AzTrials
AzTrials

Transfusion Dependent Beta ThalassemiaPhase 1/2NCT05444894

EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)

A Phase 1/2 study testing EDIT-301 in people with Transfusion Dependent Beta Thalassemia and Hemoglobinopathies, run by Editas Medicine, Inc. at 8 sites in 2 countries.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
Search all trials

Trial summary

The purpose of this study is to evaluate the safety, tolerability, and efficacy of treatment with EDIT-301 in adult participants with Transfusion Dependent beta Thalassemia Read more about this study

Who is it for?
Ages 18 to 35 years
What is being tested?
EDIT-301
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
9 participants
Where
8 sitesin 2 countries
Study dates
29 Apr 2022to Dec 2025
Phase
Phase 1/2
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 18 to 35 years
  • All sexes
  • No healthy volunteers
  • Adult

Who may be able to join

  • Diagnosis of Transfusion Dependent B-Thalassemia as defined by:
  • Documented homozygous β-thalassemia or compound heterozygous β-thalassemia including β-thalassemia/hemoglobin E (HbE) based on historical data in medical records, and
  • History of at least 100 mL/kg/year or 10 U/year of packed red blood cell (RBC) transfusions in the 2 years prior to signing informed consent
  • Clinically stable and eligible to undergo autologous HSCT
  • Karnofsky Performance Status ≥ 70

Who cannot join

  • Available 10/10 human leukocyte antigen (HLA)-matched related donor
  • Prior HSCT or contraindications to autologous HSCT
  • Participants with associated a history of α-thalassemia and > 1 alpha chain deletion, or alpha multiplications as documented in medical records
  • Participants with a history of other inherited hemoglobinopathy or thalassemic mutation (Hb S, C, D or other) as documented in medical records
  • Prior receipt of gene therapy
  • Inadequate bone marrow function, as defined by white blood cell count of < 3 x 10^9/L or a platelet count < 100 x 10^9/L (without hypersplenism), per investigator judgement
Show all 11Show fewer
  • Inadequate organ function
  • Advanced liver disease
  • Any prior or current malignancy, or immunodeficiency disorder,
  • Immediate family member with a known or suspected Familial Cancer Syndrome
  • Clinically significant and active bacterial, viral, fungal, or parasitic infection

Trial locations and contacts

8 sites in 2 countries.

8 sites in 2 countries

United States7 sites

  • Cleveland Clinic

    Cleveland, Ohio, 44195

  • University of Minnesota

    Minneapolis, Minnesota, 55410

  • Tristar Medical Group Children's Specialists/Sarah Cannon Center for Blood Cancers

    Nashville, Tennessee, 37203

  • Columbia University Medical Center - Department of Pediatrics

    New York, New York, 10032

  • Columbia University Medical Center

    New York, New York, 10032

  • University of California San Francisco

    Oakland, California, 94609

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104

Other trials in United States

Canada1 site

  • Princess Margaret Cancer Centre-University Health Network

    Toronto, Ontario, M5G 2M9

Other trials in Canada

About this study

Official title: A Multicenter Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Dose of Autologous Clustered Regularly Interspaced Short Palindromic Repeats Gene-edited Cluster of Differentiation 34 (CD34+) Human Hematopoietic Stem and Progenitor Cells (HSPC) (EDIT-301) in Transfusion-Dependent Beta Thalassemia (TDT)

The purpose of this study is to evaluate the safety, tolerability, and efficacy of treatment with EDIT-301 in adult participants with Transfusion Dependent beta Thalassemia

Read the detailed description

This is a Phase 1/2 single-arm, open-label, multicenter study evaluating the safety, tolerability, and efficacy of a single unit dose of EDIT-301 for autologous hematopoietic stem cell transplant in adult participants with TDT, age 18 to 35 years, inclusive

Being tested
EDIT-301
Keywords
Beta-Thalassemia, Hemoglobinopathies, CRISPR-Cas 12a, Autologous CD34+
Medical subject headings
Hemoglobinopathies, beta-Thalassemia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • EDIT-301Genetic

    Also called renizgamglogene autogedtemcel, reni-cel

    Administered by intravenous infusion after myeloablative conditioning with busulfan.

