Summary

Eligibility
for people ages 12-50 (full criteria)
Location
at Oakland, California and other locations
Dates
study started
completion around

Description

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).

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

Details

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.

Keywords

Sickle Cell Disease, Hemoglobinopathies, 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, Sickle Cell Anemia, EDIT-301

Eligibility

Locations

  • UCSF Benioff Children's Hospital
    Oakland California 94609 United States
  • Children's Hospital Colorado
    Aurora Colorado 80045 United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Editas Medicine, Inc.
ID
NCT04853576
Phase
Phase 1/2 research study
Study Type
Interventional
Participants
Expecting 45 study participants
Last Updated