CompletedPhase 3

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

United States67 participantsStarted 2018-06-27

Plain-language summary

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.

Who can participate

Age range18 Years

SexMALE

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Inclusion criteria

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

Exclusion criteria

✕. History of factor IX inhibitors
✕. Positive factor IX inhibitor test at screening
✕. Select screening laboratory value \>2 times upper limit of normal
✕. Positive human immunodeficiency virus (HIV) test at screening, not controlled with anti-viral therapy
✕. Active infection with hepatitis B or C virus at screening
✕. History of Hepatitis B or C exposure, currently controlled by antiviral therapy at the end of the lead-in phase
✕. Previous gene therapy treatment
✕. Receipt of an experimental agent within 60 days prior to screening

What they're measuring

Annualized Bleeding Rate (ABR) for All Bleeding Episodes

Timeframe: Lead-in period and months 7-18 post-treatment of AMT-061 (CSL222)

Trial details

NCT IDNCT03569891

SponsorCSL Behring

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2021-09-22

Results submitted2022-08-15

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