An observational study in Hemoglobinopathy and Sickle Cell Disease, sponsored by Beam Therapeutics Inc.. Recruiting at 17 sites in United States. Open to participants aged 14 Years to 37 Years. Per ClinicalTrials.gov, last updated 2026-01-28.
Sponsored by Beam Therapeutics Inc. · Observational
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).
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.
1,134 studies on the registry are indexed under Anemia, Sickle Cell; 238 are open to participants now.
This study's planned enrollment of 50 is below the median of 100 across 296 observational studies indexed under Anemia, Sickle Cell.
Browse Anemia, Sickle Cell studies →Beam Therapeutics Inc. is the lead sponsor of 6 studies on the registry; 4 are open to participants now.
Compared within Anemia, Sickle Cell, this study's primary condition as ClinicalTrials.gg classifies it; the registry's first condition term is Hemoglobinopathies. The counts are of registry records indexed under each term, refreshed daily. How we classify →
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
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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
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
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
Plan to share: No
No publications or documents are linked to this record.
Started Dec 2025 (actual). Primary completion is estimated for Feb 2043, 205 months after the start.
Of 139 observational studies under Anemia, Sickle Cell, half reached primary completion within 49 months of starting (the median), counting those still running; a quarter within 24 and three quarters within 109. This study's planned 205 months is above that.
How these figures are worked out, and which studies are left out of them.
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
Contact study teamGet an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Beam Therapeutics Inc.