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Clinical Trial Tool

BioMarin has an ongoing commitment to the hemophilia community, which includes our growing understanding of valoctocogene roxaparvovec (BMN 270), through ongoing registries and research.

Please click on the trial numbers to find out more information, where available.

201

Enrollment completed

Phase 1/2 Dose Escalation Study

  • 15 patients (AAV5-), 4 dose cohorts; UK

701/702

Enrollment ongoing (701)

Seroprevalence/Seroconversion Studies

  • 701: Approximately 350 patients across 4 US geographic locations
  • 702: Population size TBD. KSA and others (TBD)

901

Enrollment completed

Global Seroprevalence Study

  • N = 546; US, UK, France, Russia, Germany, Italy, Japan, S. Africa, Brazil

902

Enrollment completed

Non-Interventional Study

  • N = 294; AAV5-/HIV- patients, 13 Countries

GENEr8-1a

Enrollment completed

Phase 3 Study with 6 x 1013vg/kg dose

  • N = 136 patients, 13 Countries

GENEr8-2a

Enrollment stopped

Phase 3 Study with 4 x 1013vg/kg dose

  • N = 1 patient, US

203

Enrollment ongoing

AAV5+ Phase 1/2 Titer Escalation Study with 6 x 1013vg/kg dose

  • 10 patients; 2 titer cohorts (≤500, >500); UK, S. Africa, US, Taiwan, S. Korea

303

Enrollment completed

6 x 1013vg/kg dose with Prophylactic Corticosteroids Study

  • N = 22; Australia, Brazil, US, Taiwan

205

Enrollment ongoing

Active or Prior Inhibitor Phase 1/2 Study with 6 x 1013vg/kg dose

  • 20 patients; 2 parts (active and prior inhibitors); UK, US; 5 other countries pending

401

In strart-up

Long-term follow up for patients who received valoctocogene roxaparvovec

  • ~200 patients, 13 countries

aBioMarin announces its decision to cease development of the 4 x 1013 dose (GENEr8-2) in August 2019

KSA, Kingdom of Saudi Arabia; AAV, adeno-associated virus