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

NCT04853576 | Active Not Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: EM-SCD-301-001
• Study Type: interventional
• Target: 45
• Locations: 2 countries
• Sites: 24

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).

Conditions

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

Outcome Measures

Primary Outcomes (1)

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

  from Month 6 through Month 18 post EDIT-301 infusion

Secondary Outcomes (17)

• Proportion of subjects achieving complete resolution of VOEs

  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

  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

  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

  starting from 6 months up to 2 years post EDIT-301 infusion

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

  starting from 6 months up to 2 years post EDIT-301 infusion

Study Arms (1)

EDIT-301

EXPERIMENTAL

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

Genetic: EDIT-301

Interventions

EDIT-301 GENETIC

Administered by IV infusion after myeloablative conditioning with busulfan.

Also known as: renizgamglogene autogedtemcel, reni-cel

EDIT-301

Eligibility Criteria

• Age: 12 Years - 50 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Editas Medicine, Inc.: lead

Study Sites (24)

UCSF Benioff Children's Hospital

Oakland, California, 94609, United States

Location

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

Smilow Cancer Hospital

New Haven, Connecticut, 06511, United States

Location

Johns Hopkins All Children's Hospital

St. Petersburg, Florida, 33701, United States

Location

Children's Healthcare of Atlanta

Atlanta, Georgia, 30322, United States

Location

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

University of Mississippi Medical Center

Jackson, Mississippi, 39216, United States

Location

Hackensack University Medical Center

Hackensack, New Jersey, 07601, United States

Location

Columbia University Medical Center - Department of Pediatrics

New York, New York, 10032, United States

Location

Columbia University Medical Center

New York, New York, 10032, United States

Location

The University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, 27599, United States

Location

Atrium Health

Charlotte, North Carolina, 28204, United States

Location

University Hospitals Rainbow Babies & Children's Hospital

Cleveland, Ohio, 44106, United States

Location

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

The James Cancer Hospital

Columbus, Ohio, 43210, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

Tristar Medical Group Children's Specialists/Sarah Cannon Center for Blood Cancers

Nashville, Tennessee, 37203, United States

Location

Texas Oncology - Baylor Charles A. Sammons Cancer Center

Dallas, Texas, 75246, United States

Location

Cook Children's

Fort Worth, Texas, 76104, United States

Location

Ottawa Hospital Research Institute

Ottawa, Ontario, K1H 8L6, Canada

Location

Princess Margaret Cancer Centre

Toronto, Ontario, M5G 2M9, Canada

Location

Centre Hospitalier Universitaire Sainte-Justine

Montreal, Quebec, H3T 1C5, Canada

Location

MeSH Terms

Conditions

Anemia, Sickle Cell; Hemoglobinopathies; Anemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Genetic Diseases, Inborn; Hematologic Diseases

Condition Hierarchy (Ancestors)

Hemic and Lymphatic Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 16, 2021
• First Posted: April 21, 2021
• Study Start: May 4, 2021
• Primary Completion: August 1, 2025
• Study Completion: August 1, 2025
• Last Updated: January 31, 2025

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will not share

Locations

CA (3); US (21)