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RECRUITINGPhase 1 / Phase 2INTERVENTIONAL

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

A Two-Part Open-Label Study of REGV131-LNP1265, A CRISPR/Cas9 Based Coagulation Factor IX Gene Insertion Therapy in Participants With Hemophilia B

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

Participants in this study have a genetic mutation, specifically in the coagulation (blood clotting) Factor 9 gene that causes severe or moderately severe hemophilia B. This study is researching an experimental gene insertion therapy (the adding of a gene into your DNA) called REGV131-LNP1265, also called the "study drug". Gene insertion therapy aims to teach the body how to produce clotting factor long-term, without the need for factor replacement therapy. The main aim of this study is to find a safe and well-tolerated dose of the study drug by checking the side effects that may happen from taking it. The study is looking at several other research questions including: * How much study drug is in the blood at different times * Whether the body makes antibodies against parts of the study drug, which could make the drug less effective or could lead to side effects. Antibodies are proteins produced by the body's immune system in response to a foreign substance * Whether the body makes antibodies against the clotting factor replacement therapy * How quality of life is affected by hemophilia B and if it changes after taking study drug * How joint health is affected by hemophilia B and if it changes after taking study drug * How often visits are required for the emergency room, urgent care center, physician's office, hospital, telephone or online are required as a result of bleeding events, and if the frequency changes after taking study drug * How often factor replacement therapy is needed, both on a regular basis for prevention of bleeding, and as needed to treat bleeding events (and it if changes after taking study drug) * Whether there is a difference in 2 different methods for measuring Factor 9 activity in the blood

Who May Be Eligible (Plain English)

Key Who May Qualify: 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 Who Should NOT Join This Trial: 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 Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
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

Treatments Being Tested

DRUG

REGV131

Single dose administered via intravenous (IV) infusion before LNP1265

DRUG

LNP1265

Single dose administered via IV infusion following REGV131

Locations (20)

Orthopaedic Hemophilia Treatment Center
Los Angeles, California, United States
David Geffen School of Medicine at UCLA
Los Angeles, California, United States
Children's Hospital Los Angeles
Los Angeles, California, United States
University of California Davis
Sacramento, California, United States
University California San Francisco
San Francisco, California, United States
University of Colorado Hemophilia and Thrombosis Center
Aurora, Colorado, United States
Yale HTC
New Haven, Connecticut, United States
University of Florida
Gainesville, Florida, United States
Indiana Hemophilia and Thrombosis Center
Indianapolis, Indiana, United States
Tulane University School of Medicine, Louisiana Center for Bleeding and Clotting Disorders
New Orleans, Louisiana, United States
University of Michigan
Ann Arbor, Michigan, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
Children's Hospital of Philadelphia (CHOP)
Philadelphia, Pennsylvania, United States
Royal Prince Alfred Hospital, Haemophilia Treatment Centre
Camperdown, New South Wales, Australia
University of Alberta Hospital
Edmonton, Alberta, Canada
McMaster University Medical Centre - Hamilton Health Sciences
Hamilton, Ontario, Canada
McGill University Health Center (MUHC)
Montreal, Quebec, Canada
Hospices Civils de Lyon
Bron, Lyon, France
Hemostase Clinique, Institut Coeur Poumon
Lille, Nord, France
Hopital Necker
Paris, Île-de-France Region, France