Long-term follow-up after BEAM-101 treatment
A Long-term Follow-up Study in Patients With Hemoglobinopathy Who Received Autologous CD34+ Edited Hematopoietic Stem Cells
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 |
|---|---|
| Enrollment | 50 (estimated) |
| Ages | 14 Years to 37 Years |
| Sex | All |
| Sponsor | Beam Therapeutics Inc. Industry-sponsored |
| Locations | 17 sites (Birmingham, Alabama and 16 other locations) |
| Trial ID | NCT07373639 on ClinicalTrials.gov |
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.
Who should consider this trial
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.
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.
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 performed: 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.
Eligibility criteria
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 this trial is running
Birmingham, Alabama and 16 other locations
- 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)
- Boston Children's Hospital — Boston, Massachusetts, United States (Recruiting)
- Henry Ford Cancer Center — Detroit, Michigan, United States (Not_yet_recruiting)
- 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
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.