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Gene Therapy

Valoctocogene Roxaparvovec for Hemophilia A (GENEr8-2 Trial)

Phase 3

Waitlist Available

Research Sponsored by BioMarin Pharmaceutical

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up weeks 5 though week 52

Awards & highlights

GENEr8-2 Trial Summary

This trial will test whether BMN 270 is effective at raising FVIII activity and reducing the need for exogenous replacement therapy and the number of bleeding episodes.

GENEr8-2 Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ weeks 5 though week 52

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~weeks 5 though week 52

This trial's timeline: 3 weeks for screening, Varies for treatment, and weeks 5 though week 52 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Change of the Median Factor VIII (FVIII) Activity

Secondary outcome measures

Change in the Annualized Number of Bleeding Episodes Requiring Exogenous FVIII Replacement Treatment

Change in the Annualized Utilization (IU/kg) of Exogenous FVIII Replacement Therapy

Side effects data

From 2023 Phase 3 trial • 1 Patients • NCT03392974

100%

Alanine aminotransferase increased

100%

Oropharyngeal pain

100%

Nasopharyngitis

100%

Myalgia

100%

Hepatocellular injury

100%

Pyrexia

100%

80%

60%

40%

20%

Study treatment Arm

BMN 270 4E13 vg/kg

Total

GENEr8-2 Trial Design

1Treatment groups

Experimental Treatment

Group I: Valoctocogene Roxaparvovec Open LabelExperimental Treatment1 Intervention

Single administration of valoctocogene roxaparvovec at a dose of 4E13 vg/kg

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Valoctocogene roxaparvovec

Not yet FDA approved

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

BioMarin PharmaceuticalLead Sponsor

157 Previous Clinical Trials

190,010 Total Patients Enrolled

11 Trials studying Hemophilia A

1,219 Patients Enrolled for Hemophilia A

Medical Director, MDStudy DirectorBioMarin Pharmaceutical

77 Previous Clinical Trials

16,415 Total Patients Enrolled

3 Trials studying Hemophilia A

190 Patients Enrolled for Hemophilia A

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

How many individuals are trialing this medication?

"This study is no longer recruiting patients. The listing originally appeared on 3/14/2018 and was last updated on 8/2/2022. For those looking for other studies, 91 studies for patients with hemophilia a are actively enrolling participants and 6 studies for Valoctocogene Roxaparvovec are also currently enrolling."

Answered by AI

Is this study the first of its kind?

"Since 2015, there has been an increase in the amount of research dedicated to Valoctocogene Roxaparvovec. The first study was sponsored by BioMarin Pharmaceutical and was conducted in 2015 with a sample size of 15. After the success of the initial study, Phase 1 & 2 drug approval was received. There are currently 6 active studies being conducted in 13 different countries across 15 cities."

Answered by AI

Has Valoctocogene Roxaparvovec been cleared by the FDA?

"Valoctocogene Roxaparvovec has some efficacy data to support its safety, which is why it was given a 3."

Answered by AI

Might there be an opportunity for me to enroll in this research project?

"Unfortunately, this study is not recruiting patients at this time, as per the information available on clinicaltrials.gov. The trial was initially posted on March 14th, 2018, and was updated for the last time on August 2nd, 2022. There are, however, 97 other clinical trials actively recruiting patients."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

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

2018. "Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03392974.