A Phase 3 interventional study of Valoctocogene Roxaparvovec in Hemophilia A, sponsored by BioMarin Pharmaceutical. Completed at 1 site in United States. Open to male participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2023-10-03.
Sponsored by BioMarin Pharmaceutical · Phase 3, Interventional, and Treatment
This Phase III clinical study will assess the efficacy of BMN 270 defined as FVIII activity, during weeks 49-52 following intravenous infusion of BMN 270 and assess the impact of BMN 270 on usage of exogenous FVIII replacement therapy and the number of bleeding episodes from week 5 to week 52.
844 studies on the registry are indexed under Hemophilia A; 138 are open to participants now.
This study's enrollment of 1 is below the median of 30 across 499 interventional studies indexed under Hemophilia A.
Browse Hemophilia A studies →BioMarin Pharmaceutical is the lead sponsor of 112 studies on the registry; 13 are open to participants now.
Of its 17 completed or terminated interventional studies of FDA-regulated products, 12 (71%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Single administration of valoctocogene roxaparvovec at a dose of 4E13 vg/kg
Biological: Valoctocogene Roxaparvovec
Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A
Also known as: BMN 270
Change of the Median Factor VIII (FVIII) Activity
Change of the FVIII activity, as measured by chromogenic substrate assay, at Week 52 post-BMN 270 infusion.
Time frame: Week 52
Change in the Annualized Utilization (IU/kg) of Exogenous FVIII Replacement Therapy
Change in the annualized utilization (IU/kg) of exogenous FVIII replacement therapy during Week 5 to Week 52 post-BMN 270 infusion from the baseline utilization of exogenous FVIII replacement therapy
Time frame: Weeks 5 through Week 52
Change in the Annualized Number of Bleeding Episodes Requiring Exogenous FVIII Replacement Treatment
Change in the annualized number of bleeding episodes requiring exogenous FVIII replacement treatment (annualized bleeding rate, ABR) during Week 5 to Week 52 of the study post-BMN 270 infusion from the baseline ABR
Time frame: Weeks 5 though Week 52
| Milestone | BMN 270 4E13 vg/kg |
|---|---|
| Started | 1 |
| Completed | 1 |
| Not completed | 0 |
Change of the FVIII activity, as measured by chromogenic substrate assay, at Week 52 post-BMN 270 infusion.
| IU/dL | BMN 270 4E13 vg/kg |
|---|---|
| Change of the Median Factor VIII (FVIII) Activity | 4.1 ± NA |
Change in the annualized utilization (IU/kg) of exogenous FVIII replacement therapy during Week 5 to Week 52 post-BMN 270 infusion from the baseline utilization of exogenous FVIII replacement therapy
| IU/kg/yr | BMN 270 4E13 vg/kg |
|---|---|
| Change in the Annualized Utilization (IU/kg) of Exogenous FVIII Replacement Therapy | -4058.24 ± NA |
Change in the annualized number of bleeding episodes requiring exogenous FVIII replacement treatment (annualized bleeding rate, ABR) during Week 5 to Week 52 of the study post-BMN 270 infusion from the baseline ABR
| episodes/year | BMN 270 4E13 vg/kg |
|---|---|
| Change in the Annualized Number of Bleeding Episodes Requiring Exogenous FVIII Replacement Treatment | 5.77 ± NA |
Collected over First 52 weeks post infusion. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| BMN 270 4E13 vg/kg | 0/1 (0%) | 0/1 (0%) | 1/1 (100%) |
| Total | 0/1 (0%) | 0/1 (0%) | 1/1 (100%) |
| Event | BMN 270 4E13 vg/kg | Total |
|---|---|---|
| PyrexiaGeneral disorders | 1/1 | 1/1 |
| Hepatocellular injuryHepatobiliary disorders | 1/1 | 1/1 |
| NasopharyngitisInfections and infestations | 1/1 | 1/1 |
| Alanine aminotransferase increasedInvestigations | 1/1 | 1/1 |
| MyalgiaMusculoskeletal and connective tissue disorders | 1/1 | 1/1 |
| Oropharyngeal painRespiratory, thoracic and mediastinal disorders | 1/1 | 1/1 |
| Age, Continuous(years) | BMN 270 4E13 vg/kg |
|---|---|
| Mean | 45.0 ± NA |
| Age, Customized(participants) | BMN 270 4E13 vg/kg |
|---|---|
| 18 - <65 | 1 |
| >= 65 | 0 |
| Sex: Female, Male(Participants) | BMN 270 4E13 vg/kg |
|---|---|
| Female | 0 |
| Male | 1 |
| Ethnicity (NIH/OMB)(Participants) | BMN 270 4E13 vg/kg |
|---|---|
| Hispanic or Latino | 0 |
| Not Hispanic or Latino | 1 |
| Unknown or Not Reported | 0 |
| Race/Ethnicity, Customized(participants) | BMN 270 4E13 vg/kg |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 0 |
| White | 1 |
| Other | 0 |
| Region of Enrollment(participants) | BMN 270 4E13 vg/kg |
|---|---|
| United States | 1 |
PDFs hosted by the registry, ClinicalTrials.gov.
This study is completed, as verified in Sep 2023. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Valoctocogene Roxaparvovec