Long-term follow-up after BEAM-101 treatment
Official title: A Long-term Follow-up Study in Patients With Hemoglobinopathy Who Received Autologous CD34+ Edited Hematopoietic Stem Cells
Run by Beam Therapeutics Inc. · NCT07373639 · Data updated 2026-11-12
This follow-up will see how people with sickle cell disease who received BEAM-101 are doing over 15 years.
Quick facts
| Study type | Observational |
|---|---|
| Ages | 14 Years to 37 Years |
| Sex | All |
| Enrollment | 50 (estimated) |
| Sponsor | Beam Therapeutics Inc. Industry-sponsored |
| Locations | 17 sites (Birmingham, Alabama and 16 other locations) |
| Status | Recruiting |
| Started | December 15, 2025 |
| Main results expected | February 12, 2043 |
| Study ends | February 12, 2043 |
| Registry record updated | January 28, 2026 |
| Trial ID | NCT07373639 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates are patients with sickle cell disease who received BEAM-101 in BTX-AUT-001, are completing that study's end-of-study visit, and can provide informed consent and attend scheduled visits.
✕Probably not a fit
Patients who did not receive BEAM-101 or who are unable or unwilling to complete long-term in-person or remote follow-up are unlikely to benefit from this follow-up.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This observational long-term follow-up enrolls patients who received BEAM-101 in the parent BTX-AUT-001 protocol and consent to extended monitoring. Participants will have periodic safety and efficacy assessments with annual in-person visits through Year 5, then in-person visits every three years through Year 11, and a final visit at Year 15. Virtual or phone check-ins occur every six months through Year 5 and annually thereafter to capture interim events. There are no exclusion criteria beyond having received BEAM-101 and providing informed consent, and collected data will inform durability and late-effect profiles of the base-editing therapy.
Why it matters
Potential benefit: If successful, this follow-up could provide important long-term safety and durability information that helps guide care for people treated with BEAM-101.
How similar studies have gone: Long-term follow-up of other gene and cell therapies has yielded valuable safety and durability data, but BEAM-101's base-editing approach is relatively novel and lacks extensive long-term results.
Who can join: the full rules
Show full inclusion / exclusion 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.
Where it's running
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Boston Children's Hospital
Boston, Massachusetts, United States Recruiting -
University of Alabama at Birmingham
Birmingham, Alabama, United States Not yet recruiting -
Phoenix Children's Hospital
Phoenix, Arizona, United States Not yet recruiting -
Mayo Clinic Florida
Jacksonville, Florida, United States Not yet recruiting -
Children's Healthcare of Atlanta - Aflac Cancer and Blood Disorders Center- Egleston
Atlanta, Georgia, United States Not yet recruiting -
Henry Ford Cancer Center
Detroit, Michigan, United States Not yet recruiting
Show 11 more locations
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University of Minnesota
Minneapolis, Minnesota, United States Not yet recruiting -
Washington University School of Medicine in St. Louis
St Louis, Missouri, United States Not yet recruiting -
Hackensack University Medical Center
Hackensack, New Jersey, United States Not yet recruiting -
Columbia University Irving Medical Center
New York, New York, United States Not yet recruiting -
University Hospitals Clevland Medical Center
Cleveland, Ohio, United States Not yet recruiting -
The Clevland Clinic Foundation
Cleveland, Ohio, United States Not yet recruiting -
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States Not yet recruiting -
Medical University of South Carolina
Charleston, South Carolina, United States Not yet recruiting -
St. Jude Children's Research Hospital
Memphis, Tennessee, United States Not yet recruiting -
The Children's Hospital at TriStar Centennial
Nashville, Tennessee, United States Not yet recruiting -
Medical College of Wisconsin
Milwaukee, Wisconsin, United States Not yet recruiting
Study contacts
- Study coordinator: Medical Information
- Email: clinicalinfo@beamtx.com
- Phone: +1 857-327-8641
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How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.