Study groups in NCT05444894
Study groupRoleWhat they receive
EDIT-301ExperimentalEDIT-301 (autologous gene edited (CD)34+ hematopoietic stem cells) will be administered as a one-time intravenous infusion.
Receives: Genetic: EDIT-301

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Proportion of participants achieving engraftment defined as neutrophil engraftment (defined as demonstrating absolute neutrophil count (ANC) ≥ 0.5 x 10^9/L post EDIT-301 infusion for 3 consecutive measurements obtained on different days)

    Measured: EDIT-301 infusion (Day 0) to 42 days post EDIT-301 infusion

  2. Frequency and severity of adverse events (AEs) (incidence of AEs and Grade 3 or higher serious adverse events, using National Cancer Institute Common Terminology Criteria for Adverse Events [NCI CTCAE] v.5.0)

    Measured: Screening through up to 24 months post EDIT-301 infusion

Secondary outcomes (13)

  1. Kinetics of HSPC engraftment

    Time to neutrophil engraftment

    Measured: EDIT-301 infusion (Day 0) to first day in which 3 consecutive measurements obtained on different days demonstrate ANC ≥ 0.5 x 10^9/L up to 24 months post EDIT-301 infusion

  2. Kinetics of HSPC engraftment

    Time to platelet engraftment

    Measured: EDIT-301 infusion (Day 0) to first day of 3 consecutive measurements of platelets ≥ 50 x 10^9/L for at least 1 week following the last platelet transfusion and 10 days following thrombopoietin mimetics use up to 24 months post EDIT-301 infusion.

  3. Incidence of transplant related mortality

    Measured: EDIT-301 infusion (Day 0) through Day 100 post EDIT-301 infusion and from EDIT-301 infusion (Day 0) through 12 months post EDIT-301 infusion

  4. Incidence of all-cause mortality

    Measured: Screening through up to 24 months post EDIT-301 infusion

  5. Proportion of alleles per participant with intended genetic modification present in peripheral blood over time

    Measured: EDIT-301 infusion (Day 0) through up to 24 months post EDIT-301 infusion

  6. Proportion of alleles per participant with intended genetic modification present in bone marrow cells over time

    Measured: EDIT-301 infusion (Day 0) through up to 24 months post EDIT-301 infusion

  7. Change in the fetal hemoglobin (HbF) concentration compared to baseline overtime

    Measured: Baseline through up to 24 months post EDIT-301 infusion

  8. Change in the total hemoglobin concentration compared to baseline overtime

    Measured: Baseline through up to 24 months post EDIT-301 infusion

  9. Proportion of participants with hemoglobin concentration ≥ 9 g/dL

    Measured: EDIT-301 infusion (Day 0) through 3, 6, 12 months up to 24 months post EDIT-301 infusion

  10. Proportion of participants achieving the sustained transfusion reduction (TR) for at least 6 months and at least 12 months from 3 months post-EDIT-301 infusion

    Measured: 3 months post EDIT-301 infusion through up to 24 months post EDIT-301 infusion

  11. Proportion of participants achieving the sustained transfusion independence (TI) for at least 6 months and, at least 12 months from 3 months post EDIT-301 infusion

    Measured: 3 months through up to 24 months post EDIT-301 infusion

  12. Change in parameters of iron overload compared to baseline over time

    Measured: Baseline through up to 24 months post EDIT-301 infusion

  13. Proportion of participants receiving iron chelation therapy over time

    Measured: EDIT-301 infusion (Day 0) through up to 24 months 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 posted6 Jul 2022
  2. Study start29 Apr 2022
  3. Primary completionSep 2025 (estimated)
  4. Study completionDec 2025 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
9 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05444894
Sponsor's protocol ID
EM-301-BThal-001
Status last verified
Mar 2025
First submitted
27 Jun 2022
Last updated
2 Apr 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • Frangoul H, Hanna R, Walters MC, Kao RL, Carroll C, McManus M, Chang KH, Jaskolka MC, Kim K, Yu Q, Badamosi N, Mei B, Afonja O, Thompson A; EdiThal Investigators. CRISPR-Cas12a Gene Editing of HBG1 and HBG2 Promoters to Treat beta-Thalassemia. N Engl J Med. 2026 Apr 2;394(13):1292-1301. doi: 10.1056/NEJMoa2501277.

    PubMed 41931048

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing EDIT-301 in people with Transfusion Dependent Beta Thalassemia and Hemoglobinopathies, run by Editas Medicine, Inc. at 8 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 5 inclusion and 11 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: under way, not looking for participants, 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 8 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.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

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?

Proportion of participants achieving engraftment defined as neutrophil engraftment (defined as demonstrating absolute neutrophil count (ANC) ≥ 0.5 x 10^9/L post EDIT-301 infusion for 3 consecutive measurements obtained on different days) (time frame: EDIT-301 infusion (Day 0) to 42 days post EDIT-301 infusion). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05444894, which also lists 1 linked publication.

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.

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

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in