Hemophilia B study: REGV131 vs LNP1265
Official title 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
ClinicalTrials.gov ID: NCT06379789
What this study is testing
What is REGV131?
an investigational gene insertion therapy being studied as a potential treatment for hemophilia B is designed to insert a functional copy of the Factor IX gene into the body’s cells to enable long‑term production of clotting factor
Plain-language explanation of the investigational treatment. It is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The study wants to find a safe dose of the gene‑insertion therapy and see how it affects blood clotting factor levels and the need for regular factor replacement. It also checks for immune reactions to the therapy.
- Who it's for
- People of any age who have severe or moderately severe hemophilia B caused by a mutation in the Factor 9 gene.
- What's involved
- Participants receive a single dose of the experimental gene‑insertion therapy and have regular blood tests to measure factor levels, antibodies, and bleeding episodes. They may continue their usual factor replacement as needed.
- Time commitment
- The study lasts several months with multiple clinic visits for dosing, blood draws, and safety check‑ups.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 and older, men only
You may be able to join if
- Confirmed diagnosis of severe or moderately severe hemophilia B with medical history of FIX functional activity (≤2% or \<0.02 IU/mL) or documented...
- Currently taking FIX prophylaxis and previous experience with FIX therapy, as defined in the protocol
- Participation in the lead-in period of this treatment study OR a separate lead-in study (R0000-HEMB-2187 [NCT05568459]) for at least 6 months for ABR...
You likely can't join if
- 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...
- Any significant underlying liver disease such as: cholestatic liver disease, liver cirrhosis, portal hypertension, splenomegaly, hepatic...
- 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...
See the full eligibility criteria
- 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 treatment 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
- 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 NOTE: Other Inclusion/Exclusion Protocol Defined...
The study team makes the final eligibility decision.
Where it is enrolling
- Phoenix, Arizona, United States
- Los Angeles, California, United States
- Sacramento, California, United States
- San Francisco, California, United States
- Aurora, Colorado, United States
- New Haven, Connecticut, United States
- Gainesville, Florida, United States
- Indianapolis, Indiana, United States
- New Orleans, Louisiana, United States
- Ann Arbor, Michigan, United States
- Columbus, Ohio, United States
- Philadelphia, Pennsylvania, United States
- Nashville, Tennessee, United States
- Dallas, Texas, United States
- Houston, Texas, United States
- Camperdown, New South Wales, Australia
- Herston, Queensland, Australia
- Adelaide, South Australia, Australia
- Melbourne, Victoria, Australia
- Vitória, Espírito Santo, Brazil
+ 27 more site(s).
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling male, 2 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at Phoenix, Arizona, United States; Los Angeles, California, United States; Sacramento, California, United States; San Francisco, California, United States; Aurora, Colorado, United States; New Haven, Connecticut, United States and 41 more locations. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.