Recruiting

BEAM-101

Sponsor:

Beam Therapeutics Inc.

Code:

NCT07373639

Conditions

Hemoglobinopathy

Sickle Cell Disease

Eligibility Criteria

Sex: All

Age: 14 - 37

Healthy Volunteers: Not accepted

Interventions

Long-Term Follow-up Study of patients who received BEAM-101

Study Details

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).

Conditions

Hemoglobinopathy

Sickle Cell Disease

Study ID

NCT07373639

Start date

Dec 15, 2025

Status verified date

Nov, 2025

Completion date

Feb 12, 2043

Anticipated

Primary completion date

Feb 12, 2043

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 14 - 37

Healthy Volunteers: Not accepted

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 Design

Enrollment

50 participants

Anticipated

Interventions and Outcome Measures

Arms

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

Interventions

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 measure

  • Long-Term Safety Monitoring and Mortality Assessment [ Time Frame: Up to 13 years ]

Central Contacts and Locations

Central contacts

Medical Information

+1 857-327-8641clinicalinfo@beamtx.com

Locations

Boston Children's Hospital

Recruiting

Boston, Massachusetts, United States, 02215

Contacts

Medical Information

8573278641clinicalinfo@beamtx.com

More Information

Sponsor

Beam Therapeutics Inc.

Last update posted

Jan 28, 2026

Last verified

Nov, 2025

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Beam Therapeutics Inc. on 2026-01-28.