Comparing 7 approaches 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
A Two-Part Open-Label Study of REGV131-LNP1265, A CRISPR/Cas9 Based Coagulation Factor IX Gene Insertion Therapy in Participants With Hemophilia B
- Phase 1
- 7 groups
- Sites in Edmonton, Hamilton and 1 more city
- Recruiting
Interventions
- Medication
REGV131
Administered per the protocol before LNP1265
- Medication
LNP1265
Administered per the protocol following REGV131
Canadian Sites (3)
3 of 3 recruiting
- Recruiting
University of Alberta Hospital
Edmonton, Alberta
- Recruiting
McMaster University Medical Centre - Hamilton Health Sciences
Hamilton, Ontario
- Recruiting
McGill University Health Center (MUHC)
Montreal, Quebec
Eligibility Criteria
See who this study is looking for13 criteria
The trial’s own eligibility text, word for word from the registry. Only the trial site can say who takes part.
Inclusion
- +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 protocolProtocolThe detailed plan a study must follow.Read more →
- +Participation in the lead-in periodRun-in periodA short trial period before the study proper begins.Read more → of this interventional studyInterventional studyA study where participants are given something to see what happens.Read more → 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
- −History of hypersensitivity to corticosteroids or known medical condition that requires chronic administration of corticosteroids
- −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 screeningScreeningThe checks done before joining, to see whether a study fits.Read more →
- −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 protocolProtocolThe detailed plan a study must follow.Read more →
- −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
- −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 InclusionInclusion criteriaThe things you must have or be for a study to consider you.Read more →/ExclusionExclusion criteriaThe things that would prevent someone from taking part.Read more → Protocol Defined Criteria Apply
In plain language
Assembled directly from this trial’s registry record. Every sentence traces to a field below — nothing here is generated or interpreted.
What this study is
This study is testing a treatment for a condition.
Phase 1Phase 1The earliest stage of human testing, in a small group, focused on safety.Read more →/2 — a combined study that starts with early safety and continues into what the treatment does.
From the trial registry
Built from these fields:
- designModule.designInfo.primaryPurpose
- designModule.phases
Who receives what
There are 7 groups in this study.
Groups A, B, C, D, E, F and G receive one or more of: REGV131 and LNP1265.
Registry label: A: Part 1: Cohort 1 Dose Escalation for RDE · B: Part 1: Cohort 2 Dose Escalation for RDE · C: Part 1: Cohort 3 Dose Escalation for RDE · D: Part 1: Cohort 4 Dose Escalation for RDE · E: Part 2: Dose Expansion A · F: Part 2: Dose Expansion B · G: Part 2: Dose Expansion C
From the trial registry
Built from these fields:
- armsInterventionsModule.armGroups[].label
- armsInterventionsModule.armGroups[].type
- armsInterventionsModule.armGroups[].interventionNames
How the study is run
Groups are assigned by the study team using set rules, rather than by chance.
This study is open-labelOpen-labelEveryone knows which treatment is being given — nothing is hidden.Read more →: everyone knows which treatment is being given, including you and the study team.
From the trial registry
Built from these fields:
- designModule.designInfo.allocation
- designModule.designInfo.maskingInfo.masking
Is there a placebo?
This study does not list a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group.
From the trial registry
Built from these fields:
- armsInterventionsModule.armGroups[].type
- armsInterventionsModule.armGroups[].interventionNames
Who the study is looking for
The study lists a minimum age of 2 years, with no upper limit given.
The study lists male participants only.
The study does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
These are the criteria the study lists. Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part.
From the trial registry
Built from these fields:
- eligibilityModule.minimumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How big and how long
The study aims to enrol about 130 people.
The study is currently expected to finish around August 2047.
The main measurement is taken over: Up to 2 Years.
From the trial registry
Built from these fields:
- designModule.enrollmentInfo.count
- statusModule.completionDateStruct.date
- outcomesModule.primaryOutcomes[].timeFrame
What the study measures
Occurrence of Treatment-Emergent Adverse EventsAdverse eventAny medical problem that happens during a study, whether or not the treatment caused it.Read more → (TEAEs) — measured over Up to 2 Years.
Severity of TEAEs — measured over Up to 2 Years.
Coagulation Factor IX (FIX) functional activity measured using the chromogenic substrate assay — measured over Up to 2 Years.
Change in FIX functional activity in plasma, measured using the chromogenic substrate assay — measured over Up to 2 Years.
The study lists 7 further main measurements.
From the trial registry
Built from these fields:
- outcomesModule.primaryOutcomes[].measure
- outcomesModule.primaryOutcomes[].timeFrame
Source: NCT06379789 on ClinicalTrials.gov. The full registry text is further down this page — this summary never replaces it.
Not medical advice. What do these terms mean?
What is being tested — in plain terms
About REGV131Drug
Administered per the protocol before LNP1265
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
About LNP1265Drug
Administered per the protocol following REGV131
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
Common questions
Answered from this trial’s registry record. Where the record doesn’t say, these answers say so rather than filling the gap.
Am I eligible for this trial?
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part. Concord cannot make that determination, and neither can any tool that has not examined you.
What the study lists: a minimum age of 2 years.
The full inclusionInclusion criteriaThe things you must have or be for a study to consider you.Read more → and exclusion criteriaExclusion criteriaThe things that would prevent someone from taking part.Read more → are published on this page, exactly as the study team wrote them.
The useful next step is to bring this trial to your doctor. Answering a few questions first gives them something concrete to review.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.eligibilityCriteria
Is there a placebo?
This study does not list a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group.
From the trial registry
- armsInterventionsModule.armGroups[].type
Would I know which treatment I am getting?
This study is open-labelOpen-labelEveryone knows which treatment is being given — nothing is hidden.Read more →: everyone knows which treatment is being given, including you and the study team.
Groups are assigned by the study team using set rules, rather than by chance.
From the trial registry
- designModule.designInfo.maskingInfo.masking
- designModule.designInfo.allocation
Who can join?
The study lists a minimum age of 2 years, with no upper limit given.
It lists male participants only.
It does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can confirm whether a particular person can take part.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How long would this take?
The study's main measurement is taken over: Up to 2 Years.
The study as a whole is currently expected to finish around 2047-08-14.
How long any one person takes part can differ from the study length. The study team can tell you what the schedule looks like in practice.
From the trial registry
- outcomesModule.primaryOutcomes[].timeFrame
- statusModule.completionDateStruct.date
How many people are taking part?
The study aims to enrol about 130 people.
From the trial registry
- designModule.enrollmentInfo.count
Where is this happening?
This study lists 4 locations, including: Edmonton, Alberta, Canada; Hamilton, Ontario, Canada; Montreal, Quebec, Canada; Ann Arbor, Michigan, United States.
Sites can open and close during a study, so confirm with the team before travelling.
From the trial registry
- trial_locations
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, both in the near term and over time. 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 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
Other Sites (1)
University of Michigan
Ann Arbor, Michigan, United States
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See if this trial could fit youThis page is for informational purposes only and does not constitute medical advice. Clinical trial eligibility can only be determined by the trial site after proper screening. Trial information is sourced from ClinicalTrials.gov and may not reflect the most current status. Always consult your healthcare provider before making decisions about clinical trial participation.