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).
Patients who were treated with BEAM-101 in Study BTX-AUT-001 will be asked to sign an informed consent form (ICF) to enter this LTFU study (during the EOS/Month 24 visit for BTX-AUT-001). Patients in this study will have periodic safety and efficacy assessments per the schedule of assessments (SoA). Visits will occur annually through Year 5 following BEAM-101 administration and then every 3 years through Year 11, with a final visit at Year 15. Virtual/phone call check-in visits will occur every 6 months through Year 5 and then annually thereafter.
Study Type
OBSERVATIONAL
Enrollment
50
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
University of Alabama at Birmingham
Birmingham, Alabama, United States
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.
Time frame: Up to 13 years
Proportion of patients without severe VOCs overtime
Time frame: Through year 15
Duration of being severe VOC-free
Time frame: Through year 15
Frequency of RBC transfusions over time
Time frame: Through year 15
Proportion (%) of patients requiring treatment for SCD related complications
Time frame: Through year 15
Total Hemoglobin (Hb) Concentration (g/dL) over time
Time frame: Through year 15
Total Fetal Hemoglobin (HbF) Concentration (%) over time
Time frame: Through year 15
Total Sickle Hemoglobin (HbS) Concentration (%)
Time frame: Through year 15
Change from baseline in indirect bilirubin (mg/dL) overtime
Time frame: Up to year 5
Change from baseline in haptoglobin (mg/dL) over time
Time frame: Up to year 5
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Phoenix Children's Hospital
Phoenix, Arizona, United States
NOT_YET_RECRUITINGMayo Clinic Florida
Jacksonville, Florida, United States
NOT_YET_RECRUITINGChildren's Healthcare of Atlanta - Aflac Cancer and Blood Disorders Center- Egleston
Atlanta, Georgia, United States
NOT_YET_RECRUITINGBoston Children's Hospital
Boston, Massachusetts, United States
RECRUITINGHenry Ford Cancer Center
Detroit, Michigan, United States
NOT_YET_RECRUITINGUniversity of Minnesota
Minneapolis, Minnesota, United States
NOT_YET_RECRUITINGWashington University School of Medicine in St. Louis
St Louis, Missouri, United States
NOT_YET_RECRUITINGHackensack University Medical Center
Hackensack, New Jersey, United States
NOT_YET_RECRUITINGColumbia University Irving Medical Center
New York, New York, United States
NOT_YET_RECRUITING...and 7 more locations
Change from baseline in reticulocyte (%) count over time
Time frame: Up to year 5