A Long-term Follow-up Study in Patients Who Received BEAM-101
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Long-Term Follow-up Study of patients who received BEAM-101.
- Who it may be relevant to
- Registry conditions: Hemoglobinopathy, Sickle Cell Disease. Basic parameters: 14 years — 37 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Long-term Follow-up Study in Patients With Hemoglobinopathy Who Received Autologous CD34+ Edited Hematopoietic Stem Cells
Overview
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
- 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
Primary outcome measures
- Long-Term Safety Monitoring and Mortality Assessment [Time frame: Up to 13 years]
Secondary outcome measures (10)
- 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]
Eligibility criteria
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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Case-only
Study locations
United States · 17 centers
- University of Alabama at Birmingham — Birmingham
- Phoenix Children's Hospital — Phoenix
- Mayo Clinic Florida — Jacksonville
- Children's Healthcare of Atlanta - Aflac Cancer and Blood Disorders Center- Egleston — Atlanta
- Boston Children's Hospital — Boston
- Henry Ford Cancer Center — Detroit
- University of Minnesota — Minneapolis
- Washington University School of Medicine in St. Louis — St Louis
- … and 9 more centers
Identifiers
NCT: NCT07373639 · BTX-101-002