Sickle Cell DiseaseNCT06363760
A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301
An observational study of Sickle Cell Disease and Transfusion-dependent Beta-Thalassemia, run by Editas Medicine, Inc. at 18 sites in 2 countries.
Trial summary
The purpose of this study is to evaluate the long-term safety and efficacy of EDIT-301 in participants with severe sickle cell disease (SCD) or transfusion-dependent β-thalassemia (TDT) who have received EDIT-301. Read more about this study
- Who is it for?
- Ages 12 to 50 years
- Conditions the study is for
- Sickle Cell Disease, Transfusion-dependent Beta-Thalassemia, Hemoglobinopathies
- What is being tested?
- Safety and efficacy assessments
- How many people
- 54 participants
- Where
- 18 sitesin 2 countries
- Study dates
- 17 Jun 2024to Aug 2040
- 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 12 to 50 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: The study population will be subjects currently enrolled in any one of the two parent studies ( EM-SCD-301-001 or EM-301-BThal-001)
Who may be able to join
- Participant must have received an EDIT-301 infusion as part of a clinical study.
- Participant or legal representative/guardian (if applicable) must sign and date the informed consent form (ICF) or assent, if applicable for this long-term follow-up study.
Who cannot join
- Participant is still actively enrolled in an EDIT-301 treatment study and has not yet met eligibility for long term follow-up in this study.
Trial locations and contacts
18 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
18 sites in 2 countries
United States16 sites
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322
Children's Hospital Colorado
Aurora, Colorado, 80045
Medical University of South Carolina
Charleston, South Carolina, 29425
Atrium Health
Charlotte, North Carolina, 28204
Cleveland Clinic
Cleveland, Ohio, 44195
Nationwide Children's Hospital
Columbus, Ohio, 43205
Texas Oncology - Baylor Charles A. Sammons Cancer Center
Dallas, Texas, 75246
Cook Children's
Fort Worth, Texas, 76104
University of Minnesota
Minneapolis, Minnesota, 55410
Tristar Medical Group Children's Specialists/Sarah Cannon Center for Blood Cancers
Nashville, Tennessee, 37203
Smilow Cancer Hospital
New Haven, Connecticut, 06511
Columbia University Medical Center - Department of Pediatrics
New York, New York, 10032
Columbia University Medical Center
New York, New York, 10032
UCSF Benioff Children's Hospital
Oakland, California, 94609
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Johns Hopkins All Children's Hospital
St. Petersburg, Florida, 33701
Canada2 sites
Ottawa Hospital Research Institute
Ottawa, Ontario, K1H 8L6
Princess Margaret Cancer Centre-University Health Network
Toronto, Ontario, M5G 2M9
About this study
The purpose of this study is to evaluate the long-term safety and efficacy of EDIT-301 in participants with severe sickle cell disease (SCD) or transfusion-dependent β-thalassemia (TDT) who have received EDIT-301.
Read the detailed description
This is a non-interventional, multicenter study evaluating the long-term safety and efficacy of EDIT-301 in participants with severe sickle cell disease (SCD) or transfusion dependent b-thalassemia (TDT) who received EDIT-301 in parent studies EM-SCD-301-001 (NCT04853576) or EDIT-301-BThal-001 (NCT05444894). No investigational drug product will be administered in the LTFU study.
- Being tested
- Safety and efficacy assessments
- Keywords
- Sickle Cell Disease, Sickle Cell Anemia, Sickling Disorder Due to Hemoglobin S, Beta-Thalassemia, Thalassemia Major, Thalassemia Intermedia, Hemoglobinopathies, CRISPR-Cas12a, Autologous CD34+, Genetic Diseases, Inborn, Hematologic Diseases, Anemia, Anemia, Hemolytic, Congenital, Anemia, Hemolytic
- Medical subject headings
- Anemia, Sickle Cell, Hemoglobinopathies, beta-Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases, Anemia, Anemia, Hemolytic, Congenital, Anemia, Hemolytic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Safety and efficacy assessmentsOther
Assessed throughout the duration of the study.
