Hemophilia A With InhibitorPhase 1/2GENEr8-INHNCT04684940
Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors
A Phase 1/2 study testing Valoctocogene roxaparvovec in people with Hemophilia A With Inhibitor and Hemophilia A With Anti Factor VIII, run by BioMarin Pharmaceutical at 9 sites in 7 countries.
Trial summary
This Phase I/II clinical study will evaluate the safety and efficacy of valoctocogene roxaparvovec in patients with severe haemophilia A and inhibitors to FVIII. Part A of the study will involve subjects who have active inhibitors to FVIII, and Part B involving subjects with a prior history of inhibitors. Read more about this study
- Who is it for?
- Ages 18 years and older, men only
- Conditions the study is for
- Hemophilia A With Inhibitor, Hemophilia A With Anti Factor VIII
- What is being tested?
- Valoctocogene roxaparvovec
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 9 sitesin 7 countries
- Study dates
- 10 Dec 2020to 20 Apr 2026
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
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
Who may be able to join
- Males ≥ 18 years of age with hemophilia A and documented prior residual FVIII activity ≤ 1 IU/dL including, but not limited to, at the time of detected inhibitors, at the time of signing the informed consent.
- History of a positive inhibitor result with the first positive result at least 12 month prior to Screening.
- Part A: Demonstrated no immunological tolerance to exogenous FVIII. Part B: Demonstrated tolerance to exogenous FVIII and negative FVIII inhibitor screening titer < 0.6 BU.
- Prophylactic or on-demand hemophilia therapy in the last 12 months. Bleeding, inhibitor \& hemophilia therapy Hx over previous 12 months.
- Sexually active participants must agree to use an acceptable method of effective contraception. Participants must agree to contraception use for at least 12 weeks post-infusion.
- Willing to abstain from consumption of alcohol for at least the first 52 weeks following BMN 270 infusion.
Who cannot join
- Detectable pre-existing antibodies to the AAV5 capsid.
- Any evidence of active infection or any immunosuppressive disorder; patients with HIV infection and undetectable viral load are not excluded.
- Currently undergoing, or plan to receive during the study, immune tolerance induction therapy or prophylaxis with FVIII (Part A only).
- Significant renal dysfunction or liver dysfunction, infection or history of hepatic malignancy.
- Evidence of any bleeding disorder not related to hemophilia A.
Trial locations and contacts
9 sites in 7 countries.
9 sites in 7 countries
Taiwan3 sites
Kaohsiung Medical University - Chung-Ho Memorial Hospital
Kaohsiung City
Taichung Veterans General Hospital
Taichung
National Taiwan University Hospital
Taipei
United States1 site
Children's Hospital Los Angeles
Los Angeles, California, 90027
About this study
Official title: A Phase 1/2 Safety, Tolerability, and Efficacy Study of BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients With Active or Prior Inhibitors
This Phase I/II clinical study will evaluate the safety and efficacy of valoctocogene roxaparvovec in patients with severe haemophilia A and inhibitors to FVIII. Part A of the study will involve subjects who have active inhibitors to FVIII, and Part B involving subjects with a prior history of inhibitors.
- Being tested
- Valoctocogene roxaparvovec
- Keywords
- Gene Therapy, Clotting Disorders, Blood Disorder, Blood Coagulation Disorders, Inherited Blood Coagulation Disorders, Hematologic Diseases, Coagulation Protein Disorders, Hemorrhagic Disorders, Genetic DIseases, Factor VIII, Coagulants, Hemophilia A, AAV5 vector
- Medical subject headings
- Hemostatic Disorders, Hematologic Diseases, Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, Hemorrhagic Disorders, Genetic Diseases, Inborn, Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Valoctocogene roxaparvovecBiological
Also called BMN 270 (GENEr8)
Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A
| Study group | Role | What they receive |
|---|---|---|
| Valoctocogene roxaparvovec Open Label | Experimental | Single administration of valoctocogene roxaparvovec at a dose of 6E13 vg/kg in Active Inhibitor Population (Part A) and Prior Inhibitor Population (Part B). Receives: Biological: Valoctocogene roxaparvovec |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with treatment-related adverse events, as assessed by Common Terminology Criteria for Adverse Events (CTCAE) v5.0 after administration of BMN 270.
Measured: 60 months
Secondary outcomes (5)
Change of the median Factor VIII activity.
Changes in the median Factor VIII activity (IU/mL) after administration of BMN 270 which will be measured using the chromogenic FVIII assay.
Measured: 60 months
A change in Factor VIII inhibitor titer (Part A) after administration of BMN 270.
FVIII inhibitor titer will be measured using a chromogenic Nijmegen-Bethesda assay.
Measured: 60 months
Absence of recurrence of Factor VIII inhibitors (Part B) after administration of BMN 270.
FVIII inhibitor titer will be measured using a chromogenic Nijmegen-Bethesda assay.
Measured: 60 months
Change in the annualized utilization of hemophilia therapy after administration of BMN 270
Measured: 60 months
Change in the annualized number of bleeding episodes requiring exogenous hemophilia therapy after administration of BMN 270.
