An observational study in Hemoglobinopathy and Sickle Cell Disease, sponsored by Beam Therapeutics Inc.. Recruiting at 17 sites in United States. Open to participants aged 14 Years to 37 Years. Per ClinicalTrials.gov, last updated 2026-01-28.
Sponsored by Beam Therapeutics Inc. · Observational
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.
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
Exclusion Criteria:
All participants who received BEAM-101 in the BTX-AUT-001 trial will be asked to participate in this long-term follow-up study
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
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
Change from baseline in reticulocyte (%) count over time
Time frame: Up to year 5
Plan to share: No
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Beam Therapeutics Inc.