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EDIT-301 for Autologous HSCT in Subjects With Severe Sickle Cell Disease

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ClinicalTrials.gov Identifier: NCT04853576
Recruitment Status : Recruiting
First Posted : April 21, 2021
Last Update Posted : June 15, 2022
Information provided by (Responsible Party):
Editas Medicine, Inc.

Brief Summary:
The purpose of this study is to evaluate the efficacy, safety and tolerability of treatment with EDIT-301 in adult subjects with severe sickle cell disease (SCD).

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Hemoglobinopathies Genetic: EDIT-301 Phase 1 Phase 2

Detailed Description:
This is a Phase 1/2 single-arm, open-label, multicenter study evaluating the safety and efficacy of a single unit dose of EDIT-301 for autologous hematopoietic stem cell transplant (HSCT) in subjects with severe SCD. Planned study subjects will be comprised of male and female adult subjects with severe SCD, from 18 to 50 years of age, inclusive.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 40 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 1/2 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous Clustered Regularly Interspaced Short Palindromic Repeats Gene-edited CD34+ Human Hematopoietic Stem and Progenitor Cells (EDIT-301) in Subjects With Severe Sickle Cell Disease
Actual Study Start Date : May 4, 2021
Estimated Primary Completion Date : August 2025
Estimated Study Completion Date : August 2025

Resource links provided by the National Library of Medicine

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 IV infusion after myeloablative conditioning with busulfan.

Primary Outcome Measures :
  1. Difference (pre-treatment versus post-treatment) in the rates of severe vaso-occlusive events (VOEs) requiring medical attention. [ Time Frame: up to 2 years post EDIT-301 infusion ]

Secondary Outcome Measures :
  1. Proportion of subjects with mean HbF > 20% (HbF/Hb) compared with pre-conditioning Baseline [ Time Frame: up to 2 years post EDIT-301 infusion ]
  2. Proportion of subjects with mean Hb ≥ 10 g/dL starting ≥ 60 days after last packed red blood cell (pRBC) transfusion compared with preconditioning Baseline [ Time Frame: up to 2 years post EDIT-301 infusion ]
  3. Annualized number of units of pRBC transfused for SCD-related indications [ Time Frame: up to 2 years post EDIT-301 infusion ]
  4. Change from baseline in annualized rate of hospitalization for severe VOE [ Time Frame: up to 2 years post EDIT-301 infusion ]
  5. Change from baseline in annualized rate of severe VOE by at least 75% [ Time Frame: up to 2 years post EDIT-301 infusion ]
  6. Change from baseline in annualized rate of severe VOE by at least 90% [ Time Frame: up to 2 years post EDIT-301 infusion ]
  7. Complete resolution of severe VOE [ Time Frame: up to 2 years post EDIT-301 infusion ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 50 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Key Inclusion Criteria:

Diagnosis of severe sickle cell disease as defined by:

  • Documented severe SCD genotype (βS/βS, βS/β0, or βS/β+)
  • History of at least two severe vaso-occlusive crisis events per year requiring medical attention despite hydroxyurea or other supportive care measures in the two year-period prior to provision of informed consent

Karnofsky Performance Status ≥ 80

Key Exclusion Criteria:

  • Available 10/10 HLA-matched related donor
  • Prior HSCT or contraindications to autologous HSCT
  • Any contraindications to the use of plerixafor during the mobilization of hematopoietic stem cells (HSCs) and any contraindications to the use of busulfan and any other medicinal products required during the myeloablative conditioning, including hypersensitivity to the active substances or to any of the excipients
  • Unable to receive red blood cell (RBC) transfusion for any reason
  • Unable or unwilling to comply with standard of care changes in background medical treatment in preparation of, during, or following HSCT, including and not limited to discontinuation of hydroxyurea, voxelotor, crizanlizumab, or L-glutamine
  • Any history of severe cerebral vasculopathy
  • Inadequate end 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

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): NCT04853576

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

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United States, California
UCSF Benioff Children's Hospital Recruiting
Oakland, California, United States, 94609
United States, Colorado
Children's Hospital Colorado Recruiting
Aurora, Colorado, United States, 80045
United States, Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago Recruiting
Chicago, Illinois, United States, 60611
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, North Carolina
The University of North Carolina at Chapel Hill Recruiting
Chapel Hill, North Carolina, United States, 27599
United States, Ohio
Cleveland Clinic Recruiting
Cleveland, Ohio, United States, 44195
Nationwide Children's Hospital Recruiting
Columbus, Ohio, United States, 43205
United States, Pennsylvania
Children's Hospital of Philadelphia Recruiting
Philadelphia, Pennsylvania, United States, 19104
United States, South Carolina
Medical University of South Carolina Recruiting
Charleston, South Carolina, United States, 29425
United States, Tennessee
Tristar Medical Group Children's Specialists/Sarah Cannon Center for Blood Cancers Recruiting
Nashville, Tennessee, United States, 37203
United States, Texas
Texas Oncology - Baylor Charles A. Sammons Cancer Center Recruiting
Dallas, Texas, United States, 75246
Canada, Ontario
Ottawa Hospital Research Institute Recruiting
Ottawa, Ontario, Canada, K1H 8L6
Canada, Quebec
Centre Hospitalier Universitaire Sainte-Justine Recruiting
Montréal, Quebec, Canada, H3T 1C5
Sponsors and Collaborators
Editas Medicine, Inc.
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Responsible Party: Editas Medicine, Inc.
ClinicalTrials.gov Identifier: NCT04853576    
Other Study ID Numbers: EM-SCD-301-001
First Posted: April 21, 2021    Key Record Dates
Last Update Posted: June 15, 2022
Last Verified: June 2022
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.:
Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Genetic Diseases, Inborn
Cell Disorders, Sickle
HbS Disease
Sickling Disorder Due to Hemoglobin S
Hematologic Diseases
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hematologic Diseases
Genetic Diseases, Inborn