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

January 29, 2025 updated by: Editas Medicine, Inc.

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

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).

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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 Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
45

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • Canada
    • Ontario
      • Ottawa, Ontario, Canada, K1H 8L6
        • Ottawa Hospital Research Institute
      • Toronto, Ontario, Canada, M5G 2M9
        • Princess Margaret Cancer Centre
    • Quebec
      • Montréal, Quebec, Canada, H3T 1C5
        • Centre Hospitalier Universitaire Sainte-Justine
  • United States
    • California
      • Oakland, California, United States, 94609
        • UCSF Benioff Children's Hospital
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Children's Hospital Colorado
    • Connecticut
      • New Haven, Connecticut, United States, 06511
        • Smilow Cancer Hospital
    • Florida
      • Saint Petersburg, Florida, United States, 33701
        • Johns Hopkins All Children's Hospital
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Children's Healthcare of Atlanta
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Ann & Robert H. Lurie Children's Hospital of Chicago
    • Mississippi
      • Jackson, Mississippi, United States, 39216
        • University of Mississippi Medical Center
    • New Jersey
      • Hackensack, New Jersey, United States, 07601
        • Hackensack University Medical Center
    • New York
      • New York, New York, United States, 10032
        • Columbia University Medical Center
      • New York, New York, United States, 10032
        • Columbia University Medical Center - Department of Pediatrics
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27599
        • The University of North Carolina at Chapel Hill
      • Charlotte, North Carolina, United States, 28204
        • Atrium Health
    • Ohio
      • Cleveland, Ohio, United States, 44195
        • Cleveland Clinic
      • Cleveland, Ohio, United States, 44106
        • University Hospitals Rainbow Babies & Children's Hospital
      • Columbus, Ohio, United States, 43205
        • Nationwide Children's Hospital
      • Columbus, Ohio, United States, 43210
        • The James Cancer Hospital
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Children's Hospital of Philadelphia
    • South Carolina
      • Charleston, South Carolina, United States, 29425
        • Medical University of South Carolina
    • Tennessee
      • Nashville, Tennessee, United States, 37203
        • Tristar Medical Group Children's Specialists/Sarah Cannon Center for Blood Cancers
    • Texas
      • Dallas, Texas, United States, 75246
        • Texas Oncology - Baylor Charles A. Sammons Cancer Center
      • Fort Worth, Texas, United States, 76104
        • Cook Children's

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years to 50 years (Child, Adult)

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Proportion of subjects achieving complete resolution of severe vaso-occlusive events (VOEs)
Time Frame: from Month 6 through Month 18 post EDIT-301 infusion
from Month 6 through Month 18 post EDIT-301 infusion

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Time Frame
Change from baseline in HbF concentration (g/dL)
Time Frame: up to 2 years post EDIT-301 infusion
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
up to 2 years post EDIT-301 infusion
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
up to 2 years post EDIT-301 infusion
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
up to 24 months after EDIT-301 infusion
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
up to 24 months after EDIT-301 infusion
Frequency and severity of adverse events (AEs)
Time Frame: up to 24 months post EDIT-301 infusion
up to 24 months post EDIT-301 infusion
Proportion of subjects achieving complete resolution of VOEs
Time Frame: from Month 6 through Month 18 post EDIT-301 infusion
from Month 6 through Month 18 post EDIT-301 infusion
Proportion of subjects with 90% reduction in annualized rate of severe VOE compared to pre-treatment period
Time Frame: starting from 6 months up to 2 years post EDIT-301 infusion
starting from 6 months up to 2 years post EDIT-301 infusion
Proportion of subjects with 75% reduction in annualized rate of severe VOE compared to pre-treatment period
Time Frame: starting from 6 months up to 2 years post EDIT-301 infusion
starting from 6 months up to 2 years post EDIT-301 infusion
Proportion of subjects with 50% reduction in annualized rate of severe VOE compared to pre-treatment period
Time Frame: starting from 6 months up to 2 years post EDIT-301 infusion
starting from 6 months up to 2 years post EDIT-301 infusion
Difference (pre-treatment vs. post-treatment) in annualized rates of severe VOEs
Time Frame: starting from 6 months up to 2 years post EDIT-301 infusion
starting from 6 months up to 2 years post EDIT-301 infusion
Difference (pre-treatment vs. post-treatment) in annualized rate of hospitalization for severe VOEs
Time Frame: starting from 6 months up to 2 years post EDIT-301 infusion
starting from 6 months up to 2 years post EDIT-301 infusion
Proportion of subjects with sustained HbF ≥ 20% (HbF/Hb) compared with baseline
Time Frame: starting from 6 months up to 2 years post EDIT-301 infusion
starting from 6 months up to 2 years post EDIT-301 infusion
Proportion of subjects with mean HbF ≥ 30% (HbF/Hb) compared with baseline
Time Frame: starting from 6 months up to 2 years post EDIT-301 infusion
starting from 6 months up to 2 years post EDIT-301 infusion
Proportion of subjects with mean total Hb ≥ 10 g/dL compared with baseline
Time Frame: starting from 6 months up to 2 years post EDIT-301 infusion
starting from 6 months up to 2 years post EDIT-301 infusion
Proportion of subjects with mean total Hb increase from baseline of ≥ 2 g/dL
Time Frame: starting from 6 months up to 2 years post EDIT-301 infusion
starting from 6 months 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: starting from 6 months up to 2 years post EDIT-301 infusion
starting from 6 months up to 2 years post EDIT-301 infusion

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Editas Medicine, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
May 4, 2021

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
August 1, 2025

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
August 1, 2025

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
April 16, 2021

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 16, 2021

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
April 21, 2021

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
March 25, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 29, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
January 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • EM-SCD-301-001

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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