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Gene Therapy

Factor IX Gene Therapy for Hemophilia B (BENEGENE-2 Trial)

Q: Are we still enrolling patients for this research project?

The clinical trial is no longer recruiting patients as of the last update on October 5th, 2022. This was determined by information found on clinicaltrials.gov. The study initially posted on July 29th, 2019 and had a total of 91 participants enrolled at its conclusion.

Q: Who does this clinical trial hope to include in its research?

Those with hemophilia b and between the ages of 18-65 may be eligible for this study. Currently, around 45 people have been accepted into the trial.

Q: Does this research involve elderly citizens?

The ideal candidate for this study is someone who is younger than 65 years old and has already reached the age of majority.

Q: Do patients report any adverse effects from PF-06838435/ fidanacogene elaparvovec?

PF-06838435/ fidanacogene elaparvovec is a Phase 3 clinical trial medication, meaning that there is efficacy data as well as multiple rounds of safety data. Therefore, our team Power has given it a score of 3 for safety.

Q: In how many different geographic areas is this study being done?

This study is still looking for participants and has 16 enrolment sites. If you are interested in taking part in this trial, consider selecting a site near to your location to reduce the amount of travel required. Jackson, Aurora and Philadelphia are a few of the locations with open enrolment.

Phase 3

Waitlist Available

Research Sponsored by Pfizer

Eligibility Criteria Checklist

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

Must have

Documented moderately severe to severe hemophilia B (Factor IX activity <=2%)

Suspension of prophylaxis therapy for hemophilia B after administration of the study drug

Timeline

Screening 3 weeks

Treatment Varies

Follow Up maximum up to 6 years (week 312) after pf-06838435 infusion

Awards & highlights

BENEGENE-2 Trial Summary

This trial will test if a gene therapy drug is effective and safe in adult men with hemophilia B. The therapy is designed to replace Factor IX, which is missing or non-functioning in these patients. Those who participate will be given the therapy once and monitored for six years. The objective is to compare the annualized bleeding rate for those treated with gene therapy against the standard of care therapy.

Who is the study for?

Adult males with moderately severe to severe hemophilia B (Factor IX activity <=2%) who have completed at least 6 months of Factor IX prophylaxis therapy. Participants must not have antibodies against the gene therapy vector, no history of Factor IX inhibitors, and agree to contraception post-treatment.Check my eligibility

What is being tested?

The trial is testing PF-06838435, a gene therapy intended to correct the genetic defect in hemophilia B by providing a functional copy of the Factor IX gene. Patients will receive one intravenous dose and be monitored over six years against standard prophylactic treatment.See study design

What are the potential side effects?

Potential side effects may include immune reactions to the viral vector used for gene delivery, liver enzyme elevations indicating liver inflammation or damage, development of inhibitors (antibodies) that can neutralize Factor IX's effect, and general symptoms like fatigue or headaches.

BENEGENE-2 Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

My hemophilia B is severe with Factor IX activity at 2% or less.

Select...

I will stop my preventive hemophilia B treatment after starting the study drug.

BENEGENE-2 Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ maximum up to 6 years (week 312) after pf-06838435 infusion

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~maximum up to 6 years (week 312) after pf-06838435 infusion

This trial's timeline: 3 weeks for screening, Varies for treatment, and maximum up to 6 years (week 312) after pf-06838435 infusion for reporting.

Treatment Details

Study Objectives

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

Primary outcome measures

Annualized Bleeding Rate (ABR) for Total Bleeds (Treated and Untreated) From Week 12 to Month 15

Secondary outcome measures

ABR for Spontaneous Bleeds From Week 12 to Month 15

ABR for Spontaneous and Traumatic, and Untreated Bleeds Through the Study

ABR for Total Bleeds (Treated and Untreated) Through the Study

+23 more

BENEGENE-2 Trial Design

1Treatment groups

Experimental Treatment

Group I: PF-06838435/ fidanacogene elaparvovecExperimental Treatment1 Intervention

0 / 2Eligibility criteria met

Find a Location

Who is running the clinical trial?

PfizerLead Sponsor

4,567 Previous Clinical Trials

10,907,999 Total Patients Enrolled

20 Trials studying Hemophilia B

4,917 Patients Enrolled for Hemophilia B

Pfizer CT.gov Call CenterStudy DirectorPfizer

3,474 Previous Clinical Trials

8,089,167 Total Patients Enrolled

21 Trials studying Hemophilia B

1,943 Patients Enrolled for Hemophilia B

Media Library

PF-06838435 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT03861273 — Phase 3

PF-06838435 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03861273 — Phase 3

Frequently Asked Questions

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

Are we still enrolling patients for this research project?

"The clinical trial is no longer recruiting patients as of the last update on October 5th, 2022. This was determined by information found on clinicaltrials.gov. The study initially posted on July 29th, 2019 and had a total of 91 participants enrolled at its conclusion."

Answered by AI

Who does this clinical trial hope to include in its research?

"Those with hemophilia b and between the ages of 18-65 may be eligible for this study. Currently, around 45 people have been accepted into the trial."

Answered by AI

Does this research involve elderly citizens?

"The ideal candidate for this study is someone who is younger than 65 years old and has already reached the age of majority."

Answered by AI

Do patients report any adverse effects from PF-06838435/ fidanacogene elaparvovec?

"PF-06838435/ fidanacogene elaparvovec is a Phase 3 clinical trial medication, meaning that there is efficacy data as well as multiple rounds of safety data. Therefore, our team Power has given it a score of 3 for safety."

Answered by AI

In how many different geographic areas is this study being done?

"This study is still looking for participants and has 16 enrolment sites. If you are interested in taking part in this trial, consider selecting a site near to your location to reduce the amount of travel required. Jackson, Aurora and Philadelphia are a few of the locations with open enrolment."

Answered by AI

Who else is applying?

What site did they apply to?

Center for Human Phenomic Science CHPS

What portion of applicants met pre-screening criteria?

Did not meet criteria

Why did patients apply to this trial?

My Son Jeremy is pursuing a Degree in Medicine which is a physically intensive course, the trial will help him reduce the spontaneous bleeding of his target joint from long hours of hospital rounds.

PatientReceived 1 prior treatment

Recent research and studies

clinicaltrials.govStudy

A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B

2019. "A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03861273.

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