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A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY)

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ClinicalTrials.gov Identifier: NCT04853576
Recruitment Status : Recruiting
First Posted : April 21, 2021
Last Update Posted : February 1, 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 efficacy, safety and tolerability of treatment with EDIT-301 in adult and adolescent participants 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 and adolescent subjects with severe SCD, from 12 to 50 years of age, inclusive.
Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 45 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


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 IV infusion after myeloablative conditioning with busulfan.
Other Names:
  • renizgamglogene autogedtemcel
  • reni-cel


Outcome Measures
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Primary Outcome Measures :
  1. Proportion of subjects achieving complete resolution of severe vaso-occlusive events (VOEs) [ Time Frame: up to 2 years post EDIT-301 infusion ]

Secondary Outcome Measures :
  1. Proportion of subjects achieving complete resolution of VOEs [ Time Frame: up to 2 years post EDIT-301 infusion ]
  2. Proportion of subjects with 90% reduction in annualized rate of severe VOE compared to pre-treatment period [ Time Frame: up to 2 years post EDIT-301 infusion ]
  3. Proportion of subjects with 75% reduction in annualized rate of severe VOE compared to pre-treatment period [ Time Frame: up to 2 years post EDIT-301 infusion ]
  4. Proportion of subjects with 50% reduction in annualized rate of severe VOE compared to pre-treatment period [ Time Frame: up to 2 years post EDIT-301 infusion ]
  5. Difference (pre-treatment vs. post-treatment) in annualized rates of severe VOEs [ Time Frame: up to 2 years post EDIT-301 infusion ]
  6. Difference (pre-treatment vs. post-treatment) in annualized rate of hospitalization for severe VOEs [ Time Frame: up to 2 years post EDIT-301 infusion ]
  7. Proportion of subjects with sustained HbF ≥ 20% (HbF/Hb) compared with baseline [ Time Frame: up to 2 years post EDIT-301 infusion ]
  8. Proportion of subjects with mean HbF ≥ 30% (HbF/Hb) compared with baseline [ Time Frame: up to 2 years post EDIT-301 infusion ]
  9. Proportion of subjects with mean total Hb ≥ 10 g/dL compared with baseline [ Time Frame: up to 2 years post EDIT-301 infusion ]
  10. Proportion of subjects with mean total Hb increase from baseline of ≥ 2 g/dL [ Time Frame: up to 2 years post EDIT-301 infusion ]
  11. Difference (pre-treatment versus post-treatment) in annualized number of units of pRBC transfused for SCD-related indications [ Time Frame: up to 2 years post EDIT-301 infusion ]
  12. Change from baseline in HbF concentration (g/dL) [ Time Frame: up to 2 years post EDIT-301 infusion ]
  13. Change from baseline in total Hb concentration (g/dL) [ Time Frame: up to 2 years post EDIT-301 infusion ]
  14. Change from baseline in markers of hemolysis (absolute reticulocyte count, indirect bilirubin, lactate dehydrogenase, haptoglobin) [ Time Frame: up to 2 years post EDIT-301 infusion ]
  15. Time to neutrophil engraftment (the first day in which 3 consecutive absolute neutrophil count (ANC) ≥ 0.5 x 109/L laboratory values obtained on different days) [ Time Frame: up to 24 months after EDIT-301 infusion ]
  16. Time to platelet engraftment (the first day in which 3 consecutive platelets ≥ 50 x 109/L laboratory values obtained for at least 7 days following the last platelet transfusion and 10 days following any administration of thrombopoietin (TPO) mimetics) [ Time Frame: up to 24 months after EDIT-301 infusion ]
  17. Frequency and severity of adverse events (AEs) [ Time Frame: up to 24 months post EDIT-301 infusion ]

Eligibility Criteria
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Ages Eligible for Study:   12 Years to 50 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

Diagnosis of severe sickle cell disease as defined by:

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

Karnofsky (for subjects >16 years of age) or Lansky (for subjects ≤ 16 years of age) Performance Status ≥ 80%

Normal transcranial doppler velocity in subjects 16 years of age or younger

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

Other protocol defined inclusion/exclusion criteria may apply

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): NCT04853576


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

Locations
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Sponsors and Collaborators
Editas Medicine, Inc.
More Information
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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: February 1, 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.:
Anemia
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
Hemoglobinopathies
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
 Anemia
Hematologic Diseases
Genetic Diseases, Inborn