Health TrialsbyHealthPanel

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

ConditionsHemoglobinopathySickle Cell Disease
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age14-37
SponsorBeam Therapeutics Inc.

About this trial

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).

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

There are no exclusion criteria for this study.

Trial 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

Trial design

Design model

Case-only

Time perspective

Prospective

Treatments tested in this trial

  • 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

Treatment groups

50 Participants
are divided into 1 treatment group
Group A: BTX-101-002 Long-term Follow-up on Patients who received BEAM-1011 intervention

Trial outcomes

Primary outcomes

1

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.

Time frame
Up to 13 years

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

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Beam Therapeutics Inc.

Lead sponsor

Source ClinicalTrials.gov
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