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

Primary Purpose

Sickle Cell Disease, Hemoglobinopathies

Status

Recruiting

Phase

Phase 1

Locations

International

Study Type

Interventional

Intervention

EDIT-301

About this trial

This is an interventional treatment trial for Sickle Cell Disease focused on measuring 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

Eligibility Criteria

18 Years - 50 Years (Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

UCSF Benioff Children's HospitalRecruiting
Children's Hospital ColoradoRecruiting
Smilow Cancer HospitalRecruiting
Johns Hopkins All Children's HospitalRecruiting
Children's Healthcare of AtlantaRecruiting
Ann & Robert H. Lurie Children's Hospital of ChicagoRecruiting
University of Mississippi Medical CenterRecruiting
Columbia University Medical Center - Department of PediatricsRecruiting
Columbia University Medical CenterRecruiting
The University of North Carolina at Chapel HillRecruiting
Atrium HealthRecruiting
University Hospitals Rainbow Babies & Children's HospitalRecruiting
Cleveland ClinicRecruiting
Nationwide Children's HospitalRecruiting
The James Cancer HospitalRecruiting
Children's Hospital of PhiladelphiaRecruiting
Medical University of South CarolinaRecruiting
Tristar Medical Group Children's Specialists/Sarah Cannon Center for Blood CancersRecruiting
Texas Oncology - Baylor Charles A. Sammons Cancer CenterRecruiting
Cook Children'sRecruiting
Ottawa Hospital Research InstituteRecruiting
Princess Margaret Cancer CentreRecruiting
Centre Hospitalier Universitaire Sainte-JustineRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

EDIT-301

Arm Description

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

Outcomes

Primary Outcome Measures

Proportion of subjects achieving complete resolution of severe vaso-occlusive events (VOEs)

Secondary Outcome Measures

Complete resolution of severe VOE

Change from baseline in annualized rate of severe VOE by at least 90%

Change from baseline in annualized rate of severe VOE by at least 75%

Change from baseline in annualized rate of severe VOE by at least 50%

Difference (pre-treatment vs. post-treatment) in annualized rates of severe VOEs

Change from baseline in annualized rate of hospitalization for severe VOE

Proportion of subjects with sustained HbF ≥ 20% (HbF/Hb) compared with pre-conditioning Baseline

Proportion of subjects with mean HbF ≥ 30% (HbF/Hb) compared with pre-conditioning Baseline

Proportion of subjects with mean total Hb ≥ 10 g/dL compared with Baseline

Proportion of subjects with mean total Hb increase from baseline ≥ 2 g/dL

Difference (pre-treatment versus post-treatment) in annualized number of units of pRBC transfused for SCD-related indications

Change from baseline in HbF concentration (g/dL)

Change from baseline in total Hb concentration (g/dL)

Change from baseline in markers of hemolysis (reticulocyte count, indirect bilirubin, lactate dehydrogenase, haptoglobin)

Full Information

NCT ID

NCT04853576

First Posted

April 16, 2021

Last Updated

July 17, 2023

Sponsor

Editas Medicine, Inc.

1. Study Identification

Unique Protocol Identification Number

NCT04853576

Brief Title

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

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

Study Type

Interventional

2. Study Status

Record Verification Date

July 2023

Overall Recruitment Status

Recruiting

Study Start Date

May 4, 2021 (Actual)

Primary Completion Date

August 2025 (Anticipated)

Study Completion Date

August 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Editas Medicine, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

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).

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Sickle Cell Disease, Hemoglobinopathies

Keywords

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

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1, Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

40 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

EDIT-301

Arm Type

Experimental

Arm Description

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

Intervention Type

Genetic

Intervention Name(s)

EDIT-301

Intervention Description

Administered by IV infusion after myeloablative conditioning with busulfan.

Primary Outcome Measure Information:

Title

Proportion of subjects achieving complete resolution of severe vaso-occlusive events (VOEs)

Time Frame