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Gene Therapy Trial Report

Summary

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


NCTID NCT06611436 (View at clinicaltrials.gov)
Description
Development Status πŸ”„ Inactive
Indication Hemophilia B
Disease Ontology Term DOID:12259
Drug Product Name BE-101
Drug Product Description Padua variant
Sponsor Be Biopharma
Funder Type Industry
Recruitment Status πŸ”„
Terminated
Enrollment Count πŸ”„ 5 (ACTUAL)
Results Posted Not Available

Therapy Information


Target Gene/Variant F9
Therapeutic Modality Gene editing
Therapy Route ex vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Autologous cells
Target Tissue/Cell B cells
Gene Delivery System Type Viral transduction
Vector Type πŸ”„ Adeno-associated virus
Viral Vector Subtype AAV6
Editor Type Cas9 RNP
Dose 1 πŸ”„ Undisclosed dose escalation, 3 levels

Study Record Dates


Current Phase Phase1, Phase2
Submit Date 2024-09-18
Completion Date πŸ”„ 2026-05-29
Last Update πŸ”„ 2026-06-15

Participation Criteria


Eligible Age >=18 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study MALE
Eligibility Criteria
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.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites πŸ”„ 5
Locations United States

Regulatory Information


Has US IND True
FDA Designations Fast Track, Orphan Drug Designation
Recent Updates Program terminated due to strategic business decisions, website is no longer available

Resources/Links