Hemophilia B

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

NCT02396342 | PHASE 1 | INTERVENTIONAL

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.

7 Sites

10 Participants

Recruiting

18 Years to 11 Years

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Find closest sites:

SUBMIT

uniQure Investigative Site

Copenhagen,Denmark

uniQure Investigative Site

Berlin,Germany

uniQure Investigative Site

Frankfurt,Germany

uniQure Investigative Site

Amsterdam,Netherlands

uniQure Investigative Site

Groningen,Netherlands

uniQure Investigative Site

Rotterdam,Netherlands

uniQure Investigative Site

Utrecht,Netherlands

Study Eligibility Criteria

Inclusion Criteria

1. Male
2. Age ≥ 18 years
3. Patients with congenital hemophilia B classified as one of the following:
* Known severe FIX deficiency with plasma FIX activity level \< 1% and a severe bleeding phenotype defined by one of the following:
* Currently on prophylactic FIX replacement therapy for a history of bleeding
* Currently on on-demand therapy with a current or past history of frequent bleeding defined as four or more bleeding episodes in the last 12 months or chronic hemophilic arthropathy (pain, joint destruction, and loss of range of motion) in one or more joints
* Known moderately severe FIX deficiency with plasma FIX activity level between ≥ 1% and ≤ 2% and a severe bleeding phenotype defined by one of the following:
* Currently on prophylactic FIX replacement therapy for a history of bleeding
* Currently on on-demand therapy with a current or past history of frequent bleeding defined as four or more bleeding episodes in the last 12 months or chronic hemophilic arthropathy (pain, joint destruction, and loss of range of motion) in one or more joints
4. More than 150 previous exposure days of treatment with FIX protein.
5. 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)
6. 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

1. History of FIX inhibitors measured to be ≥ 0.6 Bethesda Units (BU)/mL
2. FIX inhibitors ≥ 0.6 BU/mL at Visit 1 (measured by the local laboratory)
3. Neutralizing antibodies against AAV5 at Visit 1 (measured by the central laboratory)
4. Visit 1 laboratory values (measured by the central laboratory):
* alanine aminotransferase \> 2 times upper normal limit
* aspartate aminotransferase \> 2 times upper normal limit
* total bilirubin \> 2 times upper normal limit
* alkaline phosphatase \> 2 times upper normal limit
* creatinine \> 1.5 times upper normal limit
5. 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)
6. 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).
7. History of Hepatitis B or C exposure, currently controlled by antiviral therapy
8. Any coagulation disorder other than hemophilia B
9. Thrombocytopenia, defined as a platelet count below 50 × 10E9 / L, at Visit 1 (measured by the central laboratory)
10. Body mass index \< 16 or ≥ 35 kg/m2
11. Planned surgery for the initial 6 months after IMP administration in this trial
12. Previous arterial or venous thrombotic event (e.g. acute myocardial infarction, cerebrovascular disease and venous thrombosis)
13. Active severe infection or any other significant concurrent, uncontrolled medical condition including, but not limited to, renal, hepatic, haematological, gastrointestinal, endocrine, pulmonary, neurological, cerebral or psychiatric disease, alcoholism, drug dependency or any other psychological disorder evaluated by the investigator to interfere with adherence to the protocol procedures or with the degree of tolerance to the IMP
14. Known significant medical condition including disseminated intravascular coagulation, fibrinolysis and liver fibrosis which, in the opinion of the investigator, may confo

Additional Studies

Additional studies can be found at ClinicalTrials.gov