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The goal of this multicenter, two-stage, open-label Phase 1/2 study is to investigate the safety, immunogenicity and efficacy of ANB-010 in people with hemophilia A. Three dose levels are tested in a dose-escalation design with elements of a Phase 1/2 seamless adaptive design. Eligible participants are males aged 18 or older at consent with an established diagnosis of hemophilia A, documented endogenous FVIII activity of 1% or less and 2% or less at screening, and at least 150 exposure days of therapy with FVIII concentrates.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: 1\. Male subjects aged ≥18 years at the time of signing the informed consent form. 3\. Established diagnosis of hemophilia A with a documented history of endogenous FVIII activity ≤1% AND ≤2% at screening. 4\. Therapy with FVIII concentrates for at least 150 exposure days. Exclusion Criteria: 1. History of use of any gene therapy product. 2. Use of emicizumab within less than 6 months before the date of signing the ICF. 3. The presence of other blood or hematopoietic disorders other than hemophilia A. 4. Presence of AAV6 antibodies detected by ELISA. 5. BMI \<16 kg/m² or ≥35 kg/m². 6. Diagnosis of HIV infection. 7. HBV infection. 8. HCV infection. 9. Any active systemic infections or recurrent infections requiring systemic therapy at screening. 10. Any other disorders associated with severe immunodeficiency. 11. Relevant hepatic disorders or conditions that can be a symptom of existing liver disorder. 12. Malignancies with remission duration of less than 5 years at the time of signing the ICF, except for cured basal cell carcinoma.