A Long-term Follow-up Study in Patients With Hemoglobinopathy Who Received Autologous CD34+ Edited Hematopoietic Stem Cells
Beam Therapeutics Inc.
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).
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.
Eligibility
- Age range
- 14–37 years
- Sex
- All
- Healthy volunteers
- No
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.
Interventions
- DrugLong-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 BirminghamBirmingham, Alabama
- Phoenix Children's HospitalPhoenix, Arizona
- Mayo Clinic FloridaJacksonville, Florida
- Children's Healthcare of Atlanta - Aflac Cancer and Blood Disorders Center- EglestonAtlanta, Georgia
- Boston Children's HospitalBoston, Massachusetts
- Henry Ford Cancer CenterDetroit, Michigan