Trial · NCT03370913
Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)
NCT03370913, Phase 3, Completed, BioMarin Pharmaceutical; results first posted 2023-11-28
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301). Trials, record NCT03370913. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03370913
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)
- NCT number
- NCT03370913
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 144 participants, actual
- Lead sponsor
- BioMarin Pharmaceutical
- Conditions
- Hemophilia A
- Interventions
- valoctocogene roxaparvovec
- First posted
- 2017-12-13
- Last updated
- 2025-03-25
- Other registries' IDs
- eudract:2017-003215-19
- Results first posted
- 2023-11-28
Source ClinicalTrials.gov
Registered outcomes8
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline in Annualized Number of Bleeding Episodes Irrespective of Exogenous FVIII Replacement Treatment [Annualized Bleeding Rate (ABR) for All Bleeds] in EEP. | 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 (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 products of extended half-life), whichever is later, to last visit by the data cut-off for the 2-year analysis, hereafter referred to as "Post FVIII Prophylaxis to Last Visit"). |
| Secondary | Change From Baseline in the Annualized Number of Bleeding Episodes Requiring Exogenous FVIII Replacement Therapy (ABR for Treated Bleeds) in the EEP. | 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 products of extended half-life), whichever is later, to last visit by the data cut-off for the 2-year analysis, hereafter referred to as "Post FVIII Prophylaxis to Last Visit"). |
| Secondary | Change From Baseline in FVIII Activity at Week 104 | Baseline to Week 104 | The change from baseline (assuming no treatment for severe hemophilia A) in FVIII activity, as measured by chromogenic substrate assay, at Weeks 104 post-BMN 270 infusion. Each subject's FVIII activity level at Week 104 is defined as the median of the values obtained at Week 104 with the analysis window defined. The baseline value is imputed as 1 IU/dL for each subject. Note: One of the subject's wk104 duplicate data issue was corrected in the 3 year analysis per which the mean (standard deviation) values are reported in outcome measure table. Baseline: prior to BMN 270 infusion while receiving FVIII prophylaxis. |
| Secondary | Change From Baseline in Annualized FVIII Utilization in EEP. | Baseline to EEP | The change from baseline in the annualized utilization (IU/kg/year) of exogenous FVIII replacement therapy in the Post FVIII Prophylaxis to Last Visit in the EEP. 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 plus the washout period (3 days for products of standard half-life or plasma-derived and 5 days for products of extended half-life), whichever is later, to last visit by the data cut-off for the 2-year analysis, hereafter referred to as "Post FVIII Prophylaxis to Last Visit"). |
| Secondary | Change From Baseline in Haemo-QoL-A Quality of Life: Total Score at Week 104 | Baseline to Week 104 | The change from baseline(assuming no treatment for severe hemophilia A) in Haemo-Qol-A score, at wk104 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-QoL-A domain(physical functioning, role functioning, worry, consequences of bleeding, emotional impact, treatment concern) scores range from 0 to 5 and the total score is derived by summing each domain score (range, 0 to 30). Domain and total scores are transformed to a 0 (minimum) to 100 (maximum) scale with higher scores indicating a better or less impaired haemophilia related quality of life. |
