Gene-editing Study for Sickle Cell Disease
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 plus Human Hematopoietic Stem and Progenitor Cells (EDIT-301) in Subjects with Severe Sickle Cell Disease
- Sex at Birth: Any
- Age: Adult (18 - 64)
- Accepting Healthy People: No
- Type: Interventional / Therapeutic
- Trial Phase: Phase I/II
- Conditions Being Studied: Other Hematopoietic, Hemoglobin SC Disease
Study Purpose
This study will evaluate the safety, efficacy and tolerability of treatment with EDIT-301 in reducing and/or resolving number of severe Vaso-Occlusive Events (VOEs). A vaso-occlusive event (VOEs), or may be referred to as a pain crisis, is defined as occurring when the flow of blood is blocked to an area because the sickle-shaped cells have become stuck in the blood vessel. Pain can occur anywhere but most often occurs in the bones of the arms, legs, chest, and spine. EDIT-301 (study medicine) is a new investigational therapy which uses the patients’ own stem cells, modifies them by CRISPR technology, and infuses them back to the patient to treat Sickle Cell Disorder (SCD). CRISPR is a gene editing tool that allows scientists to change DNA by adding, removing, or modifying specific portions of DNA.
Who Can Participate
Age: 18-50
Locations
UH Cleveland Medical Center
11100 Euclid Avenue
Cleveland OH, 44106
- UH IRB: STUDY20211538
- StudyID: 2021-0797
- ClinicalTrials.gov: NCT04853576
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