Measured: 60 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Dec 2020
- Study start10 Dec 2020
- Primary completion20 Apr 2026
- Study completion20 Apr 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia A With InhibitorStudies 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
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- BioMarin Pharmaceutical
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Monitor, MD, Study director, BioMarin Pharmaceutical
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04684940
- Sponsor's protocol ID
- 270-205
- Other IDs
- 2019-003213-34, 2019
- Acronym
- GENEr8-INH
- Status last verified
- Jul 2026
- First submitted
- 30 Nov 2020
- Last updated
- 10 Jul 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2 Safety, Tolerability, and Efficacy Study of BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients With Active or Prior Inhibitors
- ClinicalTrials.gov ID
- NCT04684940
- Acronym / sponsor ID
- GENEr8-INH / 270-205
- Keywords
- Gene Therapy, Clotting Disorders, Blood Disorder, Blood Coagulation Disorders, Inherited Blood Coagulation Disorders, Hematologic Diseases, Coagulation Protein Disorders, Hemorrhagic Disorders
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- BioMarin PharmaceuticalIndustry (pharma, biotech, devices)
- Study start
- 10 Dec 2020 (actual)
- Primary completion
- 20 Apr 2026 (actual)
- Study completion
- 20 Apr 2026 (actual)
- First posted
- 28 Dec 2020 (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.
This Phase I/II clinical study will evaluate the safety and efficacy of valoctocogene roxaparvovec in patients with severe haemophilia A and inhibitors to FVIII. Part A of the study will involve subjects who have active inhibitors to FVIII, and Part B involving subjects with a prior history of inhibitors.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Valoctocogene roxaparvovec Open Label | Experimental | Biological: Valoctocogene roxaparvovecSingle administration of valoctocogene roxaparvovec at a dose of 6E13 vg/kg in Active Inhibitor Population (Part A) and Prior Inhibitor Population (Part B). |
Who may qualify
- Ages
- 18 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Males ≥ 18 years of age with hemophilia A and documented prior residual FVIII activity ≤ 1 IU/dL including, but not limited to, at the time of detected inhibitors, at the time of signing the informed consent.
- History of a positive inhibitor result with the first positive result at least 12 month prior to Screening.
- Part A: Demonstrated no immunological tolerance to exogenous FVIII. Part B: Demonstrated tolerance to exogenous FVIII and negative FVIII inhibitor screening titer < 0.6 BU.
- Prophylactic or on-demand hemophilia therapy in the last 12 months. Bleeding, inhibitor \& hemophilia therapy Hx over previous 12 months.
- Sexually active participants must agree to use an acceptable method of effective contraception. Participants must agree to contraception use for at least 12 weeks post-infusion.
Key exclusion criteria (5 listed)
- Detectable pre-existing antibodies to the AAV5 capsid.
- Any evidence of active infection or any immunosuppressive disorder; patients with HIV infection and undetectable viral load are not excluded.
- Currently undergoing, or plan to receive during the study, immune tolerance induction therapy or prophylaxis with FVIII (Part A only).
- Significant renal dysfunction or liver dysfunction, infection or history of hepatic malignancy.
- Evidence of any bleeding disorder not related to hemophilia A.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Valoctocogene roxaparvovecAlso: BMN 270 (GENEr8)Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A | Biological | Valoctocogene roxaparvovec Open Label |
Primary outcome
Number of participants with treatment-related adverse events, as assessed by Common Terminology Criteria for Adverse Events (CTCAE) v5.0 after administration of BMN 270.
Time frame: 60 months
5 secondary outcomes
Change of the median Factor VIII activity.
Time frame: 60 months
A change in Factor VIII inhibitor titer (Part A) after administration of BMN 270.
Time frame: 60 months
Absence of recurrence of Factor VIII inhibitors (Part B) after administration of BMN 270.
Time frame: 60 months
Change in the annualized utilization of hemophilia therapy after administration of BMN 270
Time frame: 60 months
Change in the annualized number of bleeding episodes requiring exogenous hemophilia therapy after administration of BMN 270.
Time frame: 60 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Jul 2026.
- Individual participant dataSharing planned: No
Related trials for Hemophilia A With Inhibitor
RecruitingPhase 3
Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors
Hemophilia A, Hemophilia B
RecruitingPhase 3
A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B
Hemophilia A, Hemophilia B
RecruitingObservational study
Treatment of Hemophilia A Patients With FVIII Inhibitors
Hemophilia A
Not yet recruitingObservational study
Prevalence of Inhibitor Development in Hemophilia A Patients Receiving Coageight: A Multicenter Cross-sectional Observational Study
Hemophilia A, Hemophilia
Not yet recruiting
A Study of Elranatamab for Inhibitor Eradication in Patients With Hemophilia A
Hemophilia A With Inhibitors
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. This study is not recruiting at the moment.
Medical Monitor, MD study director
BioMarin Pharmaceutical
9 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Valoctocogene roxaparvovec in people with Hemophilia A With Inhibitor and Hemophilia A With Anti Factor VIII, run by BioMarin Pharmaceutical at 9 sites in 7 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 6 inclusion and 5 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: finished, as of the sponsor's last update on 10 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in Taiwan, United States, Brazil, Israel, South Korea and 2 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.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Number of participants with treatment-related adverse events, as assessed by Common Terminology Criteria for Adverse Events (CTCAE) v5.0 after administration of BMN 270. (time frame: 60 months). 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 10 Jul 2026.