An observational study in Hemophilia A, sponsored by BioMarin Pharmaceutical. Enrolling by invitation at 41 sites in 12 countries. Open to male participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-01-12.
Sponsored by BioMarin Pharmaceutical · Observational
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).
844 studies on the registry are indexed under Hemophilia A; 138 are open to participants now.
This study's planned enrollment of 172 is above the median of 81 across 310 observational studies indexed under Hemophilia A.
Browse Hemophilia A studies →BioMarin Pharmaceutical is the lead sponsor of 112 studies on the registry; 13 are open to participants now.
Of its 17 completed or terminated interventional studies of FDA-regulated products, 12 (71%) have results posted.
Counted across the registry records on this site, refreshed daily.
Subjects must have completed their primary treatment study in which they were dosed BMN 270. Subjects may enroll in 270-401 even if they have restarted FVIII prophylaxis or other hemophilia A treatment.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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)
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
No contact was published for this record. The registry link below has the sponsor’s details.
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