| Study group | Role | What they receive |
|---|---|---|
| All participants | All participants who complete or discontinue one of the parent studies (EM-SCD-301-001 or EDIT-301-BThal-001) Receives: Other: Safety and efficacy assessments |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse events of special interest (AESI)
Measured: up to 15 years post EDIT-301 infusion
Adverse events of interest (AEI)
Measured: up to 15 years post EDIT-301 infusion
All-cause mortality
Measured: up to 15 years post EDIT-301 infusion
Adverse events (AEs) related to EDIT-301
Measured: up to 15 years post EDIT-301 infusion
Serious adverse events (SAEs)
Measured: up to 15 years post EDIT-301 infusion
Secondary outcomes (18)
Proportion of SCD participants with severe vaso-occlusive events (sVOEs) over time post-EDIT-301 infusion
Measured: up to 15 years post EDIT-301 infusion
Proportion of SCD participants with vaso-occlusive events (VOEs) over time post-EDIT-301 infusion
Measured: up to 15 years post EDIT-301 infusion
Frequency of SCD-related transfusions over time post-EDIT-301 infusion for SCD participants
Measured: up to 15 years post EDIT-301 infusion
Proportion of TDT participants with sustained transfusion independence (TI) over time
Measured: up to 15 years post EDIT-301 infusion
Proportion of TDT participants with sustained transfusion reduction (TR) over time
Measured: up to 15 years post EDIT-301 infusion
Frequency of TDT-related transfusions over time post-EDIT-301 infusion for TDT participants
Measured: up to 15 years post EDIT-301 infusion
SCD and TDT: Percent of peripheral red blood cells (RBCs) containing HbF (F-cells) over time
Measured: up to 15 years post EDIT-301 infusion
SCD and TDT: Total hemoglobin (Hb) concentration (g/dL) over time
Measured: up to 15 years post EDIT-301 infusion
SCD and TDT: Proportion of alleles with intended genetic modification present in peripheral blood nucleated cells and bone marrow derived CD34+ over time
Measured: up to 15 years post EDIT-301 infusion
Average HbF (pg) per RBC over time for SCD participants
Measured: up to 15 years post EDIT-301 infusion
Complete Blood Count (CBC) red cell indices assay over time for SCD participants
Measured: up to 15 years post EDIT-301 infusion
Average HbF (pg) per F-cell over time for SCD participants
Measured: up to 15 years post EDIT-301 infusion
HbF and HbS concentration (g/dL) over time for SCD participants
Measured: up to 15 years post EDIT-301 infusion
The level of HbF production determined by fractionation of different forms of Hb (including but not limited to HbA, HbA2, HbC, HbD, HbE, and HbS) over time for SCD participants.
Measured: up to 15 years post EDIT-301 infusion
Percentage of HbF over total non-transfused total Hb (non-transfused total Hb refers to the total g/dL of all Hb variants, excluding HbA) over time for TDT participants.
Measured: up to 15 years post EDIT-301 infusion
HbF concentration (g/dL) over time for TDT participants
Measured: up to 15 years post EDIT-301 infusion
Iron overload of TDT participants
As measured by a) Liver iron concentration (LIC) as assessed by R2* magnetic resonance imaging (MRI) for the first 3 years in this study; b) Cardiac iron concentration (CIC) as assessed by T2* MRI for the first 3 years of this study; c) Serum ferritin levels over time.
Measured: up to 15 years post EDIT-301 infusion
Proportion of TDT participants receiving iron chelation therapy over time.
Measured: up to 15 years post EDIT-301 infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Apr 2024
- Study start17 Jun 2024
- Primary completionAug 2040 (estimated)
- Study completionAug 2040 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell DiseaseStudies 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
- Other
- Time perspective
- Prospective
- Enrolment
- 54 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Editas Medicine, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06363760
- Sponsor's protocol ID
- EDIT-301-LTFU-001
- Status last verified
- Mar 2025
- First submitted
- 9 Apr 2024
- Last updated
- 2 Apr 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301
- ClinicalTrials.gov ID
- NCT06363760
- Acronym / sponsor ID
- EDIT-301-LTFU-001
- Keywords
- Sickle Cell Disease, Sickle Cell Anemia, Sickling Disorder Due to Hemoglobin S, Beta-Thalassemia, Thalassemia Major, Thalassemia Intermedia, Hemoglobinopathies, CRISPR-Cas12a
- Study type
- Observational
- Recruitment status
- Enrolling by invitation
- Enrolment
- 54 participants (estimated)
- Lead sponsor
- Editas Medicine, Inc.Industry (pharma, biotech, devices)
- Study start
- 17 Jun 2024 (actual)
- Primary completion
- Aug 2040 (estimated)
- Study completion
- Aug 2040 (estimated)
- First posted
- 12 Apr 2024 (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.
The purpose of this study is to evaluate the long-term safety and efficacy of EDIT-301 in participants with severe sickle cell disease (SCD) or transfusion-dependent β-thalassemia (TDT) who have received EDIT-301.
Detailed description
This is a non-interventional, multicenter study evaluating the long-term safety and efficacy of EDIT-301 in participants with severe sickle cell disease (SCD) or transfusion dependent b-thalassemia (TDT) who received EDIT-301 in parent studies EM-SCD-301-001 (NCT04853576) or EDIT-301-BThal-001 (NCT05444894). No investigational drug product will be administered in the LTFU study.
