RecruitingNCT07373639

A Long-term Follow-up Study in Patients Who Received BEAM-101

A Long-term Follow-up Study in Patients With Hemoglobinopathy Who Received Autologous CD34+ Edited Hematopoietic Stem Cells


Sponsor

Beam Therapeutics Inc.

Enrollment

50 participants

Start Date

Dec 15, 2025

Study Type

OBSERVATIONAL

Conditions

Summary

This is a Long-term Follow-up (LTFU) study in patients who received BEAM-101 in the parent study (Study BTX-AUT-001). Eligible patients who received BEAM-101 will be asked to participate in this LTFU study prior to completing Study BTX-AUT-001. Patients who enter into this study will be followed for 13 years (a total of 15 years after receiving BEAM-101).


Eligibility

Min Age: 14 YearsMax Age: 37 Years

Inclusion Criteria2

  • Have received BEAM-101 in Study BTX-AUT-001 and are in the process of completing that study's end-of-study (EOS) visit.
  • Provide signed, written informed consent according to local institutional review board (IRB)/independent ethics committee (IEC) and institutional requirements.

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Interventions

DRUGLong-Term Follow-up Study of patients who received BEAM-101

This is a Long-Term Follow-up Study of patients who received a single dose of BEAM-101 administered by IV following myeloablative conditioning with busulfan


Locations(17)

University of Alabama at Birmingham

Birmingham, Alabama, United States

Phoenix Children's Hospital

Phoenix, Arizona, United States

Mayo Clinic Florida

Jacksonville, Florida, United States

Children's Healthcare of Atlanta - Aflac Cancer and Blood Disorders Center- Egleston

Atlanta, Georgia, United States

Boston Children's Hospital

Boston, Massachusetts, United States

Henry Ford Cancer Center

Detroit, Michigan, United States

University of Minnesota

Minneapolis, Minnesota, United States

Washington University School of Medicine in St. Louis

St Louis, Missouri, United States

Hackensack University Medical Center

Hackensack, New Jersey, United States

Columbia University Irving Medical Center

New York, New York, United States

University Hospitals Clevland Medical Center

Cleveland, Ohio, United States

The Clevland Clinic Foundation

Cleveland, Ohio, United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Medical University of South Carolina

Charleston, South Carolina, United States

St. Jude Children's Research Hospital

Memphis, Tennessee, United States

The Children's Hospital at TriStar Centennial

Nashville, Tennessee, United States

Medical College of Wisconsin

Milwaukee, Wisconsin, United States

View Full Details on ClinicalTrials.gov

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NCT07373639


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