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

RecruitingObservational study

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

Who can join

Ages 14 to 37 · All sexes

Full eligibility criteria
Inclusion Criteria:

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

Exclusion Criteria:

* There are no exclusion criteria for this study.

About the study

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).

What is being tested

Sponsor: Beam Therapeutics Inc. · Participants: 50 · Started: Dec 15, 2025

Contact the study team

Official record on ClinicalTrials.gov — NCT07373639

Locations in the U.S.

AlabamaUniversity of Alabama at Birmingham, Birmingham (Not yet recruiting)
ArizonaPhoenix Children's Hospital, Phoenix (Not yet recruiting)
FloridaMayo Clinic Florida, Jacksonville (Not yet recruiting)
GeorgiaChildren's Healthcare of Atlanta - Aflac Cancer and Blood Disorders Center- Egleston, Atlanta (Not yet recruiting)
MassachusettsBoston Children's Hospital, Boston
MichiganHenry Ford Cancer Center, Detroit (Not yet recruiting)
MinnesotaUniversity of Minnesota, Minneapolis (Not yet recruiting)
MissouriWashington University School of Medicine in St. Louis, St Louis (Not yet recruiting)
New JerseyHackensack University Medical Center, Hackensack (Not yet recruiting)
New YorkColumbia University Irving Medical Center, New York (Not yet recruiting)
OhioThe Clevland Clinic Foundation, Cleveland (Not yet recruiting)
University Hospitals Clevland Medical Center, Cleveland (Not yet recruiting)
PennsylvaniaChildren's Hospital of Philadelphia, Philadelphia (Not yet recruiting)
South CarolinaMedical University of South Carolina, Charleston (Not yet recruiting)
TennesseeSt. Jude Children's Research Hospital, Memphis (Not yet recruiting)
The Children's Hospital at TriStar Centennial, Nashville (Not yet recruiting)
WisconsinMedical College of Wisconsin, Milwaukee (Not yet recruiting)

Conditions

From ClinicalTrials.gov, data retrieved Sep 29, 2026. Each study sets its own eligibility; the study team decides who can join.