Hemophilia AGENEr8-LTENCT05768386
A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)
An observational study of Hemophilia A, run by BioMarin Pharmaceutical at 41 sites in 12 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older, men only
- Conditions the study is for
- Hemophilia A
- How many people
- 172 participants
- Where
- 41 sitesin 12 countries
- Study dates
- 1 Jan 2023to Jan 2040
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- Men only
- No healthy volunteers
- Adult, Older adult
Study population: 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.
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
41 sites in 12 countries. Contact the study team or a site near you to ask about taking part.
41 sites in 12 countries
United States13 sites
University of Michigan, Pediatric Hematology and Oncology
Ann Arbor, Michigan, 48109-5718
Hemophilia and Thrombosis Center at the University of Colorado Anschutz Medical Campus
Aurora, Colorado, 80045
Nationwide Children's Hospital
Columbus, Ohio, 43205
Children's Hospital of Michigan, Hemophilia and Thrombosis Center
Detroit, Michigan, 48201
Orthopedic Institute for Children, Orthopedic Hemophilia Treatment Center
Los Angeles, California, 90007
University Medical Center, Inc,; DBA University of Louisville Hospital, James Graham Brown Cancer Center
Louisville, Kentucky, 40202-3229
Hemophilia Center of Western Pennsylvania
Pittsburgh, Pennsylvania, 15213
UC Davis Hemophilia Treatment Center
Sacramento, California, 95817
University of California San Diego, Hemophilia & Thrombosis Treatment Center
San Diego, California, 92121
University of California San Francisco - Moffit Hospital, Outpatient Hematology Clinic
San Francisco, California, 94143
UCN Hemophilia and Thrombosis Center
Shandon, California, 27517
Washington University School of Medicine, Department of Pediatrics, Division of Hematology/Oncology
St Louis, Missouri, 63110-1093
St Joseph's Children's Hospital, Center for Bleeding and Clotting Disorders
Tampa, Florida, 33614
United Kingdom8 sites
Addenbrooke's Hospital, Hemophilia and Thrombophilia Center
Cambridge
Glasgow Royal Infirmary, Department of Hematology
Glasgow, G4 0SF
Hammersmith Hospital, Clinical Research Facility, Centre for Translational and Experimental Medicine
London
Queen Elizabeth Hospital Birmingham. Adult Hemophilia Centre, West Midlands Adult Comprehensive Care Hemophilia Centre
London
Royal London Hospital, Barts and the London Hemophilia Center
London
St Thomas' Hospital, Centre for Hemophilia, Hemostasis and Thrombosis
London
Nuffield Orthopaedic Centre, Hemophilia & Thrombosis Center
Oxford, OX3 7LD
University Hospital Southampton NHS Foundation Trust
Southampton
Australia5 sites
Royal Adelaide Hospital
Adelaide, South Australia, 5000
Royal Brisbane and Women's Hospital
Herston, Queensland, 4029
The Alfred Hospital
Melbourne, Victoria, 3004
Fiona Stanley Hospital
Murdoch, Western Australia, 6150
Royal Prince Alfred Hospital, Institute of Haematology
Sydney, New South Wales, 2050
Brazil4 sites
Campinas University Clinical Hospital, Hematology and Hemotherapy Center (Hemocentro UNICAMP)
Campinas, São Paulo, 13083-878
Arthur De Siqueira Cavalcanti Hematology State Institute / Coordinating Hemocenter
Rio de Janeiro, Rio de Janeiro, 24000-000
Sao Paulo University Clinical Hospital
São Paulo, São Paulo, 05403 000
Holy Spirit Hematology and Hemotherapy Center
Vitória, Espírito Santo, 29040-090
France2 sites
CHRU Lille Hospital Center Heart-Lung Institute
Lille, 59000
CHU La Timone - La Timone Children's Hospital
Marseille, 13005
Germany2 sites
Vivantes Hospital in Friedrichshain
Berlin, State of Berlin, 10249
University Hospital Bonn, Institute of Experimental Hematology and Transfusion Medicine, Hemophilia Care Center
Bonn, North Rhine-Westphalia, 53127
Spain2 sites
Hospital Teresa Herrera, Department of Hematology and Hemotherapy, Unit of Hemostasis and Thrombosis
A Coruña, Galicia, 15006
University Hospital Virgen del Rocio (HUVR), Hematology Pediatrics
Seville, Andalusia, 41013
Belgium1 site
University Hospitals Leuven, Campus Gasthuisberg, Department of Heart and Vascular Disease
Leuven, 3000
Israel1 site
Chaim Sheba Medical Center, National Institute of Hemophilia and Coagulation
Ramat Gan, 5266202
Italy1 site
Maggiore Polyclinic Hospital, Foundation IRCCS Ca' Granda, Center for Hemophilia and Thrombosis "Angelo Bianchi Bonomi"
Milan, 20122
South Africa1 site
Charlotte Maxeke Johannesburg Academic Hospital, Hemophilia Comprehensive Care Center
Johannesburg, 2193
About this study
Official 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
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.
