RecruitingPHASE1, PHASE2INTERVENTIONAL

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

This study is investigating a new treatment called REGV131-LNP1265, which is a type of gene therapy, for children, teenagers, and adults with haemophilia B. Haemophilia B is a condition where the blood doesn't clot properly due to a missing blood clotting factor. The new gene therapy aims to teach the body how to produce this important clotting factor itself, possibly reducing or even replacing the need for ongoing treatments. The main goal is to find a safe and effective dose of this new treatment by carefully watching for any side effects, both short-term and long-term. Researchers also want to understand how the body reacts to the treatment and if it helps improve blood clotting over time.

At a glance

Status

Recruiting

Phase

PHASE1, PHASE2

Sponsor

Regeneron Pharmaceuticals

Enrolment target

130

Start

11 Sep 2024

Estimated completion

14 Aug 2047

Hemophilia B

What is this study about?

This research is looking into a new and experimental treatment called REGV131-LNP1265 for a condition called haemophilia B. People with haemophilia B have a problem with a specific gene that means their blood doesn't clot as it should. This new treatment is a type of 'gene insertion therapy', which means it aims to add a working copy of the missing gene into your body. The hope is that this new gene will then help your body produce its own blood clotting factor, known as Factor IX (Factor 9), on a long-term basis.

Currently, many people with haemophilia B need regular treatments to replace this missing Factor 9. If this new gene therapy works, it could mean you wouldn't need these regular treatments as often, or maybe even at all. This study is in its early stages (Phase 1 and 2), which means the main focus right now is to find a dose of the treatment that is safe and doesn't cause too many side effects, and to see how well it's tolerated by the body. Researchers will also be checking to see how much of the treatment stays in your blood and if your body develops any immune responses to it.

Additionally, the study will explore whether the gene therapy changes how often you need your usual Factor 9 replacement therapy, both for preventing bleeds and for treating them when they happen. The results from this study will help us understand if this new gene therapy could be a good future treatment option for people living with haemophilia B.

Key takeaways

This study is testing a new gene therapy for haemophilia B.
It aims to help your body make its own blood clotting Factor 9.
The main goal is to check for safety and find the right dose.
It could potentially reduce or replace the need for regular Factor 9 treatments.
The study involves children, teenagers, and adults with severe or moderately severe haemophilia B.

Who may be eligible?

To be considered for this study, you would need to have a clear diagnosis of severe or moderately severe haemophilia B. This means your body produces very little or no Factor 9, or you have a known genetic change that causes severe haemophilia B.

You also need to be currently receiving regular Factor 9 treatment to prevent bleeding, and have experience with this type of treatment. Before joining the main study, you would also need to take part in a separate lead-in period (or a similar earlier study) for at least 6 months while continuing your Factor 9 prevention treatment.

However, you would not be able to join if you have a history of your body making antibodies against your Factor 9 treatment, or if you already have certain antibodies that might stop this new gene therapy from working. The study also cannot include people with significant liver problems, a history of certain types of blood clots, or if you've had a similar gene therapy before.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Do you have a diagnosis of severe or moderately severe haemophilia B?
Are you currently receiving regular Factor 9 treatment?
Have you participated in a 6-month lead-in period for this type of study?
Do you have any history of your body making antibodies against Factor 9?
Do you have any significant liver problems?

Answer every question to see your result.

What does participation involve?

If you join this study, it will be split into two main parts. The first part will involve a small number of adult participants to find the safest and most effective dose of the study drug. Once that dose is found, more adults, then teenagers (aged 12 to 18), and finally younger children (aged 2 to 12) will receive the treatment.

The treatment, REGV131-LNP1265, will be given directly into your body. You will have regular check-ups, blood tests, and other assessments to monitor your health, how your body is reacting to the treatment, and how well your blood is clotting. The study will also look at whether you need less of your usual Factor 9 treatment. The exact number of visits and the full duration of your participation will be explained in detail by the study team, but typically, gene therapy studies involve long-term follow-up.

Potential risks and benefits

Participating in this study might offer the potential benefit of your body producing its own Factor 9, which could lead to needing less or no regular Factor 9 treatments. However, as this is an experimental treatment, there are potential risks, including side effects from the study drug, your body developing an immune reaction to the treatment, or the treatment not working as expected. You will be closely monitored for any reactions. You have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (42)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Orthopaedic Hemophilia Treatment Center
Verified postcode
Los Angeles, United States· Recruiting
David Geffen School of Medicine at UCLA
Verified postcode
Los Angeles, United States· Recruiting
Children's Hospital Los Angeles
Verified postcode
Los Angeles, United States· Recruiting
University of California Davis
Verified postcode
Sacramento, United States· Recruiting
University California San Francisco
Verified postcode
San Francisco, United States· Recruiting
University of Colorado Hemophilia and Thrombosis Center
Verified postcode
Aurora, United States· Recruiting
Yale HTC
Verified postcode
New Haven, United States· Recruiting
University of Florida
Verified postcode
Gainesville, United States· Recruiting
Indiana Hemophilia and Thrombosis Center
Verified postcode
Indianapolis, United States· Recruiting
Tulane University School of Medicine, Louisiana Center for Bleeding and Clotting Disorders
Verified postcode
New Orleans, United States· Recruiting
University of Michigan
Verified postcode
Ann Arbor, United States· Recruiting
Nationwide Children's Hospital
Verified postcode
Columbus, United States· Recruiting

Common questions

What is gene insertion therapy?

It's a new type of treatment where scientists try to add a working gene into your body to correct a problem caused by a faulty gene, like the one in haemophilia B.

What does 'Phase 1' and 'Phase 2' mean?

These are early stages of clinical trials. Phase 1 mainly checks if a treatment is safe and finds the right dose, while Phase 2 further checks safety and how well it works in more people.

What is Factor IX (Factor 9)?

Factor IX is a vital protein in your blood that helps it clot. People with haemophilia B don't have enough of it, leading to bleeding problems.

Will I still need my usual Factor 9 treatment?

Initially, yes. The study will assess if the new gene therapy helps reduce how much regular Factor 9 treatment you need in the long term, or even if you can stop it.

What are 'antibodies' and why are they important?

Antibodies are proteins made by your immune system to fight off foreign substances. In this study, they're important because if your body makes antibodies against the gene therapy, it might make the treatment less effective.

How to find out more

Clinical Trials Administrator

clinicaltrials@regeneron.com 844-734-6643 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.