Gene Therapy for Hemophilia B

Recruiting at 41 trial locations

Age: 18+

Sex: Male

Trial Phase: Phase 3

Sponsor: UniQure Biopharma B.V.

Must be taking: Factor IX prophylaxis

Must not be taking: Experimental agents

Disqualifiers: HIV, Hepatitis B/C, Gene therapy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 1

Duration: Single dose

67 Participants Needed

Trial Summary

What is the purpose of this trial?

This is an open-label, single-dose, multi-center, multinational trial to demonstrate the efficacy of AMT-061 and to further describe its safety profile. The study drug is identified as AAV5-hFIXco-Padua (AMT- 061). AMT-061 is a recombinant adeno-associated viral vector of serotype 5 (AAV5) containing the Padua variant of a codon-optimized human FIX complementary deoxyribonucleic acid (cDNA) under the control of a liver-specific promoter. The pharmaceutical form of AMT-061 is a solution for intravenous infusion administered at a dose of 2 x 10\^13 gc/kg.

Research Team

Steven Pipe, MD

Principal Investigator

University of Michigan

Eligibility Criteria

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.

Inclusion Criteria

I am male.

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

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

Exclusion Criteria

I have had gene therapy before.

Receipt of an experimental agent within 60 days prior to screening

I have Hepatitis B or C but it's under control with antiviral therapy.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Lead-in Period

Subjects recorded their use of FIX replacement therapy and bleeding episodes in their dedicated e-diary

26 weeks

Treatment

Single infusion of AAV5-hFIXco-Padua (AMT-061) administered at baseline

Single dose

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

18 months

Long-term Follow-up

Follow up and assess any adverse events reported for safety

5 years

Treatment Details

Interventions

• AAV5-hFIXco-Padua
• Factor IX (FIX)

Trial Overview 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.

Participant Groups

Group I: AMT-061 Experimental Treatment 1 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 Control 1 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.

Who Is Running the Clinical Trial?

UniQure Biopharma B.V.

Lead Sponsor

Trials

12

Recruited

260+

CSL Behring

Lead Sponsor

Trials

204

Recruited

1,207,000+

Dr. Paul McKenzie

CSL Behring

Chief Executive Officer since 2023

PhD in Chemical Engineering from Carnegie Mellon University, B.S. in Chemical Engineering from the University of Pennsylvania

Dr. Bill Mezzanotte

CSL Behring

Chief Medical Officer since 2021

MD from Duke University