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NCT06379789RECRUITINGIRB Ready

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

(BEYOND-9)

Sponsored by Regeneron Pharmaceuticals

Quick Self-Assessment

See if you qualify and express your interest

Preparing your pre-screening questions...
๐Ÿ“Š
1 of 130 participants interested
1% interest

Express Your Interest Today

Interested in learning more? Complete the form below.

Secure & Confidential

Your information is protected and will only be shared with the research team.

Why Participate?

  • No-Cost Study Care

  • Advance Medical Science

    Contribute to groundbreaking research

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel (varies by study)

  • Help Others

    Benefit future patients

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3In-person assessment if eligible
  4. 4Begin participation

About This Study

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.

Who Can Participate

Inclusion Criteria

Confirmed diagnosis of severe or moderately severe hemophilia B with medical history of FIX functional activity (โ‰ค2% or \<0.02 IU/mL) or documented genotype known to produce severe hemophilia B 2. Currently taking FIX prophylaxis and previous experience with FIX therapy, as defined in the protocol 3. 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 Key

Exclusion Criteria

History of FIX inhibitor (clinical or laboratory-based assessment) on 2 or more occasions 2. Bethesda inhibitor titer greater than the upper limit of normal (ULN) at screening 3. Detectable pre-existing antibodies to the adeno-associated virus serotype 8 (AAV8) capsid; as measured by enzyme-linked immunosorbent assay (ELISA) at prescreening (or final lead-in visit, if applicable). 4. Any significant underlying liver di

โ“ Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility during the screening process.

Study Details

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Study Type

INTERVENTIONAL

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Target Enrollment

130 participants

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Gender

MALE

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Sponsor

Regeneron Pharmaceuticals

Study Locations

Choose your preferred location or select flexible during enrollment

RECRUITING

Los Angeles

California

Location available
View Los Angeles location page
RECRUITING

Los Angeles

California

Location available
View Los Angeles location page
RECRUITING

Los Angeles

California

Location available
View Los Angeles location page
RECRUITING

Sacramento

California

Location available
View Sacramento location page
RECRUITING

San Francisco

California

Location available
View San Francisco location page
RECRUITING

Aurora

Colorado

Location available
View Aurora location page
RECRUITING

New Haven

Connecticut

Location available
View New Haven location page
RECRUITING

Gainesville

Florida

Location available
View Gainesville location page
RECRUITING

Indianapolis

Indiana

Location available
View Indianapolis location page

๐Ÿ“ And 4 more locations available

Frequently Asked Questions

Q:What is a clinical trial?

A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.

Q:Will I be compensated?

Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.

Q:Will I receive a placebo instead of treatment?

When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.

Q:Can I leave a trial if I change my mind?

Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.

Q:How long does a clinical trial last?

Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.

Still have questions? Our study coordinators are here to help.

Ready to Make a Difference?

Express your interest in this groundbreaking clinical trial

๐Ÿ”’ Secure ยท ๐Ÿฅ Expert Care

Looking for Hemophilia B Treatment in Los Angeles?

Join others in California exploring innovative treatment options through clinical research

Hemophilia B Treatment Options in Los Angeles, California

If you're searching for Hemophilia B treatment in Los Angeles, participating in a clinical research study may provide access to innovative approaches under expert medical supervision. This study is actively recruiting participants in Los Angeles, Los Angeles, Los Angeles and surrounding areas.

Clinical trials offer participants the opportunity to receive cutting-edge treatments while contributing to medical research that may help future patients with Hemophilia B. All study-related care is provided at no cost to participants.

Local Sites
3 locations in California
Now Enrolling
Up to 130 participants
Quick Start
Screening available now

Why Consider a Clinical Trial for Hemophilia B?

Potential Benefits

  • โœ“Access to new treatment approaches before public availability
  • โœ“Close monitoring by experienced medical professionals
  • โœ“Study-related care provided at no cost
  • โœ“Contribute to medical research for Hemophilia B

What to Expect

  • โ†’Initial screening to determine eligibility
  • โ†’Regular check-ups and monitoring visits
  • โ†’Possible compensation for time and travel
  • โ†’You can withdraw at any time

Frequently Asked Questions About This Hemophilia B Study

Important Clinical Trial Information

This information is provided for educational purposes and does not constitute medical advice. Clinical trial participation involves potential risks and benefits. Eligibility requirements apply and will be assessed during the screening process.

Study identifier: NCT06379789. For complete study details, visit ClinicalTrials.gov. Always consult with your healthcare provider before making decisions about your medical care or participating in clinical research.