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A Trial to Learn if REGV131-LNP1265 is Safe and Works to Help the Body Make Clotting Factor in Adult Patients with Hemophilia B
EU CTIS ID: 2023-507260-40-00
What this study is testing
Part 1: To evaluate the safety and tolerability of a single IV administration of REGV131-LNP1265 in participants with hemophilia B. To evaluate clotting FIX functional activity following a single IV administration of REGV131-LNP1265 in participants with hemophilia B to establish the recommended dose for further development Part 2A: To evaluate clotting FIX functional activity following a single IV administration of REGV131-LNP1265 in participants with hemophilia B receiving the recommended dose for expansion (RDE). To assess the bleeding event rate (all bleeding events) following a single IV administration of REGV131-LNP1265 at the RDE Long-term Follow Up (LTFU) Period: To evaluate the long-term safety of a single IV administration of REGV131-LNP1265 in participants with hemophilia B
- Phase I and Phase II (Integrated)- First administration to humans
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Male gender at birth and ≥18 years of age.
- 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.
- NOTE: Other Inclusion Protocol Defined Criteria Apply.
You likely can't join if
- History of FIX inhibitor (clinical or laboratory-based assessment) on 2 or more occasions.
- NOTE: Other Inclusion/Exclusion Protocol Defined Criteria Apply
- 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 [L-Final], 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.
See the full eligibility criteria
- Male gender at birth and ≥18 years of age.
- 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.
- NOTE: Other Inclusion Protocol Defined Criteria Apply.
- History of FIX inhibitor (clinical or laboratory-based assessment) on 2 or more occasions.
- NOTE: Other Inclusion/Exclusion Protocol Defined Criteria Apply
- 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 [L-Final], 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.
The study team makes the final eligibility decision.
Where it's taking place
- Australia
- United Kingdom
- Brazil
- Canada
- United States
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, 18-64 years, 65+ years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Australia; United Kingdom; Brazil; Canada; United States. 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 EU CTIS; the study team decides eligibility.