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

January 23, 2026 updated by: Beam Therapeutics Inc.

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

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).

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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.

Study Type

Observational

Enrollment (Estimated)

50

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35233
        • Not yet recruiting
        • University of Alabama at Birmingham
        • Contact:
    • Arizona
      • Phoenix, Arizona, United States, 85016
        • Not yet recruiting
        • Phoenix Children's Hospital
        • Contact:
    • Florida
      • Jacksonville, Florida, United States, 32224
        • Not yet recruiting
        • Mayo Clinic Florida
        • Contact:
    • Georgia
      • Atlanta, Georgia, United States, 30329
        • Not yet recruiting
        • Children's Healthcare of Atlanta - Aflac Cancer and Blood Disorders Center- Egleston
        • Contact:
    • Massachusetts
      • Boston, Massachusetts, United States, 02215
        • Recruiting
        • Boston Children's Hospital
        • Contact:
    • Michigan
      • Detroit, Michigan, United States, 48202
        • Not yet recruiting
        • Henry Ford Cancer Center
        • Contact:
    • Minnesota
      • Minneapolis, Minnesota, United States, 55455
        • Not yet recruiting
        • University of Minnesota
        • Contact:
    • Missouri
      • St Louis, Missouri, United States, 63110
        • Not yet recruiting
        • Washington University School of Medicine in St. Louis
        • Contact:
    • New Jersey
      • Hackensack, New Jersey, United States, 07601
        • Not yet recruiting
        • Hackensack University Medical Center
        • Contact:
    • New York
      • New York, New York, United States, 10032
        • Not yet recruiting
        • Columbia University Irving Medical Center
        • Contact:
    • Ohio
      • Cleveland, Ohio, United States, 44106
        • Not yet recruiting
        • University Hospitals Clevland Medical Center
        • Contact:
      • Cleveland, Ohio, United States, 44195
        • Not yet recruiting
        • The Clevland Clinic Foundation
        • Contact:
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Not yet recruiting
        • Children's Hospital of Philadelphia
        • Contact:
    • South Carolina
      • Charleston, South Carolina, United States, 29425
        • Not yet recruiting
        • Medical University of South Carolina
        • Contact:
    • Tennessee
      • Memphis, Tennessee, United States, 38105
        • Not yet recruiting
        • St. Jude Children's Research Hospital
        • Contact:
      • Nashville, Tennessee, United States, 37203
        • Not yet recruiting
        • The Children's Hospital at TriStar Centennial
        • Contact:
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Not yet recruiting
        • Medical College of Wisconsin
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

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

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
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
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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
Up to 13 years

Secondary Outcome Measures

Outcome Measure
Time Frame
Proportion of patients without severe VOCs overtime
Time Frame: Through year 15
Through year 15
Duration of being severe VOC-free
Time Frame: Through year 15
Through year 15
Frequency of RBC transfusions over time
Time Frame: Through year 15
Through year 15
Proportion (%) of patients requiring treatment for SCD related complications
Time Frame: Through year 15
Through year 15
Total Hemoglobin (Hb) Concentration (g/dL) over time
Time Frame: Through year 15
Through year 15
Total Fetal Hemoglobin (HbF) Concentration (%) over time
Time Frame: Through year 15
Through year 15
Total Sickle Hemoglobin (HbS) Concentration (%)
Time Frame: Through year 15
Through year 15
Change from baseline in indirect bilirubin (mg/dL) overtime
Time Frame: Up to year 5
Up to year 5
Change from baseline in haptoglobin (mg/dL) over time
Time Frame: Up to year 5
Up to year 5
Change from baseline in reticulocyte (%) count over time
Time Frame: Up to year 5
Up to year 5

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Beam Therapeutics Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 15, 2025

Primary Completion (Estimated)

February 12, 2043

Study Completion (Estimated)

February 12, 2043

Study Registration Dates

First Submitted

January 6, 2026

First Submitted That Met QC Criteria

January 23, 2026

First Posted (Actual)

January 28, 2026

Study Record Updates

Last Update Posted (Actual)

January 28, 2026

Last Update Submitted That Met QC Criteria

January 23, 2026

Last Verified

November 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • BTX-101-002

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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