A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B

Launched by REGENERON PHARMACEUTICALS · Apr 18, 2024

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called REGV131-LNP1265 for people with Hemophilia B, a genetic condition that prevents blood from clotting properly. The goal is to see if this treatment is safe and effective in helping the body produce a necessary protein called Factor 9 that helps with blood clotting. If successful, this therapy could reduce the need for regular treatment with Factor replacement therapy, which is often needed to prevent bleeding episodes. The trial will also look at how this treatment affects participants' quality of life, joint health, and the frequency of bleeding events.

To participate, individuals must be diagnosed with severe or moderately severe Hemophilia B and have been receiving a specific type of treatment called FIX prophylaxis for at least six months. The study is open to males aged 18 and older. Participants can expect to receive the study drug and will be monitored for any side effects, how their body responds to the treatment, and how their overall health is impacted. It's important to note that people with certain conditions, like liver disease or a history of specific blood-related issues, may not be eligible to join the trial.

Gender

MALE

Eligibility criteria

• 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 adeno-associated virus serotype 8 (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, 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