RECRUITINGOBSERVATIONAL
A Long-term Follow-up Study in Patients Who Received BEAM-101
A Long-term Follow-up Study in Patients With Hemoglobinopathy Who Received Autologous CD34+ Edited Hematopoietic Stem Cells
About This Trial
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).
Who May Be Eligible (Plain English)
Who May Qualify:
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 willing to sign a consent form according to local institutional review board (IRB)/independent ethics committee (IEC) and institutional requirements.
Who Should NOT Join This Trial:
- There are no exclusion criteria for this study.
Always talk to your doctor about whether this trial is right for you.
Original Eligibility Criteria
View original clinical language
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.
Treatments Being Tested
DRUG
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
Locations (17)
University of Alabama at Birmingham
Birmingham, Alabama, United States
Phoenix Children's Hospital
Phoenix, Arizona, United States
Mayo Clinic Florida
Jacksonville, Florida, United States
Children's Healthcare of Atlanta - Aflac Cancer and Blood Disorders Center- Egleston
Atlanta, Georgia, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Henry Ford Cancer Center
Detroit, Michigan, United States
University of Minnesota
Minneapolis, Minnesota, United States
Washington University School of Medicine in St. Louis
St Louis, Missouri, United States
Hackensack University Medical Center
Hackensack, New Jersey, United States
Columbia University Irving Medical Center
New York, New York, United States
University Hospitals Clevland Medical Center
Cleveland, Ohio, United States
The Clevland Clinic Foundation
Cleveland, Ohio, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Medical University of South Carolina
Charleston, South Carolina, United States
St. Jude Children's Research Hospital
Memphis, Tennessee, United States
The Children's Hospital at TriStar Centennial
Nashville, Tennessee, United States
Medical College of Wisconsin
Milwaukee, Wisconsin, United States