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HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients

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CSL Behring

Status and phase

Active, not recruiting
Phase 3

Conditions

Hemophilia B

Treatments

Biological: Factor IX (FIX)
Genetic: AAV5-hFIXco-Padua

Study type

Interventional

Funder types

Industry

Identifiers

NCT03569891
CSL222_3001 (CT-AMT-061-02)

Details and patient eligibility

About

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.

Enrollment

67 patients

Sex

Male

Ages

18+ years old

Volunteers

No Healthy Volunteers

Inclusion criteria

  1. Male
  2. Age ≥18 years
  3. Subjects with congenital hemophilia B, classified as severe or moderately severe, and are currently on factor IX prophylaxis
  4. >150 previous exposure days of treatment with factor IX protein

Exclusion criteria

  1. History of factor IX inhibitors
  2. Positive factor IX inhibitor test at screening
  3. Select screening laboratory value >2 times upper limit of normal
  4. Positive human immunodeficiency virus (HIV) test at screening, not controlled with anti-viral therapy
  5. Active infection with hepatitis B or C virus at screening
  6. History of Hepatitis B or C exposure, currently controlled by antiviral therapy at the end of the lead-in phase
  7. Previous gene therapy treatment
  8. Receipt of an experimental agent within 60 days prior to screening
  9. Current participation or anticipated participation within one year after study drug administration in this trial in any other interventional clinical trial involving drugs or devices

Trial design

Primary purpose

Treatment

Allocation

Non-Randomized

Interventional model

Sequential Assignment

Masking

None (Open label)

67 participants in 2 patient groups

AMT-061
Experimental group
Description:
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.
Treatment:
Genetic: AAV5-hFIXco-Padua
FIX replacement (Lead-in Period)
Active Comparator group
Description:
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.
Treatment:
Biological: Factor IX (FIX)

Trial documents
2

Trial contacts and locations

33

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Data sourced from clinicaltrials.gov

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