AzTrials
AzTrials

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.

Enrolling by invitation

Enrolling by invitation. The study team selects and invites participants, usually from an existing group of patients.

Sites and contacts
Official record

Search all trials

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
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

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

Other trials in United States

Canada2 sites

  • Ottawa Hospital Research Institute

    Ottawa, Ontario, K1H 8L6

  • Princess Margaret Cancer Centre-University Health Network

    Toronto, Ontario, M5G 2M9

Other trials in Canada

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 groups in NCT06363760
Study groupRoleWhat they receive
All participantsAll 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.

  1. Adverse events of special interest (AESI)

    Measured: up to 15 years post EDIT-301 infusion

  2. Adverse events of interest (AEI)

    Measured: up to 15 years post EDIT-301 infusion

  3. All-cause mortality

    Measured: up to 15 years post EDIT-301 infusion

  4. Adverse events (AEs) related to EDIT-301

    Measured: up to 15 years post EDIT-301 infusion

  5. Serious adverse events (SAEs)

    Measured: up to 15 years post EDIT-301 infusion

Secondary outcomes (18)

  1. 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

  2. Proportion of SCD participants with vaso-occlusive events (VOEs) over time post-EDIT-301 infusion

    Measured: up to 15 years post EDIT-301 infusion

  3. Frequency of SCD-related transfusions over time post-EDIT-301 infusion for SCD participants

    Measured: up to 15 years post EDIT-301 infusion

  4. Proportion of TDT participants with sustained transfusion independence (TI) over time

    Measured: up to 15 years post EDIT-301 infusion

  5. Proportion of TDT participants with sustained transfusion reduction (TR) over time

    Measured: up to 15 years post EDIT-301 infusion

  6. Frequency of TDT-related transfusions over time post-EDIT-301 infusion for TDT participants

    Measured: up to 15 years post EDIT-301 infusion

  7. 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

  8. SCD and TDT: Total hemoglobin (Hb) concentration (g/dL) over time

    Measured: up to 15 years post EDIT-301 infusion

  9. 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

  10. Average HbF (pg) per RBC over time for SCD participants

    Measured: up to 15 years post EDIT-301 infusion

  11. Complete Blood Count (CBC) red cell indices assay over time for SCD participants

    Measured: up to 15 years post EDIT-301 infusion

  12. Average HbF (pg) per F-cell over time for SCD participants

    Measured: up to 15 years post EDIT-301 infusion

  13. HbF and HbS concentration (g/dL) over time for SCD participants

    Measured: up to 15 years post EDIT-301 infusion

  14. 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

  15. 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

  16. HbF concentration (g/dL) over time for TDT participants

    Measured: up to 15 years post EDIT-301 infusion

  17. 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

  18. 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.

  1. First posted12 Apr 2024
  2. Study start17 Jun 2024
  3. Primary completionAug 2040 (estimated)
  4. Study completionAug 2040 (estimated)

Resources

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)
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

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Keep searching with a free account

You’ve used your 3 free searches for this session. Log in or create a free AzTrials account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in