERVATIONAL
Enrollment
172
Evaluate the long-term safety of BMN 270
Occurrence of adverse drug reactions, serious adverse events, and events of special interest.
Time frame: Duration of study (10 years)
Evaluate the long-term effects of BMN 270 in subjects with hemophilia A previously treated in a BioMarin clinical trial
Changes in annualized bleeding rate (ABR) (treated bleeds and all bleeds) and FVIII activity measured over time (CSA and OSA)
Time frame: Duration of study (10 years)
Evaluate the use of hemostatic agents (ie, emicizumab)
Annualized use of concomitant hemostatic medications (annualized FVIII utilization and annualized FVIII infusion rate)
Time frame: Duration of study (10 years)
Evaluate the long-term impact of BMN 270 on HRQoL
Changes in Haemo-QOL-A
Time frame: Duration of study (10 years)
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