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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

Must not have

Previous gene therapy treatment

Positive human immunodeficiency virus (HIV) test at screening, not controlled with anti-viral therapy

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 5 years

Awards & highlights

No Placebo-Only Group

Pivotal Trial

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.

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.

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.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have had gene therapy before.

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I have HIV that is not controlled by medication.

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

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Bleeding time procedure

Secondary study objectives

EuroQol-5 Dimensions-5 Levels (EQ-5D-5L) VAS Overall Score

International Physical Activity Questionnaire (iPAQ) Overall Score

Number of Adverse Events

+1 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

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 / 6Eligibility criteria met