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Gene Therapy

verbrinacogene setparvovec for Hemophilia B

Phase 1 & 2

Waitlist Available

Research Sponsored by Freeline Therapeutics

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 pre-dose and weeks 26, 52 post-dose

Awards & highlights

Study Summary

This trial is to study a new gene therapy for Hemophilia B in adults. Up to 9 patients will be enrolled to receive a single dose of the therapy and be followed for 52 weeks. Results will help to confirm the dose for a future Phase 3 study.

Eligible Conditions

Hemophilia B

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ pre-dose and weeks 26, 52 post-dose

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~pre-dose and weeks 26, 52 post-dose

This trial's timeline: 3 weeks for screening, Varies for treatment, and pre-dose and weeks 26, 52 post-dose for reporting.

Treatment Details

Study Objectives

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

Primary outcome measures

Assessment of FIX activity levels to allow dose confirmation for future Phase 3 study

Safety and tolerability of FLT180a as assessed by incidence and severity of AEs and SAEs

Secondary outcome measures

Abnormal or change from baseline findings for liver ultrasound

Abnormal or change from baseline findings for serum alpha-fetoprotein (AFP) levels.

Assessment of Clinically significant changes in 12-lead ECG

+15 more

Trial Design

1Treatment groups

Experimental Treatment

Group I: FLT180aExperimental Treatment1 Intervention

A single dose of FLT180a will be administered. Dose will be determined by enrollment cohort. The first 3 patients will receive 7.7 x 10e11 vg/kg. The dose in subsequent cohorts will be determined by the DMC based on review of data from the prior cohort(s).

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Find a Location

Who is running the clinical trial?

Freeline TherapeuticsLead Sponsor

5 Previous Clinical Trials

71 Total Patients Enrolled

2 Trials studying Hemophilia B

47 Patients Enrolled for Hemophilia B

Frequently Asked Questions

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

How can I sign up for this opportunity to test a new medication?

"The prerequisites for enrolment in this study are hemophilia b and falling within the age bracket of 18 to 65 years old. So far, 9 individuals have been accepted into the trial."

Answered by AI

Does this particular clinical trial have an age limit?

"The age limit for patients that wish to participate in this trial is 65 years old. Those under 18 and over 66 do not meet the requirements set by the study's inclusion criteria. There are currently 41 clinical trials underway with a similar age restriction and 66 trials for patients outside of this age bracket."

Answered by AI

Is this study still enrolling patients who wish to participate?

"This study, which was first posted on December 6th 2021, is not looking for patients at this time. The last update to the posting was on October 3rd 2022. There are 91 other clinical trials with open recruitment at this time."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Phase 1/2 Dose Confirmation Study of FLT180a in Hemophilia B

2021. "Phase 1/2 Dose Confirmation Study of FLT180a in Hemophilia B". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05164471.