Summary

Eligibility
for people ages 18-35 (full criteria)
Location
at Oakland, California and other locations
Dates
study started
estimated completion

Description

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

Official Title

A Multicenter Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Dose of Autologous Clustered Regularly Interspaced Short Palindromic Repeats Gene-edited Cluster of Differentiation 34 (CD34+) Human Hematopoietic Stem and Progenitor Cells (HSPC) (EDIT-301) in Transfusion-Dependent Beta Thalassemia (TDT)

Details

This is a Phase 1/2 single-arm, open-label, multicenter study evaluating the safety, tolerability, and efficacy of a single unit dose of EDIT-301 for autologous hematopoietic stem cell transplant in adult participants with TDT, age 18 to 35 years, inclusive

Keywords

Transfusion Dependent Beta Thalassemia, Hemoglobinopathies, Thalassemia Major, Thalassemia Intermedia, Beta-Thalassemia, CRISPR-Cas 12a, Autologous CD34+, Thalassemia, EDIT-301

Eligibility

You can join if…

Open to people ages 18-35

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 CAN'T join 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 109/L or a platelet count < 100 x 109/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

Locations

  • University of California San Francisco accepting new patients
    Oakland California 94609 United States
  • Tristar Medical Group Children's Specialists/Sarah Cannon Center for Blood Cancers accepting new patients
    Nashville Tennessee 37203 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Editas Medicine, Inc.
ID
NCT05444894
Phase
Phase 1/2 research study
Study Type
Interventional
Participants
Expecting 6 study participants
Last Updated