Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A
NCT06224907
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited
Hokkaido, Asahikawa, Japan
View Trial DetailsNCT Number: NCT05768386
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.
Interested in participating?
Request Info18 year and older
Male
Observational
Royal Prince Alfred Hospital, Institute of Haematology, Sydney, New South Wales, Australia
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).
Healthy volunteers accepted: No
Only the study team can determine whether someone qualifies for participation.
Inclusion criteria
Exclusion criteria
Time frame: Duration of study (10 years)
Occurrence of adverse drug reactions, serious adverse events, and events of special interest.
Time frame: Duration of study (10 years)
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)
Annualized use of concomitant hemostatic medications (annualized FVIII utilization and annualized FVIII infusion rate)
Time frame: Duration of study (10 years)
Changes in Haemo-QOL-A
BioMarin Pharmaceutical
Industry
A Long-Term Follow-Up Study in Subjects With Severe Hemophilia A Who Received BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in a Prior BioMarin Clinical Trial
Acronym: GENEr8-LTE
OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.
View the official ClinicalTrials.gov record (opens in a new tab)This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.
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