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NCT Number: NCT07373639

A Long-term Follow-up Study in Patients Who Received BEAM-101

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).

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Key information

Conditions

Hemoglobinopathy Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Anemia, Sickle Cell Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies Sickle Cell Disease

Age range

14 year–37 year

Sex eligibility

All sexes

Study type

Observational

Primary location

University of Alabama at Birmingham, Birmingham, Alabama, United States

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

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

Primary outcomes

  1. 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

  1. Proportion of patients without severe VOCs overtime

    Time frame: Through year 15

  2. Duration of being severe VOC-free

    Time frame: Through year 15

  3. Frequency of RBC transfusions over time

    Time frame: Through year 15

  4. Proportion (%) of patients requiring treatment for SCD related complications

    Time frame: Through year 15

  5. Total Hemoglobin (Hb) Concentration (g/dL) over time

    Time frame: Through year 15

  6. Total Fetal Hemoglobin (HbF) Concentration (%) over time

    Time frame: Through year 15

  7. Total Sickle Hemoglobin (HbS) Concentration (%)

    Time frame: Through year 15

  8. Change from baseline in indirect bilirubin (mg/dL) overtime

    Time frame: Up to year 5

  9. Change from baseline in haptoglobin (mg/dL) over time

    Time frame: Up to year 5

  10. Change from baseline in reticulocyte (%) count over time

    Time frame: Up to year 5

Study contacts

Contact information is provided by the study sponsor or research team.

Medical Information

CONTACT

[email protected]

+1 857-327-8641

Sponsors and collaborators

Lead sponsor

Beam Therapeutics Inc.

Industry

Registry information

Official study title

A Long-term Follow-up Study in Patients With Hemoglobinopathy Who Received Autologous CD34+ Edited Hematopoietic Stem Cells

Important dates

Study start
2025
Primary completion
2043
Study completion
2043
First posted
Jan 28, 2026
Registry last updated
Jan 28, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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