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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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 |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Long-Term Follow-up Study of patients who received BEAM-101 | Drug | 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Up to 13 years |
| Measure | Description | 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 |
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Inclusion Criteria:
Exclusion Criteria:
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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
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Medical Information | Contact | +1 857-327-8641 | clinicalinfo@beamtx.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Alabama at Birmingham | Not yet recruiting | Birmingham | Alabama | 35233 | United States |
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| 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 |
| Phoenix Children's Hospital | Not yet recruiting | Phoenix | Arizona | 85016 | United States |
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| Mayo Clinic Florida | Not yet recruiting | Jacksonville | Florida | 32224 | United States |
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| Children's Healthcare of Atlanta - Aflac Cancer and Blood Disorders Center- Egleston | Not yet recruiting | Atlanta | Georgia | 30329 | United States |
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| Boston Children's Hospital | Recruiting | Boston | Massachusetts | 02215 | United States |
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| Henry Ford Cancer Center | Not yet recruiting | Detroit | Michigan | 48202 | United States |
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| University of Minnesota | Not yet recruiting | Minneapolis | Minnesota | 55455 | United States |
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| Washington University School of Medicine in St. Louis | Not yet recruiting | St Louis | Missouri | 63110 | United States |
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| Hackensack University Medical Center | Not yet recruiting | Hackensack | New Jersey | 07601 | United States |
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| Columbia University Irving Medical Center | Not yet recruiting | New York | New York | 10032 | United States |
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| University Hospitals Clevland Medical Center | Not yet recruiting | Cleveland | Ohio | 44106 | United States |
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| The Clevland Clinic Foundation | Not yet recruiting | Cleveland | Ohio | 44195 | United States |
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| Children's Hospital of Philadelphia | Not yet recruiting | Philadelphia | Pennsylvania | 19104 | United States |
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| Medical University of South Carolina | Not yet recruiting | Charleston | South Carolina | 29425 | United States |
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| St. Jude Children's Research Hospital | Not yet recruiting | Memphis | Tennessee | 38105 | United States |
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| The Children's Hospital at TriStar Centennial | Not yet recruiting | Nashville | Tennessee | 37203 | United States |
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| Medical College of Wisconsin | Not yet recruiting | Milwaukee | Wisconsin | 53226 | United States |
|
| ID | Term |
|---|---|
| D006453 | Hemoglobinopathies |
| D000755 | Anemia, Sickle Cell |
| ID | Term |
|---|---|
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
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