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

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


Lead Sponsor: Editas Medicine, Inc.

Source 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).

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.

Overall Status Recruiting
Start Date 2021-04-16
Completion Date 2025-08-01
Primary Completion Date 2025-08-01
Phase Phase 1/Phase 2
Study Type Interventional
Primary Outcome
Measure Time Frame
Difference (pre-treatment versus post-treatment) in the rates of severe vaso-occlusive events (VOEs) requiring medical attention. up to 2 years post EDIT-301 infusion
Secondary Outcome
Measure Time Frame
Proportion of subjects with mean HbF > 20% (HbF/Hb) compared with pre-conditioning Baseline up to 2 years post EDIT-301 infusion
Proportion of subjects with mean Hb ≥ 10 g/dL starting ≥ 60 days after last packed red blood cell (pRBC) transfusion compared with preconditioning Baseline up to 2 years post EDIT-301 infusion
Annualized number of units of pRBC transfused for SCD-related indications up to 2 years post EDIT-301 infusion
Change from baseline in annualized rate of hospitalization for severe VOE up to 2 years post EDIT-301 infusion
Change from baseline in annualized rate of severe VOE by at least 75% up to 2 years post EDIT-301 infusion
Change from baseline in annualized rate of severe VOE by at least 90% up to 2 years post EDIT-301 infusion
Complete resolution of severe VOE up to 2 years post EDIT-301 infusion
Enrollment 40

Intervention Type: Genetic

Intervention Name: EDIT-301

Description: Administered by IV infusion after myeloablative conditioning with busulfan.

Arm Group Label: EDIT-301



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



Minimum Age:

18 Years

Maximum Age:

50 Years

Healthy Volunteers:


Overall Contact

Last Name: Editas Medicine's Clinical Trial Team

Phone: 617-401-9007

Email: [email protected]

Facility: Status: Cleveland Clinic
Location Countries

United States

Verification Date


Responsible Party

Type: Sponsor

Has Expanded Access No
Condition Browse
Number Of Arms 1
Arm Group

Label: EDIT-301

Type: Experimental

Description: EDIT-301 (autologous gene edited (CD)34+ hematopoietic stem cells) will be administered as a one-time intravenous infusion.

Patient Data No
Study Design Info

Allocation: N/A

Intervention Model: Single Group Assignment

Primary Purpose: Treatment

Masking: None (Open Label)

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