18 and older, male only, with Hemophilia A. 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.
Change From Baseline in FVIII Activity as Measured by Chromogenic Substrate Assay at Week 52.Primary· Baseline to Week 52
The change from baseline (assuming no treatment for severe hemophilia A) in FVIII activity, as measured by chromogenic substrate assay (CSA), at Week 52 (during Weeks 49 - 52) post-BMN 270 infusion.
Each participant's FVIII activity level at Week 52 is defined as the median of the values obtained within the analysis window at Weeks 49-52. The baseline value will be imputed as 1 IU/dL, since there will be no washout of severe hemophilia A participants' usual FVIII prophylaxis (in order to avoid increasing the risk of bleeding) prior to BMN 270 infusion. Post-BMN 270 infusion values for FVIII a
Group
Value
95% CI
BMN 270 (Valoctocogene roxaparvovec)
15.13
± 22.38
Change From Baseline in Annualized Utilization of Exogenous FVIII Replacement Therapy in EEPSecondary· Baseline to efficacy evaluation period (EEP)
The change from baseline (prior to BMN 270 infusion while receiving FVIII prophylaxis) in the annualized utilization (IU/kg/year) of exogenous FVIII replacement therapy in the efficacy evaluation period ("Post-FVIII Prophylaxis period").
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.
Baseline: prior to BMN 270 infusion while receiving FVIII prophylaxis
EEP: From Week 5 post-BMN 270 infusion (Study Day 33) or the end of FVIII prophylaxis
Group
Value
95% CI
BMN 270 (Valoctocogene Roxaparvovec)
-4150.09
± 3208.50
Change From Baseline in the Annualized Number of Bleeding Episodes Irrespective of Exogenous FVIII Replacement Treatment (Annualized Bleeding Rate, ABR for All Bleeds) in EEPSecondary· Baseline to efficacy evaluation period (EEP)
All bleeds comprises both treated and non-treated bleeds. In this definition, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded. All bleeds are any reported bleeding events regardless of the use of FVIII or other treatments.
ABR for all bleeds= Number of bleeding episodes for all bleeds during the calculation period / total number of days during the calculation period \* 365.25.
Baseline: prior to BMN 270 infusion while receiving FVIII prophylaxis.
EEP: From Week 5 post-BMN 270 infusion (St
Group
Value
95% CI
BMN 270 (Valoctocogene Roxaparvovec)
-6.15
± 17.24
Change From Baseline in the Annualized Number of Bleeding Episodes Requiring Exogenous FVIII Replacement Treatment (ABR for Treated Bleeds) in the EEP.Secondary· Baseline to EEP
ABR for treated bleeds=Number of bleeding episodes for treated bleeds during the calculation period/total number of days during the calculation period \* 365.25
Bleeds that were treated with FVIII replacement therapy (recorded as "treatment for bleed") within 72 hours and were not associated with surgery or a procedure were included.
Baseline: prior to BMN 270 infusion while receiving FVIII prophylaxis.
EEP: From Week 5 post-BMN 270 infusion (Study Day 33) or the end of FVIII prophylaxis plus the washout period (3 days for products of standard half-life or plasma-derived and 5 days for prod
Group
Value
95% CI
BMN 270 (Valoctocogene Roxaparvovec)
-3.78
± 10.14
Change From Baseline in Haemo-QoL-A Quality of Life: Total Score at Week 52Secondary· Baseline to Week 52
The change from baseline(assuming no treatment for severe hemophilia A) in Haemo-Qol-A score, at wk52 post-BMN 270 infusion.The Haemo-Qol-A questionnaire is a fit for purpose hemophilia-specific health related quality of life(HRQoL)questionnaire for adults consisting of 41 items covering 6 domains(Physical Functioning, Role Functioning,Worry,Consequences of Bleeding,Emotional Impact \&Treatment Concerns). The Haemo-Qol-A items are answered on a 6-point Likert scale ranging from 0(none of the time)to 5 (all of the time).The recall period for the Haemo-Qol-A is one month (4-weeks).
The Haemo-Qo
Group
Value
95% CI
BMN 270 (Valoctocogene Roxaparvovec)
6.70
± 8.96
Change From Baseline in Haemo-QoL-A Quality of Life: Physical Functioning Domain Score, at Week 52Secondary· Baseline to Week 52
The change from baseline (assuming no treatment for severe hemophilia A) in Haemo-Qol-A score, at week 52 post BMN 270 infusion. The Haemo-Qol-A questionnaire is a fit for purpose hemophilia-specific health related quality of life (HRQoL) questionnaire for adults consisting of 41 items covering six domains (Physical Functioning, Role Functioning, Worry, Consequences of Bleeding, Emotional Impact and Treatment Concerns).The Haemo-Qol-A items are answered on a 6-point Likert scale ranging from 0 (none of the time) to 5 (all of the time).The recall period for the Haemo-Qol-A is one month (4-weeks
Group
Value
95% CI
BMN 270 (Valoctocogene Roxaparvovec)
6.43
± 8.51
Change From Baseline in Haemo-QoL-A Quality of Life: Consequences of Bleeding Domain Score, at Week 52Secondary· Baseline to Week 52
The change from baseline(assuming no treatment for severe hemophilia A)in Haemo-Qol-A score, at week 52 post-BMN 270 infusion. The Haemo-Qol-A questionnaire is a fit for purpose hemophilia-specific health related quality of life(HRQoL)questionnaire for adults consisting of 41 items covering 6 domains(Physical Functioning,Role Functioning,Worry,Consequences of Bleeding,Emotional Impact and Treatment Concerns). The Haemo-Qol-A items are answered on a 6-point Likert scale ranging from 0(none of the time) to 5(all of the time). The recall period for the Haemo-Qol-A is one month (4-wks).
The Haemo
Group
Value
95% CI
BMN 270 (Valoctocogene Roxaparvovec)
10.68
± 16.33
Change From Baseline in Haemo-QoL-A Quality of Life: Role Functioning Domain Score, at Week 52Secondary· Baseline to Week 52
The change from baseline (assuming no treatment for severe hemophilia A) in Haemo-Qol-A score, at week 52 post BMN 270 infusion. The Haemo-Qol-A questionnaire is a fit for purpose hemophilia-specific health related quality of life (HRQoL) questionnaire for adults consisting of 41 items covering six domains (Physical Functioning, Role Functioning, Worry, Consequences of Bleeding, Emotional Impact and Treatment Concerns).The Haemo-Qol-A items are answered on a 6-point Likert scale ranging from 0 (none of the time) to 5 (all of the time). The recall period for the Haemo-Qol-A is one month (4-week
Group
Value
95% CI
BMN 270 (Valoctocogene Roxaparvovec)
6.03
± 10.29
Adverse events — posted to ClinicalTrials.gov
Time frame: Up to 112 weeks.
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
This Phase III clinical study will evaluate the safety and effectiveness of valoctocogene roxaparvovec in combination with prophylactic corticosteroids in patients with severe hemophilia A.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
NCT03370913 — Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301
· Phase 3
· completed
NCT02576795 — Gene Therapy Study in Severe Haemophilia A Patients (270-201)
· Phase 1, PHASE2
· completed
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Trials by the same sponsor.
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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: 8 October 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/NCT04323098.