Study design
- Observational model
- Other
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| All participants | – | Other: Safety and efficacy assessmentsAll participants who complete or discontinue one of the parent studies (EM-SCD-301-001 or EDIT-301-BThal-001) |
Who may qualify
- Ages
- 12 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- The study population will be subjects currently enrolled in any one of the two parent studies ( EM-SCD-301-001 or EM-301-BThal-001)
Key inclusion criteria (2 listed)
- Participant must have received an EDIT-301 infusion as part of a clinical study.
- Participant or legal representative/guardian (if applicable) must sign and date the informed consent form (ICF) or assent, if applicable for this long-term follow-up study.
Key exclusion criteria (1 listed)
- Participant is still actively enrolled in an EDIT-301 treatment study and has not yet met eligibility for long term follow-up in 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 |
|---|---|---|
| Safety and efficacy assessmentsAssessed throughout the duration of the study. | Other | All participants |
Primary outcome
Adverse events of special interest (AESI)
Time frame: up to 15 years post EDIT-301 infusion
Adverse events of interest (AEI)
Time frame: up to 15 years post EDIT-301 infusion
All-cause mortality
Time frame: up to 15 years post EDIT-301 infusion
Adverse events (AEs) related to EDIT-301
Time frame: up to 15 years post EDIT-301 infusion
Serious adverse events (SAEs)
Time frame: up to 15 years post EDIT-301 infusion
18 secondary outcomes
Proportion of SCD participants with severe vaso-occlusive events (sVOEs) over time post-EDIT-301 infusion
Time frame: up to 15 years post EDIT-301 infusion
Proportion of SCD participants with vaso-occlusive events (VOEs) over time post-EDIT-301 infusion
Time frame: up to 15 years post EDIT-301 infusion
Frequency of SCD-related transfusions over time post-EDIT-301 infusion for SCD participants
Time frame: up to 15 years post EDIT-301 infusion
Proportion of TDT participants with sustained transfusion independence (TI) over time
Time frame: up to 15 years post EDIT-301 infusion
Proportion of TDT participants with sustained transfusion reduction (TR) over time
Time frame: up to 15 years post EDIT-301 infusion
Frequency of TDT-related transfusions over time post-EDIT-301 infusion for TDT participants
Time frame: up to 15 years post EDIT-301 infusion
SCD and TDT: Percent of peripheral red blood cells (RBCs) containing HbF (F-cells) over time
Time frame: up to 15 years post EDIT-301 infusion
SCD and TDT: Total hemoglobin (Hb) concentration (g/dL) over time
Time frame: up to 15 years post EDIT-301 infusion
SCD and TDT: Proportion of alleles with intended genetic modification present in peripheral blood nucleated cells and bone marrow derived CD34+ over time
Time frame: up to 15 years post EDIT-301 infusion
Average HbF (pg) per RBC over time for SCD participants
Time frame: up to 15 years post EDIT-301 infusion
Complete Blood Count (CBC) red cell indices assay over time for SCD participants
Time frame: up to 15 years post EDIT-301 infusion
Average HbF (pg) per F-cell over time for SCD participants
Time frame: up to 15 years post EDIT-301 infusion
HbF and HbS concentration (g/dL) over time for SCD participants
Time frame: up to 15 years post EDIT-301 infusion
The level of HbF production determined by fractionation of different forms of Hb (including but not limited to HbA, HbA2, HbC, HbD, HbE, and HbS) over time for SCD participants.
Time frame: up to 15 years post EDIT-301 infusion
Percentage of HbF over total non-transfused total Hb (non-transfused total Hb refers to the total g/dL of all Hb variants, excluding HbA) over time for TDT participants.
Time frame: up to 15 years post EDIT-301 infusion
HbF concentration (g/dL) over time for TDT participants
Time frame: up to 15 years post EDIT-301 infusion
Iron overload of TDT participants
Time frame: up to 15 years post EDIT-301 infusion
Proportion of TDT participants receiving iron chelation therapy over time.
Time frame: up to 15 years post EDIT-301 infusion
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 2 Apr 2025.
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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.
The record lists no contact. Use the official record to reach the sponsor.
18 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Sickle Cell Disease and Transfusion-dependent Beta-Thalassemia, run by Editas Medicine, Inc. at 18 sites in 2 countries.
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: enrolling by invitation, as of the sponsor's last update on 2 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 18 sites in United States, Canada. 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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Adverse events of special interest (AESI) (time frame: up to 15 years post EDIT-301 infusion). The study lists 5 primary outcomes in all. 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 2 Apr 2025.