| Secondary | Change From Baseline in Haemo-QoL-A Quality of Life: Physical Functioning Domain Score, at Week 104 | Baseline to Week 104 | The change from baseline (assuming no treatment for severe hemophilia A) in Haemo-Qol-A score, at week 104 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). The Haemo-Qol-A physical functioning domain score is an average of each item value within a domain.The range of domain scores is 0 to 5; higher scores mean better HRQoL or less impairment for the domain. The physical functioning domain score is transformed to a 0 (minimum) to 100 (maximum) scale with higher scores indicating a better or less impaired haemophilia-related physical functioning |
| Secondary | Change From Baseline in Haemo-QoL-A Quality of Life: Consequences of Bleeding Domain Score, at Week 104 | Baseline to Week 104 | The change from baseline(assuming no treatment for severe hemophilia A)in Haemo-Qol-A score, at week 104 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-Qol-A consequences of bleeding domain score is an average of each item value within a domain. The range of domain scores is 0 to 5; higher scores mean better HRQoL or less impairment for the domain. The consequences of bleeding domain score is transformed to a 0 (minimum) to 100 (maximum) scale with higher scores indicating a better or less impaired haemophilia-related consequences of bleeding |
| Secondary | Change From Baseline in Haemo-QoL-A Quality of Life: Role Functioning Domain Score, at Week 104 | Baseline to Week 104 | The change from baseline (assuming no treatment for severe hemophilia A) in Haemo-Qol-A score, at week 104 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). The Haemo-Qol-A role functioning domain score is an average of each item value within a domain. The range of domain scores is 0 to 5; higher scores mean better HRQoL or less impairment for the domain. The role functioning domain score is transformed to a 0 (minimum) to 100 (maximum) scale with higher scores indicating a better or less impaired haemophilia-related role functioning. |
Source ClinicalTrials.gov
Outcomes8
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline in Annualized Number of Bleeding Episodes Irrespective of Exogenous FVIII Replacement Treatment [Annualized Bleeding Rate (ABR) for All Bleeds] in EEP. | Baseline to efficacy evaluation period (EEP) | Rollover Population (n=112): Subjects who completed approximately 6 months participation in the non-interventional study 270-902 before enrolling in 270-301, were HIV-negative at study screening, and received BMN 270 infusion in 270-301. | bleeds/year | MEAN |
| SECONDARY | Change From Baseline in the Annualized Number of Bleeding Episodes Requiring Exogenous FVIII Replacement Therapy (ABR for Treated Bleeds) in the EEP. | Baseline to EEP | Rollover Population | bleeds/year | MEAN |
| SECONDARY | Change From Baseline in FVIII Activity at Week 104 | Baseline to Week 104 | Modified Intent-to-Treat (mITT) Population (n=132): All HIV-negative subjects at study screening who were dosed in 270-301. | IU/dL | MEAN |
| SECONDARY | Change From Baseline in Annualized FVIII Utilization in EEP. | Baseline to EEP | Rollover population | IU/kg/yr | MEAN |
| SECONDARY | Change From Baseline in Haemo-QoL-A Quality of Life: Total Score at Week 104 | Baseline to Week 104 | mITT Population: Modified Intent-to-Treat Population - all HIV-negative subjects dosed in 270-301.~Change from baseline was based on the subjects with available measurements at both time points. | score on a scale | MEAN |
| SECONDARY | Change From Baseline in Haemo-QoL-A Quality of Life: Physical Functioning Domain Score, at Week 104 | Baseline to Week 104 | mITT population~Change from baseline was based on the subjects with available measurements at both time points. | score on a scale | MEAN |
| SECONDARY | Change From Baseline in Haemo-QoL-A Quality of Life: Consequences of Bleeding Domain Score, at Week 104 | Baseline to Week 104 | mITT population.~Change from baseline was based on the subjects with available measurements at both time points. | score on a scale | MEAN |
