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R131L1265-HEMB-2318; BEYOND-9

  • Status
    Accepting Candidates
  • Age
    18 Years - N/A
  • Sexes
    Male
  • Healthy Volunteers
    No
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Objective

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.

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 quality of life is affected by hemophilia B and if it changes after taking study drug

  • How joint health is affected by hemophilia B and if it changes after taking study drug

  • How often visits are required for the emergency room, urgent care center, physician's office, hospital, telephone or online are required as a result of bleeding events, and if the frequency changes after taking study drug

  • 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

Description

The study will be conducted with a 2-part adaptive design, with enrollment of patients into sequential parts of the study.

Part 1: Dose Escalation and Dose Confirmation in adult patients ≥18 years of age

  • Dose Escalation Cohorts to determine the recommended dose for expansion (RDE) of REGV131-LNP1265

  • Dose Confirmation Cohort to gain further confidence in safety, tolerability, and Coagulation Factor IX (FIX) functional activity data at the RDE

Part 2: Dose Expansion at the Rde

  • Part 2A: Adult patients ≥18 years of age: RDE of REGV131-LNP1265, as determined in Part 1

  • Part 2B: Adolescent patients

Details

Full study title A Two-Part Open-Label Study Of REGV131-LNP1265, a CRISPR/CAS9-Based Coagulation Factor IX Gene Insertion Therapy In Participants With Hemophilia B
Protocol number OCR45553
ClinicalTrials.gov ID NCT06379789
Phase Phase 1/Phase 2

Eligibility

Key Inclusion Criteria:

  1. Confirmed diagnosis of severe or moderately severe hemophilia B with medical history of FIX functional activity (≤2% or

Lead researcher

  • Tung T Wynn, MD
    Pediatric Hematologist/Oncologist (Child Cancer Specialist)
    Tung T Wynn

Participate in a study

Here are some general steps to consider when participating in a research study:

  1. Step
    1

    Contact the research team

    Call or email the research team listed within the specific clinical trial or study to let them know that you're interested. A member of the research team, such as the researcher or study coordinator, will be available to tell you more about the study and to answer any questions or concerns you may have.

    Primary contact

  2. Step
    2

    Get screened to confirm eligibility

    You may be asked to take part in prescreening to make sure you are eligible for a study. The prescreening process ensures it is safe for you to participate. During the prescreening process, you will be asked some questions and you may also be asked to schedule tests or procedures to confirm your eligibility.

  3. Step
    3

    Provide your consent to participate

    If you are eligible and want to join the clinical trial or study, a member of the research team will ask for your consent to participate. To give consent, you will be asked to read and sign a consent form for the study. This consent form explains the study's purpose, procedures, risks, benefits and provides other important information, such as the study team's contact information.

  4. Step
    4

    Participate

    If you decide to participate in a clinical trial or study, the research team will keep you informed of the study requirements and what you will need to do to throughout the study. For some trials or studies, your health care provider may work with the research team to ensure there are no conflicts with other medications or treatments.