RECRUITINGPhase 1 / Phase 2INTERVENTIONAL

BeCoMe-9: A Clinical Study of BE-101 for the Treatment of Adults With Moderately Severe or Severe Hemophilia B

BeCoMe-9: A Phase 1/2 Dose Escalation and Expansion Study of BE-101 for the Treatment of Adults With Moderately Severe or Severe Hemophilia B

About This Trial

The BeCoMe-9 Study (BE-101-01) is a Phase 1/2, first in human, multi-center, open-label, dose-escalation study to evaluate the safety and clinical activity of a single intravenous (IV) dose of BE-101 in adults with moderately severe or severe Hemophilia B. Once infused, BE-101 is designed to engraft and continuously secrete FIX into the circulation to restore clinically meaningful levels of active FIX. BE-101 is an autologous (person's own cells) B Cell Medicine (BCM) which uses CRISPR/Cas9 gene editing to precisely insert human FIX gene into those cells.

Who May Be Eligible (Plain English)

Who May Qualify: - Adult Males (≥18) with moderately severe to severe Hemophilia B (FIX deficiency) - Received ≥50 exposure days to Factor IX products preceding enrollment. - Currently receiving prophylaxis treatment - your organs (liver, kidneys, etc.) are working well enough based on blood tests and clinical labs - Able to tolerate study procedures including leukapheresis. Who Should NOT Join This Trial: - Pre-existing or history of specific diseases - B-Cell malignancy, EBV lymphoproliferative disease - Primary weakened immune system disease or disorder (PIDD) or systemic immuno-suppression - Arterial and/or venous thromboembolic events within 2 years prior to dosing - History of anaphylaxis or nephrotic syndrome - Active infection (HIV, Hep B or C) - History of inhibitor to FIX or inhibitor - History of an allergic reaction or anaphylaxis to FIX products - Planned surgical procedure within 6 months from BE-101 administration - Previously dosed with gene therapy - Participated in an interventional study and/or received an interventional study drug within 30 days or five-half-lives (whichever is longer) of consent into BeCoMe-9 and for the duration of the study - Planned participation in clinical trial within one year after BE-101 - Administration of an investigational agent or vaccine within 28 days of leukapheresis and dosing of BE-101 Other protocol-defined inclusion/exclusion criteria may apply. Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language

Inclusion Criteria: * Adult Males (≥18) with moderately severe to severe Hemophilia B (FIX deficiency) * Received ≥50 exposure days to Factor IX products preceding enrollment. * Currently receiving prophylaxis treatment * Adequate organ function and clinical labs * Able to tolerate study procedures including leukapheresis. Exclusion Criteria: * Pre-existing or history of specific diseases * B-Cell malignancy, EBV lymphoproliferative disease * Primary immunodeficiency disease or disorder (PIDD) or systemic immuno-suppression * Arterial and/or venous thromboembolic events within 2 years prior to dosing * History of anaphylaxis or nephrotic syndrome * Active infection (HIV, Hep B or C) * History of inhibitor to FIX or inhibitor * History of an allergic reaction or anaphylaxis to FIX products * Planned surgical procedure within 6 months from BE-101 administration * Previously dosed with gene therapy * Participated in an interventional study and/or received an interventional study drug within 30 days or five-half-lives (whichever is longer) of consent into BeCoMe-9 and for the duration of the study * Planned participation in clinical trial within one year after BE-101 * Administration of an investigational agent or vaccine within 28 days of leukapheresis and dosing of BE-101 Other protocol-defined inclusion/exclusion criteria may apply.

Treatments Being Tested

DRUG

BE-101

IV Infusion of BE-101 dose from Dose Level 1

DRUG

BE-101

IV Infusion of BE-101 dose from Dose Level 2

DRUG

BE-101

IV Infusion of BE-101 dose from Dose Level 3

DRUG

BE-101

IV Infusion of BE-101 with optimal dose selected from Part 1 Dose Escalation

Locations (4)

University of California, Davis

Davis, California, United States

University of Michigan

Ann Arbor, Michigan, United States

University of Minnesota

Minneapolis, Minnesota, United States

Washington Center for Bleeding Disorders

Seattle, Washington, United States