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 |
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Recruitment Status :
Recruiting
First Posted : April 21, 2021
Last Update Posted : July 19, 2023
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Sponsor:
Editas Medicine, Inc.
Information provided by (Responsible Party):
Editas Medicine, Inc.
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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 participants with severe sickle cell disease (SCD).
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Sickle Cell Disease Hemoglobinopathies | Genetic: EDIT-301 | Phase 1 Phase 2 |
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.
| 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
MedlinePlus Genetics related topics:
Sickle cell disease
| Arm | Intervention/treatment |
|---|---|
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Experimental: EDIT-301
EDIT-301 (autologous gene edited (CD)34+ hematopoietic stem cells) will be administered as a one-time intravenous infusion.
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Genetic: EDIT-301
Administered by IV infusion after myeloablative conditioning with busulfan. |
Primary Outcome Measures :
- 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 :
- Complete resolution of severe VOE [ Time Frame: up to 2 years post EDIT-301 infusion ]
- Change from baseline in annualized rate of severe VOE by at least 90% [ Time Frame: up to 2 years post EDIT-301 infusion ]
- Change from baseline in annualized rate of severe VOE by at least 75% [ Time Frame: up to 2 years post EDIT-301 infusion ]
- Change from baseline in annualized rate of severe VOE by at least 50% [ Time Frame: up to 2 years post EDIT-301 infusion ]
- Difference (pre-treatment vs. post-treatment) in annualized rates of severe VOEs [ Time Frame: up to 2 years post EDIT-301 infusion ]
- Change from baseline in annualized rate of hospitalization for severe VOE [ Time Frame: up to 2 years post EDIT-301 infusion ]
- Proportion of subjects with sustained HbF ≥ 20% (HbF/Hb) compared with pre-conditioning Baseline [ Time Frame: up to 2 years post EDIT-301 infusion ]
- Proportion of subjects with mean HbF ≥ 30% (HbF/Hb) compared with pre-conditioning Baseline [ Time Frame: up to 2 years post EDIT-301 infusion ]
- Proportion of subjects with mean total Hb ≥ 10 g/dL compared with Baseline [ Time Frame: up to 2 years post EDIT-301 infusion ]
- Proportion of subjects with mean total Hb increase from baseline ≥ 2 g/dL [ Time Frame: up to 2 years post EDIT-301 infusion ]
- 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 ]
- Change from baseline in HbF concentration (g/dL) [ Time Frame: up to 2 years post EDIT-301 infusion ]
- Change from baseline in total Hb concentration (g/dL) [ Time Frame: up to 2 years post EDIT-301 infusion ]
- Change from baseline in markers of hemolysis (reticulocyte count, indirect bilirubin, lactate dehydrogenase, haptoglobin) [ Time Frame: up to 2 years post EDIT-301 infusion ]
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| Ages Eligible for Study: | 18 Years to 50 Years (Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
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
Contacts
| Contact: Editas Medicine's Clinical Trial Team | 617-401-9007 | Patients@editasmed.com |
Locations
Show 23 study locations
Sponsors and Collaborators
Editas Medicine, Inc.
| 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: | July 19, 2023 |
| Last Verified: | July 2023 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | No |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
Keywords provided by Editas Medicine, Inc.:
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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 |