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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
130 patients around the world
Available in Brazil, United States
The study will be conducted with a 2-part adaptive design, with enrollment of patients into sequential parts of the study.
Part 1: Dose Escalation and Dose Confirmation in adult patients ≥18 years of age
- Dose Escalation Cohorts to determine the Recommended Dose for Expansion (RDE) of REGV131-LNP1265
- Dose Confirmation Cohort to gain further confidence in safety, tolerability, and Coagulation Factor IX (FIX) functional activity data at the RDE
Part 2: Dose Expansion at the RDE
- Part 2A: Adult patients ≥18 years of age: RDE of REGV131-LNP1265, as determined in Part 1
- Part 2B: Adolescent patients ≥12 to <18 years of age will be administered weight-adjusted RDE
- Part 2C: Adolescent and Pediatric patients ≥2 to <12 years may be enrolled in an age staggered sequential manner; first participants aged ≥6 to <12 years and then participants ≥2 to <6 years of age and will receive a weight-adjusted RDE
Regeneron Pharmaceuticals
2Research sites
130Patients around the world
This study is for people with
Hemophilia
Hemophilia b
Requirements for the patient
From 2 Years
Male
Medical requirements
- 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.Currently taking FIX prophylaxis and previous experience with FIX therapy, as defined in the protocol.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.
- History of FIX inhibitor (clinical or laboratory-based assessment) on 2 or more occasions.Bethesda inhibitor titer greater than the Upper Limit of Normal (ULN) at screening.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).Any significant underlying liver disease such as: cholestatic liver disease, liver cirrhosis, portal hypertension, splenomegaly, hepatic encephalopathy.Evidence of advanced liver fibrosis or significant fatty liver, as defined in the protocol.Evidence of cirrhosis and/or portal hypertension as assessed by abdominal ultrasound at screening or measured within 6 months prior to the screening visit.History of arterial or venous thrombo-embolic events, as defined in the protocol.History of hypersensitivity to corticosteroids or known medical condition that requires chronic administration of corticosteroids.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.
Sites
HEMORIO Instituto Nacional de Hematología
Recruiting
Hospital de Clínicas - Universidade Estadual de Campinas - UNICAMP
Recruiting
StudyBEYOND-9
SponsorRegeneron Pharmaceuticals
Study typeInterventional
Conditions
Hemophilia b
Requirements
From 2 YearsMale
Unique study IDclinicaltrials.gov
NCT06379789
