HemoglobinopathyNCT07373639
A Long-term Follow-up Study in Patients Who Received BEAM-101
An observational study of Hemoglobinopathy and Sickle Cell Disease, run by Beam Therapeutics Inc. at 17 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 14 to 37 years
- Conditions the study is for
- Hemoglobinopathy, Sickle Cell Disease
- What is being tested?
- Long-Term Follow-up Study of patients who received BEAM-101
- Type of treatment
- Injection or infusion
- How many people
- 50 participants
- Where
- 17 sitesUnited States
- Study dates
- 15 Dec 2025to 12 Feb 2043
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 14 to 37 years
- All sexes
- No healthy volunteers
- Child, Adult
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
Who may be able to join
- 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.
Who cannot join
- There are no exclusion criteria for this study.
Trial locations and contacts
17 sites in 1 country. Contact the study team or a site near you to ask about taking part.
17 sites in 1 country
United States17 sites, 17 recruiting
- Not yet recruiting
Children's Healthcare of Atlanta - Aflac Cancer and Blood Disorders Center- Egleston
Atlanta, Georgia, 30329
Medical Information, 8573278641, clinicalinfo@beamtx.com
- Not yet recruiting
University of Alabama at Birmingham
Birmingham, Alabama, 35233
Medical Information, 8573278641, clinicalinfo@beamtx.com
- Recruiting
Boston Children's Hospital
Boston, Massachusetts, 02215
Medical Information, 8573278641, clinicalinfo@beamtx.com
- Not yet recruiting
Medical University of South Carolina
Charleston, South Carolina, 29425
Medical Information, 8573278641, clinicalinfo@beamtx.com
- Not yet recruiting
University Hospitals Clevland Medical Center
Cleveland, Ohio, 44106
Medical Information, 8573278641, clinicalinfo@beamtx.com
- Not yet recruiting
The Clevland Clinic Foundation
Cleveland, Ohio, 44195
Medical Information, 8573278641, clinicalinfo@beamtx.com
- Not yet recruiting
Henry Ford Cancer Center
Detroit, Michigan, 48202
Medical Information, 8573278641, clinicalinfo@beamtx.com
- Not yet recruiting
Hackensack University Medical Center
Hackensack, New Jersey, 07601
Medical Information, 8573278641, clinicalinfo@beamtx.com
- Not yet recruiting
Mayo Clinic Florida
Jacksonville, Florida, 32224
Medical Information, 8573278641, clinicalinfo@beamtx.com
- Not yet recruiting
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
Medical Information, 8573278641, clinicalinfo@beamtx.com
- Not yet recruiting
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
Medical Information, 8573278641, clinicalinfo@beamtx.com
- Not yet recruiting
University of Minnesota
Minneapolis, Minnesota, 55455
Medical Information, 8573278641, clinicalinfo@beamtx.com
- Not yet recruiting
The Children's Hospital at TriStar Centennial
Nashville, Tennessee, 37203
Medical Information, 8573278641, clinicalinfo@beamtx.com
- Not yet recruiting
Columbia University Irving Medical Center
New York, New York, 10032
Medical Information, 8573278641, clinicalinfo@beamtx.com
- Not yet recruiting
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Medical Information, 8573278641, clinicalinfo@beamtx.com
- Not yet recruiting
Phoenix Children's Hospital
Phoenix, Arizona, 85016
Medical Information, 8573278641, clinicalinfo@beamtx.com
- Not yet recruiting
Washington University School of Medicine in St. Louis
St Louis, Missouri, 63110
Medical Information, 8573278641, clinicalinfo@beamtx.com
About this study
Official title: 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).
Read the 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.
- Conditions
- Hemoglobinopathy, Sickle Cell Disease
- Being tested
- Long-Term Follow-up Study of patients who received BEAM-101
- Medical subject headings
- Hemoglobinopathies, Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Long-Term Follow-up Study of patients who received BEAM-101Drug
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
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: Drug: Long-Term Follow-up Study of patients who received BEAM-101 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Up to 13 years
Secondary outcomes (10)
Proportion of patients without severe VOCs overtime
Measured: Through year 15
Duration of being severe VOC-free
Measured: Through year 15
Frequency of RBC transfusions over time
Measured: Through year 15
Proportion (%) of patients requiring treatment for SCD related complications
Measured: Through year 15
Total Hemoglobin (Hb) Concentration (g/dL) over time
Measured: Through year 15
Total Fetal Hemoglobin (HbF) Concentration (%) over time
Measured: Through year 15
Total Sickle Hemoglobin (HbS) Concentration (%)
Measured: Through year 15
Change from baseline in indirect bilirubin (mg/dL) overtime
Measured: Up to year 5
Change from baseline in haptoglobin (mg/dL) over time
Measured: Up to year 5
Change from baseline in reticulocyte (%) count over time
Measured: Up to year 5
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Jan 2026
- Study start15 Dec 2025
- Primary completion12 Feb 2043 (estimated)
- Study completion12 Feb 2043 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HemoglobinopathyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Beam Therapeutics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07373639
- Sponsor's protocol ID
- BTX-101-002
- Status last verified
- Nov 2025
- First submitted
- 6 Jan 2026
- Last updated
- 28 Jan 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Long-term Follow-up Study in Patients With Hemoglobinopathy Who Received Autologous CD34+ Edited Hematopoietic Stem Cells
- ClinicalTrials.gov ID
- NCT07373639
- Acronym / sponsor ID
- BTX-101-002
- Conditions
- Hemoglobinopathy, Sickle Cell Disease
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Beam Therapeutics Inc.Industry (pharma, biotech, devices)
- Study start
- 15 Dec 2025 (actual)
- Primary completion
- 12 Feb 2043 (estimated)
- Study completion
- 12 Feb 2043 (estimated)
- First posted
- 28 Jan 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
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 design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| BTX-101-002 Long-term Follow-up on Patients who received BEAM-101 | – | Drug: Long-Term Follow-up Study of patients who received BEAM-101All participants who received BEAM-101 in the BTX-AUT-001 trial will be asked to participate in this long-term follow-up study |
Who may qualify
- Ages
- 14 to 37 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- 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
Key inclusion criteria (2 listed)
- 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.
Key exclusion criteria (1 listed)
- There are no exclusion criteria for this study.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Long-Term Follow-up Study of patients who received BEAM-101This 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 | Drug | BTX-101-002 Long-term Follow-up on Patients who received BEAM-101 |
Primary outcome
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.
10 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Jan 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
17 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemoglobinopathy and Sickle Cell Disease, run by Beam Therapeutics Inc. at 17 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 28 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 17 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Medical Information (+1 857-327-8641, clinicalinfo@beamtx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Long-Term Safety Monitoring and Mortality Assessment (time frame: Up to 13 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Jan 2026.