A Long-term Follow-up Study in Patients Who Received BEAM-101
1 other identifier
observational
50
1 country
17
Brief 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).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for all trials
Started Dec 2025
Longer than P75 for all trials
17 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
December 15, 2025
CompletedFirst Submitted
Initial submission to the registry
January 6, 2026
CompletedFirst Posted
Study publicly available on registry
January 28, 2026
CompletedPrimary Completion
Last participant's last visit for primary outcome
February 12, 2043
ExpectedStudy Completion
Last participant's last visit for all outcomes
February 12, 2043
January 28, 2026
November 1, 2025
17.2 years
January 6, 2026
January 23, 2026
Conditions
Outcome Measures
Primary Outcomes (1)
Long-Term Safety Monitoring and Mortality Assessment
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.
Up to 13 years
Secondary Outcomes (10)
Proportion of patients without severe VOCs overtime
Through year 15
Duration of being severe VOC-free
Through year 15
Frequency of RBC transfusions over time
Through year 15
Proportion (%) of patients requiring treatment for SCD related complications
Through year 15
Total Hemoglobin (Hb) Concentration (g/dL) over time
Through year 15
- +5 more secondary outcomes
Study Arms (1)
BTX-101-002 Long-term Follow-up on Patients who received BEAM-101
All participants who received BEAM-101 in the BTX-AUT-001 trial will be asked to participate in this long-term follow-up study
Interventions
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
Eligibility Criteria
The study population consists of participants enrolled at sites from the parent study (BTX-AUT-001) who meet the criteria for long-term follow-up
You may qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (17)
University of Alabama at Birmingham
Birmingham, Alabama, 35233, United States
Phoenix Children's Hospital
Phoenix, Arizona, 85016, United States
Mayo Clinic Florida
Jacksonville, Florida, 32224, United States
Children's Healthcare of Atlanta - Aflac Cancer and Blood Disorders Center- Egleston
Atlanta, Georgia, 30329, United States
Boston Children's Hospital
Boston, Massachusetts, 02215, United States
Henry Ford Cancer Center
Detroit, Michigan, 48202, United States
University of Minnesota
Minneapolis, Minnesota, 55455, United States
Washington University School of Medicine in St. Louis
St Louis, Missouri, 63110, United States
Hackensack University Medical Center
Hackensack, New Jersey, 07601, United States
Columbia University Irving Medical Center
New York, New York, 10032, United States
University Hospitals Clevland Medical Center
Cleveland, Ohio, 44106, United States
The Clevland Clinic Foundation
Cleveland, Ohio, 44195, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
Medical University of South Carolina
Charleston, South Carolina, 29425, United States
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105, United States
The Children's Hospital at TriStar Centennial
Nashville, Tennessee, 37203, United States
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226, United States
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Central Study Contacts
Study Design
- Study Type
- observational
- Observational Model
- CASE ONLY
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
January 6, 2026
First Posted
January 28, 2026
Study Start
December 15, 2025
Primary Completion (Estimated)
February 12, 2043
Study Completion (Estimated)
February 12, 2043
Last Updated
January 28, 2026
Record last verified: 2025-11
Data Sharing
- IPD Sharing
- Will not share