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Tests treatment safety and results for Hemophilia B
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?
REGV131 is an investigational medicine, being studied as a potential treatment for hemophilia b.
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
- 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".
- 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's taking place
- 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
- Camperdown, New South Wales, Australia
- Herston, Queensland, Australia
- Adelaide, South Australia, Australia
- Edmonton, Alberta, Canada
- Hamilton, Ontario, Canada
- Montreal, Quebec, Canada
- Bron, Lyon, France
- Lille, Nord, France
+ 19 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 - 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?
Study sites include 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 and 33 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.