Hemoglobinopathy, Sickle Cell Disease
Conditions
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.
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
Sponsors
Beam Therapeutics Inc.
Study design
Observational model
CASE_ONLY
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
14 Years to 37 Years
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Long-Term Safety Monitoring and Mortality Assessment | 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
| Measure | Time frame |
|---|---|
| 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 |
Countries
United States
Contacts
CONTACTMedical Information
Outcome results
None listed