A Long-term Follow-up Study in Patients With Hemoglobinopathy Who Received Autologous CD34+ Edited Hematopoietic Stem Cells
Overview
- Phase
- Not Applicable
- Status
- Recruiting
- Sponsor
- Beam Therapeutics Inc.
- Enrollment
- 50
- Locations
- 17
- Primary Endpoint
- Long-Term Safety Monitoring and Mortality Assessment
Overview
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).
Detailed Description
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 Design
- Study Type
- Observational
- Observational Model
- Case Only
- Time Perspective
- Prospective
Eligibility Criteria
- Ages
- 14 Years to 37 Years (Child, Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •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.
Exclusion Criteria
- •There are no exclusion criteria for this study.
Arms & Interventions
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
Intervention: Long-Term Follow-up Study of patients who received BEAM-101 (Drug)
Outcomes
Primary Outcomes
Long-Term Safety Monitoring and Mortality Assessment
Time Frame: Up to 13 years
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.
Secondary Outcomes
- 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)
- Total Fetal Hemoglobin (HbF) Concentration (%) over time(Through year 15)
- Total Sickle Hemoglobin (HbS) Concentration (%)(Through year 15)
- Change from baseline in indirect bilirubin (mg/dL) overtime(Up to year 5)
- Change from baseline in haptoglobin (mg/dL) over time(Up to year 5)
- Change from baseline in reticulocyte (%) count over time(Up to year 5)