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

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ClinicalTrials.gov Identifier: NCT05444894
Recruitment Status : Recruiting
First Posted : July 6, 2022
Last Update Posted : January 29, 2024
Sponsor:
Information provided by (Responsible Party):
Editas Medicine, Inc.

Study Description
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Brief 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

Condition or disease Intervention/treatment Phase
Transfusion Dependent Beta Thalassemia Hemoglobinopathies Thalassemia Major Thalassemia Intermedia Genetic: EDIT-301 Phase 1 Phase 2

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
Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 9 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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)
Actual Study Start Date : April 29, 2022
Estimated Primary Completion Date : September 2025
Estimated Study Completion Date : December 2025

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: EDIT-301
EDIT-301 (autologous gene edited (CD)34+ hematopoietic stem cells) will be administered as a one-time intravenous infusion.
 Genetic: EDIT-301
Administered by intravenous infusion after myeloablative conditioning with busulfan.
Other Names:
  • renizgamglogene autogedtemcel
  • reni-cel


Outcome Measures
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Primary Outcome Measures :
  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) [ Time Frame: 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) [ Time Frame: Screening through up to 24 months post EDIT-301 infusion ]

Secondary Outcome Measures :
  1. Kinetics of HSPC engraftment [ Time Frame: 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 ]
    Time to neutrophil engraftment

  2. Kinetics of HSPC engraftment [ Time Frame: 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. ]
    Time to platelet engraftment

  3. Incidence of transplant related mortality [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: Baseline through up to 24 months post EDIT-301 infusion ]
  8. Change in the total hemoglobin concentration compared to baseline overtime [ Time Frame: Baseline through up to 24 months post EDIT-301 infusion ]
  9. Proportion of participants with hemoglobin concentration ≥ 9 g/dL [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 3 months through up to 24 months post EDIT-301 infusion ]
  12. Change in parameters of iron overload compared to baseline over time [ Time Frame: Baseline through up to 24 months post EDIT-301 infusion ]
  13. Proportion of participants receiving iron chelation therapy over time [ Time Frame: EDIT-301 infusion (Day 0) through up to 24 months post EDIT-301 infusion ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years to 35 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

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

Key Exclusion Criteria:

  • 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
  • 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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05444894


Contacts
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Contact: Editas Medicine Clinical Trial Team 617-401-9007 Patients@editasmed.com

Locations
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United States, California
University of California San Francisco Recruiting
Oakland, California, United States, 94609
United States, Minnesota
University of Minnesota Recruiting
Minneapolis, Minnesota, United States, 55410
United States, New York
Columbia University Medical Center - Department of Pediatrics Recruiting
New York, New York, United States, 10032
Columbia University Medical Center Recruiting
New York, New York, United States, 10032
United States, Ohio
Cleveland Clinic Recruiting
Cleveland, Ohio, United States, 44195
United States, Pennsylvania
Children's Hospital of Philadelphia Recruiting
Philadelphia, Pennsylvania, United States, 19104
United States, Tennessee
Tristar Medical Group Children's Specialists/Sarah Cannon Center for Blood Cancers Recruiting
Nashville, Tennessee, United States, 37203
Canada, Ontario
Princess Margaret Cancer Centre-University Health Network Recruiting
Toronto, Ontario, Canada, M5G 2M9
Sponsors and Collaborators
Editas Medicine, Inc.
More Information
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Responsible Party: Editas Medicine, Inc.
ClinicalTrials.gov Identifier: NCT05444894    
Other Study ID Numbers: EM-301-BThal-001
First Posted: July 6, 2022    Key Record Dates
Last Update Posted: January 29, 2024
Last Verified: January 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Editas Medicine, Inc.:
Beta-Thalassemia
Hemoglobinopathies
CRISPR-Cas 12a
Autologous CD34+
Additional relevant MeSH terms:
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Thalassemia
beta-Thalassemia
Hemoglobinopathies
Anemia, Hemolytic, Congenital
 Anemia, Hemolytic
Anemia
Hematologic Diseases
Genetic Diseases, Inborn