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
NCT ID: NCT06379789
Last Updated: 2026-01-21
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE1/PHASE2
130 participants
INTERVENTIONAL
2024-09-11
2032-12-17
Brief Summary
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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
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Detailed Description
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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 \<18 and ≥12 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
Conditions
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Study Design
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NON_RANDOMIZED
SEQUENTIAL
TREATMENT
NONE
Study Groups
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Part 1: Cohort 1 Dose Escalation for RDE
Starting dose to determine the RDE of REGV131-LNP1265 and further assess the safety, tolerability, and FIX functional activity data at the RDE
REGV131
Single dose administered via intravenous (IV) infusion before LNP1265
LNP1265
Single dose administered via IV infusion following REGV131
Part 1: Cohort 2 Dose Escalation for RDE
Dose 2 of ascending dose level cohorts to determine the RDE of REGV131-LNP1265 and further assess the safety, tolerability, and FIX functional activity data at the RDE
REGV131
Single dose administered via intravenous (IV) infusion before LNP1265
LNP1265
Single dose administered via IV infusion following REGV131
Part 1: Cohort 3 Dose Escalation for RDE
Dose 3 of ascending dose level cohorts to determine the RDE of REGV131-LNP1265 and further assess the safety, tolerability, and FIX functional activity data at the RDE
REGV131
Single dose administered via intravenous (IV) infusion before LNP1265
LNP1265
Single dose administered via IV infusion following REGV131
Part 1: Cohort 4 Dose Escalation for RDE
Dose 4 of ascending dose level cohorts to determine the RDE of REGV131-LNP1265 and further assess the safety, tolerability, and FIX functional activity data at the RDE
REGV131
Single dose administered via intravenous (IV) infusion before LNP1265
LNP1265
Single dose administered via IV infusion following REGV131
Part 2: Dose Expansion A
Participants ≥18 Years of Age will receive the RDE of REGV131-LNP1265 determined by Part 1
REGV131
Single dose administered via intravenous (IV) infusion before LNP1265
LNP1265
Single dose administered via IV infusion following REGV131
Part 2: Dose Expansion B
Participants ≥12 to \<18 Years of Age will receive the administered weight-adjusted RDE of REGV131-LNP1265 determined by Part 1
REGV131
Single dose administered via intravenous (IV) infusion before LNP1265
LNP1265
Single dose administered via IV infusion following REGV131
Part 2: Dose Expansion C
Participants ≥2 to \<12 Years of Age will receive the administered weight-adjusted RDE of REGV131-LNP1265 determined by Part 1
REGV131
Single dose administered via intravenous (IV) infusion before LNP1265
LNP1265
Single dose administered via IV infusion following REGV131
Interventions
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REGV131
Single dose administered via intravenous (IV) infusion before LNP1265
LNP1265
Single dose administered via IV infusion following REGV131
Eligibility Criteria
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Inclusion Criteria
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
Exclusion Criteria
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
18 Years
MALE
No
Sponsors
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Intellia Therapeutics
INDUSTRY
Regeneron Pharmaceuticals
INDUSTRY
Responsible Party
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Principal Investigators
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Clinical Trial Management
Role: STUDY_DIRECTOR
Regeneron Pharmaceuticals
Locations
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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
Hopital Necker
Paris, Île-de-France Region, France
University Hospital Frankfurt
Frankfurt am Main, Hesse, Germany
University Hospital Hamburg Eppendorf
Hamburg, , Germany
Hospital Universitario Virgen del Rocio
Seville, Andalusia, Spain
Complejo Hospitalario Universitario de A Coruña (Edificio Teresa Herrera-Materno Infantil)
A Coruña, Galicia, Spain
Hospital Clinico Universitario Virgen De La Arrixaca
El Palmar, Murcia, Spain
Hospital Universitario Central de Asturias
Oviedo, Principality of Asturias, Spain
Hospital Universitario Vall d'Hebron
Barcelona, , Spain
Hospital Universitario La Paz
Madrid, , Spain
Haemostasis and Thrombosis Unit, Hospital La Fe
Valencia, , Spain
Hospital Universitario Miguel Servet
Zaragoza, , Spain
Glasgow Royal Infirmary - Clinical Research Facility
Glasgow, Scotland, United Kingdom
Queen Elizabeth Hospital Birmingham
Birmingham, West Midlands, United Kingdom
Addenbrooke's Hospital, Cambridge University Hospitals NHS FT
Cambridge, , United Kingdom
Pathology and Pharmacy Building, The Royal London Hospital
London, , United Kingdom
Royal Free London NHS Foundation Trust
London, , United Kingdom
St. Thomas' Hospital
London, , United Kingdom
Hammersmith Hospital Comprehensive Care Centre
London, , United Kingdom
Countries
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Central Contacts
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Related Links
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Study Information Website
Other Identifiers
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2023-507260-40-00
Identifier Type: CTIS
Identifier Source: secondary_id
R131L1265-HEMB-2318
Identifier Type: -
Identifier Source: org_study_id
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