CompletedPhase 1/2

Trial of AAV5-hFIX in Severe or Moderately Severe Hemophilia B

Denmark, Germany, Netherlands10 participantsStarted 2015-06-10

Plain-language summary

This study evaluates how safe gene therapy treatment with AAV5-hFIX is in adult patients with severe or moderately severe hemophilia B and severe bleeding type.

Who can participate

Age range18 Years

SexMALE

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Inclusion criteria

✓. Male
✓. Age ≥ 18 years
✓. Patients with congenital hemophilia B classified as one of the following:
✓. More than 150 previous exposure days of treatment with FIX protein.
✓. Acceptance to use a condom during sexual intercourse in the period from Investigational Medicinal Product (IMP) administration until AAV5 has been cleared from semen, as evidenced by the central laboratory from negative analysis results for at least 3 consecutively collected semen samples (this criterion is applicable also for subjects who are surgically sterilized)
✓. Following receipt of verbal and written information about the trial, the subject has provided signed informed consent before any trial related activity is carried out.

Exclusion criteria

✕. History of FIX inhibitors measured to be ≥ 0.6 Bethesda Units (BU)/mL
✕. FIX inhibitors ≥ 0.6 BU/mL at Visit 1 (measured by the local laboratory)
✕. Neutralizing antibodies against AAV5 at Visit 1 (measured by the central laboratory)
✕. Visit 1 laboratory values (measured by the central laboratory):
✕. Positive HIV serological test at Visit 1, not controlled with anti-viral therapy as shown by cluster of differentiation 4+ counts ≤ 200 per μL or by a viral load of \>200 copies per mL (measured by the central laboratory)
✕. Active infection with Hepatitis B or C virus as reflected by Hepatitis B Surface Antigen (HBsAg), Hepatitis B extracellular Antigen (HBeAg), Hepatitis B Virus DeoxyriboNucleic Acid (HBV DNA) or Hepatitis C Virus RiboNucleic Acid (HCV RNA) positivity, respectively, at Visit 1 (measured by the central laboratory).
✕. History of Hepatitis B or C exposure, currently controlled by antiviral therapy
✕. Any coagulation disorder other than hemophilia B

What they're measuring

Number of Participants With Adverse Events

Timeframe: From AMT-060 infusion through end of study (5 years post-dose)

Trial details

NCT IDNCT02396342

SponsorCSL Behring

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2021-04-15

Results submitted2022-06-01

View on ClinicalTrials.gov More Hemophilia B trials