Gene therapy for treating hemophilia B

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This study investigates the safety and effectiveness of a gene insertion therapy called REGV131-LNP1265 for patients with severe or moderately severe hemophilia B. It employs a two-part adaptive design, starting with dose escalation in adults to determine the recommended dose for expansion. Following this, the study will expand to include adolescents and pediatric patients, assessing the therapy's ability to enable the body to produce clotting factor IX long-term. The study will also monitor side effects and the body's immune response to the therapy.

Why it matters

Who can join: the full rules

Key Inclusion Criteria:

1. 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
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:

1. 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 AAV8 capsid; as measured by Enzyme-Linked ImmunoSorbent Assay (ELISA) at prescreening (or final lead-in visit, if applicable)
4. Any significant underlying liver disease such as: cholestatic liver disease, liver cirrhosis, portal hypertension, splenomegaly, hepatic encephalopathy
5. Evidence of advanced liver fibrosis or significant fatty liver, as defined in the protocol
6. Evidence of cirrhosis and/or portal hypertension as assessed by abdominal ultrasound at screening or measured within 6 months prior to the screening visit
7. History of arterial or venous thrombo-embolic events, as defined in the protocol
8. History of hypersensitivity to corticosteroids or known medical condition that requires chronic administration of corticosteroids
9. 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 Inclusion/Exclusion Protocol Defined Criteria Apply

Where it's running

• Phoenix Children's Hospital
  Phoenix, Arizona, United States Recruiting
• Orthopaedic Hemophilia Treatment Center
  Los Angeles, California, United States Recruiting
• David Geffen School of Medicine at UCLA
  Los Angeles, California, United States Recruiting
• Children's Hospital Los Angeles
  Los Angeles, California, United States Recruiting
• University of California Davis
  Sacramento, California, United States Recruiting
• University California San Francisco
  San Francisco, California, United States Recruiting

+2 more sites. See ClinicalTrials.gov for the full list.

Study contacts

• Study coordinator: Clinical Trials Administrator
• Email: clinicaltrials@regeneron.com
• Phone: 844-734-6643

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• SR604: a new treatment tested in adults with Hemophilia A/B or congenital Factor VII deficiency

  This study tests a single and multiple doses of SR604 to see if it is safe and effective for adults with Hemophilia A or B, with or without inhibitors, and for those with congenital Factor VII deficiency.

  Recruiting at 7 sites Phase 1/2 Shanghai RAAS Blood Products Co., Ltd. · Changsha and 8 other locations · NCT07644832

• Single-dose CSL222 gene therapy for adolescent males (12–17) with severe or moderately severe hemophilia B.

  This will give a single dose of CSL222 gene therapy to adolescent males with severe or moderately severe hemophilia B to see if it raises factor IX activity and reduces bleeding.

  Recruiting at 17 sites Phase 3 CSL Behring · Orange, California and 16 other locations · NCT07080905

• ATHNdataset registry for people with bleeding and clotting disorders

  This registry collects health, treatment, and patient-reported information from people with bleeding and clotting disorders seen at ATHN-affiliated centers to help improve care and outcomes.

  Recruiting American Thrombosis and Hemostasis Network · Hickory, North Carolina · NCT06820515

• Is synovial proliferation in haemophilia joints active or inactive?

  This project will test whether ultrasound, MRI and elastography can tell if subclinical synovial proliferation in people with severe haemophilia is currently active so clinicians can treat earlier.

  Recruiting UMC Utrecht · Utrecht · NCT06809972

• Observational study of blood clotting and bleeding disorders in Chinese patients

  This study looks at blood clotting and bleeding problems in Chinese patients to see how common they are and what factors might affect them.

  Recruiting Peking University People's Hospital · Beijing, Beijing Municipality and 4 other locations · NCT06727669

• Comparing ANB-002 to standard treatment for hemophilia B

  This study is testing a new treatment called ANB-002 to see if it works as well as the standard treatment for adults with hemophilia B who have low FIX activity and no FIX inhibitors.

  Recruiting at 11 sites Phase 3 Biocad · Chelyabinsk and 13 other locations · NCT06700096

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.