| SECONDARY | Change From Baseline in Haemo-QoL-A Quality of Life: Role Functioning Domain Score, at Week 104 | Baseline to Week 104 | mITT population~Change from baseline was based on the subjects with available measurements at both time points. | Score on a scale | MEAN |
Source ClinicalTrials.gov
Results by group8
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline in Annualized Number of Bleeding Episodes Irrespective of Exogenous FVIII Replacement Treatment [Annualized Bleeding Rate (ABR) for All Bleeds] in EEP. | BMN 270 (Valoctocogene Roxaparvovec) | -4.13 | Standard Deviation 6.93 | bleeds/year | |
| Change From Baseline in the Annualized Number of Bleeding Episodes Requiring Exogenous FVIII Replacement Therapy (ABR for Treated Bleeds) in the EEP. | BMN 270 (Valoctocogene Roxaparvovec) | -4.08 | Standard Deviation 6.57 | bleeds/year | |
| Change From Baseline in FVIII Activity at Week 104 | BMN 270 (Valoctocogene Roxaparvovec) | 21.94 | Standard Deviation 33.04 | IU/dL | |
| Change From Baseline in Annualized FVIII Utilization in EEP. | BMN 270 (Valoctocogene Roxaparvovec) | -3891.27 | Standard Deviation 1761.16 | IU/kg/yr | |
| Change From Baseline in Haemo-QoL-A Quality of Life: Total Score at Week 104 | BMN 270 (Valoctocogene Roxaparvovec) | 7.01 | Standard Deviation 12.55 | score on a scale | |
| Change From Baseline in Haemo-QoL-A Quality of Life: Physical Functioning Domain Score, at Week 104 | BMN 270 (Valoctocogene Roxaparvovec) | 4.90 | Standard Deviation 13.78 | score on a scale | |
| Change From Baseline in Haemo-QoL-A Quality of Life: Consequences of Bleeding Domain Score, at Week 104 | BMN 270 (Valoctocogene Roxaparvovec) | 10.29 | Standard Deviation 17.65 | score on a scale | |
| Change From Baseline in Haemo-QoL-A Quality of Life: Role Functioning Domain Score, at Week 104 | BMN 270 (Valoctocogene Roxaparvovec) | 7.37 | Standard Deviation 15.17 | Score on a scale |
Source ClinicalTrials.gov
Participant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | BMN 270 (Valoctocogene Roxaparvovec) | 134 | |
| Overall Study | COMPLETED | BMN 270 (Valoctocogene Roxaparvovec) | 132 | |
| Overall Study | NOT COMPLETED | BMN 270 (Valoctocogene Roxaparvovec) | 2 |
Source ClinicalTrials.gov
Adverse events94
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Alanine aminotransferase increased | Investigations | BMN 270 (Valoctocogene Roxaparvovec) | 119 | 134 | 385 |
| other | Aspartate aminotransferase increased | Investigations | BMN 270 (Valoctocogene Roxaparvovec) | 47 | 134 | 104 |
| other | Weight increased | Investigations | BMN 270 (Valoctocogene Roxaparvovec) | 22 | 134 | 36 |
| other | Blood creatine phosphokinase increased | Investigations | BMN 270 (Valoctocogene Roxaparvovec) | 17 | 134 | 22 |
| other | Blood lactate dehydrogenase increased | Investigations | BMN 270 (Valoctocogene Roxaparvovec) | 9 | 134 | 14 |
| other | Upper respiratory tract infection | Infections and infestations | BMN 270 (Valoctocogene Roxaparvovec) | 33 | 134 | 45 |
| other | Nasopharyngitis | Infections and infestations | BMN 270 (Valoctocogene Roxaparvovec) | 29 | 134 | 52 |
| other | Rhinitis | Infections and infestations | BMN 270 (Valoctocogene Roxaparvovec) | 12 | 134 | 16 |
| other | Folliculitis | Infections and infestations | BMN 270 (Valoctocogene Roxaparvovec) | 11 | 134 | 13 |
| other | Rash pustular | Infections and infestations | BMN 270 (Valoctocogene Roxaparvovec) | 11 | 134 | 12 |
1 to 10 of 94Source ClinicalTrials.gov
See all 94 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | BMN 270 (Valoctocogene Roxaparvovec) | 1 | 134 |
| Total, other adverse events | BMN 270 (Valoctocogene Roxaparvovec) | 134 | 134 |
| Total, serious adverse events | BMN 270 (Valoctocogene Roxaparvovec) | 24 | 134 |
Source ClinicalTrials.gov
Papers7
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT03370913
GET https://conductscience.org/api/trials/v1/NCT03370913/events?page=2