18 and older, male only, with Hemophilia A or Gene Therapy. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Number of Participants With Treatment Emergent Adverse EventsPrimary· Up to 5 years post-infusion.
A treatment-emergent adverse event (TEAE) is defined as any AE that newly appeared or worsened in severity following initiation of investigational product administration.
Participants with any AE
Group
Value
95% CI
BMN 270 6E13 vg/kg
3
Participants with any SAE
Group
Value
95% CI
BMN 270 6E13 vg/kg
1
Participants with any treatment-related AE
Group
Value
95% CI
BMN 270 6E13 vg/kg
3
Treatment-related SAEs
Group
Value
95% CI
BMN 270 6E13 vg/kg
1
Participants with any AE of Grade >= 3
Group
Value
95% CI
BMN 270 6E13 vg/kg
1
AEs leading to dose adjustment during infusion
Group
Value
95% CI
BMN 270 6E13 vg/kg
0
AEs leading to dose interruption during infusion
Group
Value
95% CI
BMN 270 6E13 vg/kg
0
AEs leading to study drug discontinuation
Group
Value
95% CI
BMN 270 6E13 vg/kg
0
Number of Participant With FVIII Activity >= 5 IU/dL at Week 26. Using Chromogenic Substrate Assay (CSA).Secondary· 26 weeks
Prior to BMN270 infusion, screening FVIII activity levels where participants had not received exogenous FVIII within 72 hours of assessment were below the lower limit of quantitation (LLOQ) as measured by CSA (LLOQ = 0.015 IU/mL).
Group
Value
95% CI
BMN 270 6E13 vg/kg
1
BMN 270 6E13 vg/kg
2
Mean Annualized Factor VIII Utilization During Week 5 and BeyondSecondary· Week 5 and Beyond (Follow-Up, up to 1782 Days)
The annualized utilization (IU/kg/year) of exogenous FVIII replacement therapy is defined as Sum of FVIII use (IU/kg) during calculation period/Total number of days during the calculation period ×365.25
Group
Value
95% CI
BMN 270 6E13 vg/kg
661.1
± 912.92
Mean Annualized Factor VIII Infusion Rate During Week 5 and BeyondSecondary· Week 5 and Beyond (Follow-Up, up to 1782 Days)
Annualized FVIII replacement infusion rate=(number of FVFIII replacement infusions during calculation period/sum(follow-up days) of the period)\*365.25
Group
Value
95% CI
BMN 270 6E13 vg/kg
21.9
± 30.61
Number of Participants Showed Reduction in the ABR Post-BMN 270 Infusion. Impact of BMN 270 on the Number of Bleeding Episodes Requiring Exogenous FVIII Therapy.Secondary· Week 5 and Beyond (Follow-Up, up to 1782 Days)
Annualized bleeding rate (ABR) (counts/yr.)=Number of bleeding episodes during calculation period/Total number of days during the calculation period ×365.25
Group
Value
95% CI
BMN 270 6E13 vg/kg
3
BMN 270 6E13 vg/kg
0
Adverse events — posted to ClinicalTrials.gov
Time frame: up to 5 years post-infusion..
Reporting threshold: 0%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
This study is being conducted by BioMarin Pharmaceutical Inc. as an open label, single dose study to determine the safety of valoctocogene roxaparvovec (an Adenovirus-Associated Virus (AAV) based gene therapy vector) in severe Hemophilia A patients with pre-existing antibodies against AAV5.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
NCT03392974 — Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of
· Phase 3
· completed
Other recruiting trials for Hemophilia A
Currently open trials in the same condition.
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· Phase 3
· recruiting
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· recruiting
NCT07523399 — Joint Health, Balance and Quality of Life in Adults With Hemophilia A
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NCT06833983 — To Evaluate the Clinical Study of GS1191-0445 Injection in the Treatment of Hemophilia A
· Phase 3
· recruiting
NCT06579144 — Pharmacokinetic Comparison of Efanesoctocog Alfa vs Other EHL-rFVIII Products in Participants With Severe Haemophilia A
· Phase 1
· recruiting
Other BioMarin Pharmaceutical trials
Trials by the same sponsor.
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· Phase 4
· not yet recruiting
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· Phase 2
· recruiting
NCT06309979 — A Study to Assess Growth in Children With Idiopathic Short Stature
· recruiting
NCT06455059 — Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia
· Phase 3
· active not recruiting
NCT06305234 — A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)
· recruiting
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by BioMarin Pharmaceutical
Last refreshed: 22 August 2025
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03520712.