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Gene Therapy

Gene Therapy for Hemophilia B

Phase 3

Waitlist Available

Led By Steven Pipe, MD

Research Sponsored by UniQure Biopharma B.V.

Eligibility Criteria Checklist

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

Must have

Male

Age ≥18 years

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 5 years

Awards & highlights

Study Summary

This trial is being conducted to demonstrate the efficacy of the study drug and to further describe its safety profile. The trial will enroll participants at multiple centers across the globe. All participants will receive a single dose of the study drug. The trial will evaluate the safety and efficacy of the study drug over a period of 48 weeks.

Who is the study for?

This trial is for males over 18 with severe or moderately severe hemophilia B, who have had at least 150 days of prior treatment with factor IX protein. Participants must not have a history of gene therapy, active hepatitis B or C infections, uncontrolled HIV, or recent use of experimental drugs.Check my eligibility

What is being tested?

The HOPE-B trial tests AMT-061 (AAV5-hFIXco-Padua), a gene therapy aiming to treat hemophilia B. It's an open-label study where participants receive one dose intravenously to assess efficacy and safety.See study design

What are the potential side effects?

Potential side effects may include reactions related to the infusion process, liver enzyme elevations due to the viral vector used in the treatment, and immune responses against the introduced FIX protein.

Eligibility Criteria

Inclusion Criteria

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

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I am male.

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I am 18 years old or older.

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I have severe or moderately severe hemophilia B and am on factor IX treatment.

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I have been treated with factor IX for more than 150 days.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~5 years

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

Treatment Details

Study Objectives

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

Primary outcome measures

Bleeding time procedure

Secondary outcome measures

Hemorrhage

Adjusted Annualized Infusion Rate of FIX Replacement Therapy

Annualized Exogenous Factor IX Consumption

+12 more

Trial Design

2Treatment groups

Experimental Treatment

Active Control

Group I: AMT-061Experimental Treatment1 Intervention

Single infusion of AMT-061 Subjects will receive a single infusion of AAV5-hFIXco-Padua (AMT- 061) at baseline. After study drug administration (post study drug), subjects will be monitored for tolerance to the study drug and detection of potential immediate AEs at the clinical trial site for a few hours after dosing.

Group II: FIX replacement (Lead-in Period)Active Control1 Intervention

During the lead-in phase, which lasted for a minimum of 26 weeks (i.e., ≥6 months), subjects recorded their use of FIX replacement therapy and bleeding episodes in their dedicated e-diary.

0 / 4Eligibility criteria met

Find a Location

Who is running the clinical trial?

UniQure Biopharma B.V.Lead Sponsor

10 Previous Clinical Trials

207 Total Patients Enrolled

2 Trials studying Hemophilia B

13 Patients Enrolled for Hemophilia B

CSL BehringLead Sponsor

194 Previous Clinical Trials

1,211,155 Total Patients Enrolled

18 Trials studying Hemophilia B

4,254 Patients Enrolled for Hemophilia B

Steven Pipe, MDPrincipal InvestigatorUniversity of Michigan

1 Previous Clinical Trials

3 Total Patients Enrolled

1 Trials studying Hemophilia B

3 Patients Enrolled for Hemophilia B

Media Library

AAV5-hFIXco-Padua (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT03569891 — Phase 3

Hemophilia B Research Study Groups: AMT-061, FIX replacement (Lead-in Period)

Hemophilia B Clinical Trial 2023: AAV5-hFIXco-Padua Highlights & Side Effects. Trial Name: NCT03569891 — Phase 3

AAV5-hFIXco-Padua (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03569891 — Phase 3

Frequently Asked Questions

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

Are there many hospitals or research centers participating in this trial throughout North America?

"In addition to the 22 primary clinical trial sites, there are numerous other locations across the United States of America where this study is taking place."

Answered by AI

Are volunteers still being accepted for this research project?

"This specific clinical trial, as indicated on clinicaltrials.gov, is not enrolling patients at this moment in time. The trial was first advertised on June 27th, 2018 and was last updated September 26th, 20202. However, there are many other medical trials (91 to be exact) that are still recruiting patients."

Answered by AI