Actively Recruiting
A Long-term Follow-up Study in Patients With Hemoglobinopathy Who Received Autologous CD34+ Edited Hematopoietic Stem Cells
Led by Beam Therapeutics Inc. · Updated on 2026-01-28
50
Participants Needed
17
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
This research follows patients with Hemoglobinopathy and Sickle Cell Disease who previously received BEAM-101, a gene-edited cell treatment, in an earlier study. The trial is a long-term observational follow-up to monitor safety and health outcomes over an extended period. It aims to gather important information about the long-term effects and risks after receiving BEAM-101 treatment. Participants in this study have received a single dose of BEAM-101 by intravenous infusion after a conditioning treatment with busulfan. This follow-up study will track their health for 13 years, adding up to 15 years from the initial treatment. Study visits will happen annually for the first 5 years, then every 3 years until year 11, with a final visit at year 15. Additionally, virtual or phone check-ins will occur every 6 months for the first 5 years, then annually thereafter. During the study, participants will undergo regular safety and efficacy assessments to monitor their health, including how well they remain free of severe sickle cell crises and their blood hemoglobin levels. Researchers will review long-term safety data and mortality over the 13 years. The study includes blood tests and other evaluations at scheduled visits and check-ins. Participation will last up to 15 years, with ongoing monitoring to understand the lasting impact of BEAM-101 treatment.
CONDITIONS
Brief Title
A Long-term Follow-up Study in Patients Who Received BEAM-101
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Have received BEAM-101 in Study BTX-AUT-001 and are in the process of completing that study's end-of-study visit
- Provide signed, written informed consent according to local institutional requirements
You will not qualify if you...
History of severe allergic reactions to study medication Currently pregnant or breastfeeding Recent participation in another clinical trial within the last 30 days Presence of uncontrolled medical conditions that could affect safety
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
screening and enrollment visit
Duration - Up to 15 years
Participants who received BEAM-101 in a prior study will have periodic safety and efficacy assessments including virtual or phone check-ins.
Annual visits through Year 5, then every 3 years through Year 11, and a final visit at Year 15; virtual/phone visits every 6 months through Year 5 and annually thereafter
Trial Site Locations
Total: 17 locations
1
University of Alabama at Birmingham
Birmingham, Alabama, United States, 35233
Not Yet Recruiting
2
Phoenix Children's Hospital
Phoenix, Arizona, United States, 85016
Not Yet Recruiting
3
Mayo Clinic Florida
Jacksonville, Florida, United States, 32224
Not Yet Recruiting
4
Children's Healthcare of Atlanta - Aflac Cancer and Blood Disorders Center- Egleston
Atlanta, Georgia, United States, 30329
Not Yet Recruiting
5
Boston Children's Hospital
Boston, Massachusetts, United States, 02215
Actively Recruiting
6
Henry Ford Cancer Center
Detroit, Michigan, United States, 48202
Not Yet Recruiting
7
University of Minnesota
Minneapolis, Minnesota, United States, 55455
Not Yet Recruiting
8
Washington University School of Medicine in St. Louis
St Louis, Missouri, United States, 63110
Not Yet Recruiting
9
Hackensack University Medical Center
Hackensack, New Jersey, United States, 07601
Not Yet Recruiting
10
Columbia University Irving Medical Center
New York, New York, United States, 10032
Not Yet Recruiting
11
University Hospitals Clevland Medical Center
Cleveland, Ohio, United States, 44106
Not Yet Recruiting
12
The Clevland Clinic Foundation
Cleveland, Ohio, United States, 44195
Not Yet Recruiting
13
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Not Yet Recruiting
14
Medical University of South Carolina
Charleston, South Carolina, United States, 29425
Not Yet Recruiting
15
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105
Not Yet Recruiting
16
The Children's Hospital at TriStar Centennial
Nashville, Tennessee, United States, 37203
Not Yet Recruiting
17
Medical College of Wisconsin
Milwaukee, Wisconsin, United States, 53226
Not Yet Recruiting
Research Team
M
Medical Information
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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