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

NCT07373639 · tracked via the Priya Life Science USA tracker

Sponsor

Beam Therapeutics Inc.

Phase

Observational

Started

2025-12-15

Last updated

2026-01-28

Condition(s) studied

HemoglobinopathySickle Cell Disease

Investigational drug(s) / intervention(s)

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

Long-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

Study 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

Sex

ALL

Min age

14 Years

Max age

37 Years

Healthy volunteers

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.

Primary outcome measure(s)

Long-Term Safety Monitoring and Mortality Assessment — Up to 13 years
Monitor all BEAM-101-related AEs and SAEs through Year 15 post-IMP administration, all SAEs regardless of relatedness through Year 5, new malignancies, new or worsening bone marrow-related hematologic disorders, and all-cause mortality.

Trial sites (17)

Facility	City	Region	Status
University of Alabama at Birmingham	Birmingham	Alabama	Not Yet Recruiting
Phoenix Children's Hospital	Phoenix	Arizona	Not Yet Recruiting
Mayo Clinic Florida	Jacksonville	Florida	Not Yet Recruiting
Children's Healthcare of Atlanta - Aflac Cancer and Blood Disorders Center- Egleston	Atlanta	Georgia	Not Yet Recruiting
Boston Children's Hospital	Boston	Massachusetts	Recruiting
Henry Ford Cancer Center	Detroit	Michigan	Not Yet Recruiting
University of Minnesota	Minneapolis	Minnesota	Not Yet Recruiting
Washington University School of Medicine in St. Louis	St Louis	Missouri	Not Yet Recruiting
Hackensack University Medical Center	Hackensack	New Jersey	Not Yet Recruiting
Columbia University Irving Medical Center	New York	New York	Not Yet Recruiting
University Hospitals Clevland Medical Center	Cleveland	Ohio	Not Yet Recruiting
The Clevland Clinic Foundation	Cleveland	Ohio	Not Yet Recruiting
Children's Hospital of Philadelphia	Philadelphia	Pennsylvania	Not Yet Recruiting
Medical University of South Carolina	Charleston	South Carolina	Not Yet Recruiting
St. Jude Children's Research Hospital	Memphis	Tennessee	Not Yet Recruiting
The Children's Hospital at TriStar Centennial	Nashville	Tennessee	Not Yet Recruiting
Medical College of Wisconsin	Milwaukee	Wisconsin	Not Yet Recruiting

More Beam Therapeutics Inc. trials in the USA

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A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD) Recruiting

BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease Active, not recruiting

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Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07373639 on ClinicalTrials.gov ↗ ← All trials in the USA