Status:
ACTIVE_NOT_RECRUITING
A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY)
Lead Sponsor:
Editas Medicine, Inc.
Conditions:
Sickle Cell Disease
Hemoglobinopathies
Eligibility:
All Genders
12-50 years
Phase:
PHASE1
PHASE2
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).
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 ...
Eligibility Criteria
Inclusion
- Key
- 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
- 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
Key Trial Info
Start Date :
May 4 2021
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
August 1 2025
Estimated Enrollment :
45 Patients enrolled
Trial Details
Trial ID
NCT04853576
Start Date
May 4 2021
End Date
August 1 2025
Last Update
January 31 2025
Active Locations (24)
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1
UCSF Benioff Children's Hospital
Oakland, California, United States, 94609
2
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
3
Smilow Cancer Hospital
New Haven, Connecticut, United States, 06511
4
Johns Hopkins All Children's Hospital
St. Petersburg, Florida, United States, 33701