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This study investigates the safety and effectiveness of a gene therapy (REGV131-LNP1265) that inserts clotting Factor IX in patients with hemophilia B. It enrolls children, adolescents, and adults with severe or moderately severe hemophilia B who are currently on FIX prophylaxis. The study is in Phase 1/Phase 2 and is currently recruiting participants.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
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Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Key Inclusion Criteria: 1. Confirmed diagnosis of severe or moderately severe hemophilia B with medical history of FIX functional activity (≤2% or \<0.02 IU/mL) or documented genotype known to produce severe hemophilia B 2. Currently taking FIX prophylaxis and previous experience with FIX therapy, as defined in the protocol 3. Participation in the lead-in period of this interventional study OR a separate lead-in study (R0000-HEMB-2187 \[NCT05568459\]) for at least 6 months for ABR data while taking FIX prophylaxis, as defined in the protocol Key Exclusion Criteria: 1. History of FIX inhibitor (clinical or laboratory-based assessment) on 2 or more occasions 2. Bethesda inhibitor titer greater than the Upper Limit of Normal (ULN) at screening 3. Detectable pre-existing antibodies to the AAV8 capsid; as measured by Enzyme-Linked ImmunoSorbent Assay (ELISA) at prescreening (or final lead-in visit, if applicable) 4. Any significant underlying liver disease such as: cholestatic liver disease, liver cirrhosis, portal hypertension, splenomegaly, hepatic encephalopathy 5. Evidence of advanced liver fibrosis or significant fatty liver, as defined in the protocol 6. Evidence of cirrhosis and/or portal hypertension as assessed by abdominal ultrasound at screening or measured within 6 months prior to the screening visit 7. History of arterial or venous thrombo-embolic events, as defined in the protocol 8. History of hypersensitivity to corticosteroids or known medical condition that requires chronic administration of corticosteroids 9. Previously received any AAV gene-based therapy or intends to receive approved or investigational AAV-based gene therapy other than REGV131-LNP1265 during the study period NOTE: Other Inclusion/Exclusion Protocol Defined Criteria Apply