EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)

NCT05444894 | Active Not Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: EM-301-BThal-001
• Study Type: interventional
• Target: 9
• Locations: 2 countries
• Sites: 8

Brief Summary

The purpose of this study is to evaluate the safety, tolerability, and efficacy of treatment with EDIT-301 in adult participants with Transfusion Dependent beta Thalassemia

Conditions

Transfusion Dependent Beta Thalassemia; Hemoglobinopathies; Thalassemia Major; Thalassemia Intermedia

Keywords

Beta-Thalassemia; Hemoglobinopathies; CRISPR-Cas 12a; Autologous CD34+

Outcome Measures

Primary Outcomes (2)

• Proportion of participants achieving engraftment defined as neutrophil engraftment (defined as demonstrating absolute neutrophil count (ANC) ≥ 0.5 x 10^9/L post EDIT-301 infusion for 3 consecutive measurements obtained on different days)

  EDIT-301 infusion (Day 0) to 42 days post EDIT-301 infusion

• Frequency and severity of adverse events (AEs) (incidence of AEs and Grade 3 or higher serious adverse events, using National Cancer Institute Common Terminology Criteria for Adverse Events [NCI CTCAE] v.5.0)

  Screening through up to 24 months post EDIT-301 infusion

Secondary Outcomes (13)

• Kinetics of HSPC engraftment

  EDIT-301 infusion (Day 0) to first day in which 3 consecutive measurements obtained on different days demonstrate ANC ≥ 0.5 x 10^9/L up to 24 months post EDIT-301 infusion

• Kinetics of HSPC engraftment

  EDIT-301 infusion (Day 0) to first day of 3 consecutive measurements of platelets ≥ 50 x 10^9/L for at least 1 week following the last platelet transfusion and 10 days following thrombopoietin mimetics use up to 24 months post EDIT-301 infusion.

• Incidence of transplant related mortality

  EDIT-301 infusion (Day 0) through Day 100 post EDIT-301 infusion and from EDIT-301 infusion (Day 0) through 12 months post EDIT-301 infusion

• Incidence of all-cause mortality

  Screening through up to 24 months post EDIT-301 infusion

• Proportion of alleles per participant with intended genetic modification present in peripheral blood over time

  EDIT-301 infusion (Day 0) through up to 24 months 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 intravenous infusion after myeloablative conditioning with busulfan.

Also known as: renizgamglogene autogedtemcel, reni-cel

EDIT-301

Eligibility Criteria

• Age: 18 Years - 35 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Diagnosis of Transfusion Dependent B-Thalassemia as defined by:
• Documented homozygous β-thalassemia or compound heterozygous β-thalassemia including β-thalassemia/hemoglobin E (HbE) based on historical data in medical records, and
• History of at least 100 mL/kg/year or 10 U/year of packed red blood cell (RBC) transfusions in the 2 years prior to signing informed consent
• Clinically stable and eligible to undergo autologous HSCT
• Karnofsky Performance Status ≥ 70

You may not qualify if:

• Available 10/10 human leukocyte antigen (HLA)-matched related donor
• Prior HSCT or contraindications to autologous HSCT
• Participants with associated a history of α-thalassemia and \> 1 alpha chain deletion, or alpha multiplications as documented in medical records
• Participants with a history of other inherited hemoglobinopathy or thalassemic mutation (Hb S, C, D or other) as documented in medical records
• Prior receipt of gene therapy
• Inadequate bone marrow function, as defined by white blood cell count of \< 3 x 10\^9/L or a platelet count \< 100 x 10\^9/L (without hypersplenism), per investigator judgement
• Inadequate 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 (8)

University of California San Francisco

Oakland, California, 94609, United States

Location

University of Minnesota

Minneapolis, Minnesota, 55410, 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

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

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

Nashville, Tennessee, 37203, United States

Location

Princess Margaret Cancer Centre-University Health Network

Toronto, Ontario, M5G 2M9, Canada

Location

MeSH Terms

Conditions

Hemoglobinopathies; beta-Thalassemia

Condition Hierarchy (Ancestors)

Hematologic Diseases; Hemic and Lymphatic Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Thalassemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia

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: June 27, 2022
• First Posted: July 6, 2022
• Study Start: April 29, 2022
• Primary Completion: September 1, 2025
• Study Completion: December 1, 2025
• Last Updated: April 2, 2025

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (7); CA (1)