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Enrolling by Invitation

NCT Number: NCT05768386

A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)

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.

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Key information

Age range

18 year and older

Sex eligibility

Male

Study type

Observational

Primary location

Royal Prince Alfred Hospital, Institute of Haematology, Sydney, New South Wales, Australia

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About this study

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).

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Participants must have completed their primary treatment study or be currently enrolled in the primary treatment study at the time of closure by the Sponsor. Participants may enroll in 270-401 even if they have restarted FVIII prophylaxis or other hemophilia A treatment.
  • Subjects must be capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol. If the subject is unable to provide consent, a legally authorized representative may provide written informed consent.

Exclusion criteria

  • Participants who do not directly enroll in 270-401 at the time of the study completion visit in their primary treatment study should enroll in 270-401 within 4 months of the date of that study completion visit. If a participant wishes to enroll in 270-401 after 4 months, they must receive prior approval from the Medical Monitor.
  • Participants must be overtly healthy and not have any condition that, in the opinion of the Investigator or Medical Monitor, would prevent the participant from fully complying with the requirements of the study and/or would impact or interfere with evaluation and interpretation of the study data (including, if applicable, advanced HIV disease).
  • Where applicable, per country regulation, the participant must not currently be committed to an institution by virtue of an order issued either by judicial or administrative authorities.

Treatment and study plan

Primary outcomes

  1. Evaluate the long-term safety of BMN 270

    Time frame: Duration of study (10 years)

    Occurrence of adverse drug reactions, serious adverse events, and events of special interest.

Secondary outcomes

  1. Evaluate the long-term effects of BMN 270 in subjects with hemophilia A previously treated in a BioMarin clinical trial

    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)

  2. Evaluate the use of hemostatic agents (ie, emicizumab)

    Time frame: Duration of study (10 years)

    Annualized use of concomitant hemostatic medications (annualized FVIII utilization and annualized FVIII infusion rate)

  3. Evaluate the long-term impact of BMN 270 on HRQoL

    Time frame: Duration of study (10 years)

    Changes in Haemo-QOL-A

Sponsors and collaborators

Lead sponsor

BioMarin Pharmaceutical

Industry

Registry information

Official study title

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

Important dates

Study start
2023
Primary completion
2040
Study completion
2040
First posted
Mar 14, 2023
Registry last updated
Jan 12, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

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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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