Read the detailed description
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).
- Conditions
- Hemophilia A
- Keywords
- Gene Therapy, Clotting Disorders, Blood Disorder, Blood Coagulation Disorders, Inherited Blood Coagulation disorders, Hematologic Diseases, Coagulation Protein Disorders, Hemorrhagic Disorders, Genetic Diseases, Inborn, Factor VIII, Coagulants
- Medical subject headings
- Hemophilia A, Hemostatic Disorders, Hematologic Diseases, Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, Hemorrhagic Disorders, Genetic Diseases, Inborn
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluate the long-term safety of BMN 270
Occurrence of adverse drug reactions, serious adverse events, and events of special interest.
Measured: Duration of study (10 years)
Secondary outcomes (3)
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)
Measured: 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)
Measured: Duration of study (10 years)
Evaluate the long-term impact of BMN 270 on HRQoL
Changes in Haemo-QOL-A
Measured: Duration of study (10 years)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Mar 2023
- Study start1 Jan 2023
- Primary completionJan 2040 (estimated)
- Study completionJan 2040 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia AStudies of the same condition, open and under way.
- More trials run by BioMarin PharmaceuticalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 172 participants (estimated)
Sponsor and investigators
- Lead sponsor
- BioMarin Pharmaceutical
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, MD, Study director, BioMarin Pharmaceutical
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05768386
- Sponsor's protocol ID
- 270-401
- Other IDs
- 2022-001246-38
- Acronym
- GENEr8-LTE
- Status last verified
- Jan 2026
- First submitted
- 23 Feb 2023
- Last updated
- 12 Jan 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT05768386
- Acronym / sponsor ID
- GENEr8-LTE / 270-401
- Conditions
- Hemophilia A
- Keywords
- Gene Therapy, Clotting Disorders, Blood Disorder, Blood Coagulation Disorders, Inherited Blood Coagulation disorders, Hematologic Diseases, Coagulation Protein Disorders, Hemorrhagic Disorders
- Study type
- Observational
- Recruitment status
- Enrolling by invitation
- Enrolment
- 172 participants (estimated)
- Lead sponsor
- BioMarin PharmaceuticalIndustry (pharma, biotech, devices)
- Study start
- 1 Jan 2023 (actual)
- Primary completion
- Jan 2040 (estimated)
- Study completion
- Jan 2040 (estimated)
- First posted
- 14 Mar 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- 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.
Key inclusion criteria (2 listed)
- 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.
Key exclusion criteria (3 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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.
3 secondary outcomes
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)
Evaluate the use of hemostatic agents (ie, emicizumab)
Time frame: Duration of study (10 years)
Evaluate the long-term impact of BMN 270 on HRQoL
Time frame: Duration of study (10 years)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Jan 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Medical Director, MD study director
BioMarin Pharmaceutical
41 sites in 12 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia A, run by BioMarin Pharmaceutical at 41 sites in 12 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: enrolling by invitation, as of the sponsor's last update on 12 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 41 sites in United States, United Kingdom, Australia, Brazil, France and 7 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evaluate the long-term safety of BMN 270 (time frame: Duration of study (10 years)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Jan 2026.