Trial Outcomes & Findings for Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg (NCT NCT03392974)

NCT ID: NCT03392974

Last Updated: 2023-10-03

Results Overview

Change of the FVIII activity, as measured by chromogenic substrate assay, at Week 52 post-BMN 270 infusion.

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 1 participants
• Primary outcome timeframe: Week 52
• Results posted on: 2023-10-03

Participant Flow

Participant milestones

Measure	BMN 270 4E13 vg/kg Single administration of valoctocogene roxaparvovec at a dose of 4E13 vg/kg
Overall Study STARTED	1
Overall Study COMPLETED	1
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg

Baseline characteristics by cohort

Measure	BMN 270 4E13 vg/kg n=1 Participants Single administration of valoctocogene roxaparvovec at a dose of 4E13 vg/kg
Age, Continuous	45.0 years STANDARD_DEVIATION NA • n=5 Participants
Age, Customized 18 - <65	1 participants n=5 Participants
Age, Customized >= 65	0 participants n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race/Ethnicity, Customized American Indian or Alaska Native	0 participants n=5 Participants
Race/Ethnicity, Customized Asian	0 participants n=5 Participants
Race/Ethnicity, Customized Native Hawaiian or Other Pacific Islander	0 participants n=5 Participants
Race/Ethnicity, Customized Black or African American	0 participants n=5 Participants
Race/Ethnicity, Customized White	1 participants n=5 Participants
Race/Ethnicity, Customized Other	0 participants n=5 Participants
Region of Enrollment United States	1 participants n=5 Participants

PRIMARY outcome

Timeframe: Week 52

Population: Intention-to-treat

Change of the FVIII activity, as measured by chromogenic substrate assay, at Week 52 post-BMN 270 infusion.

Outcome measures

Measure	BMN 270 4E13 vg/kg n=1 Participants Single administration of valoctocogene roxaparvovec at a dose of 4E13 vg/kg
Change of the Median Factor VIII (FVIII) Activity	4.1 IU/dL Standard Deviation NA The number of participants is 1, so the Standard Deviation was not calculated

SECONDARY outcome

Timeframe: Weeks 5 through Week 52

Population: Intention-to-treat

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

Outcome measures

Measure	BMN 270 4E13 vg/kg n=1 Participants Single administration of valoctocogene roxaparvovec at a dose of 4E13 vg/kg
Change in the Annualized Utilization (IU/kg) of Exogenous FVIII Replacement Therapy	-4058.24 IU/kg/yr Standard Deviation NA The number of participants is 1, so the Standard Deviation was not calculated

SECONDARY outcome

Timeframe: Weeks 5 though Week 52

Population: Intention-to-treat

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

Outcome measures

Measure	BMN 270 4E13 vg/kg n=1 Participants Single administration of valoctocogene roxaparvovec at a dose of 4E13 vg/kg
Change in the Annualized Number of Bleeding Episodes Requiring Exogenous FVIII Replacement Treatment	5.77 episodes/year Standard Deviation NA The number of participants is 1, so the Standard Deviation was not calculated

Adverse Events

BMN 270 4E13 vg/kg

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Total

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	BMN 270 4E13 vg/kg n=1 participants at risk Single administration of valoctocogene roxaparvovec at a dose of 4E13 vg/kg	Total n=1 participants at risk Total
General disorders Pyrexia	100.0% 1/1 • Number of events 1 • First 52 weeks post infusion	100.0% 1/1 • Number of events 1 • First 52 weeks post infusion
Hepatobiliary disorders Hepatocellular injury	100.0% 1/1 • Number of events 2 • First 52 weeks post infusion	100.0% 1/1 • Number of events 2 • First 52 weeks post infusion
Infections and infestations Nasopharyngitis	100.0% 1/1 • Number of events 4 • First 52 weeks post infusion	100.0% 1/1 • Number of events 4 • First 52 weeks post infusion
Investigations Alanine aminotransferase increased	100.0% 1/1 • Number of events 4 • First 52 weeks post infusion	100.0% 1/1 • Number of events 4 • First 52 weeks post infusion
Musculoskeletal and connective tissue disorders Myalgia	100.0% 1/1 • Number of events 1 • First 52 weeks post infusion	100.0% 1/1 • Number of events 1 • First 52 weeks post infusion
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	100.0% 1/1 • Number of events 1 • First 52 weeks post infusion	100.0% 1/1 • Number of events 1 • First 52 weeks post infusion

Additional Information

Medical Manager, Clinical Science

BioMarin Pharmaceutical Inc.

Phone: 415-996-2917

Email: divya.reddy@bmrn.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place