Study Details

Open

Study to evaluate the safety and effectiveness of a single dose of EDIT-301 in individuals with severe Sickle Cell Disease (SCD)

This study will learn about an investigational drug called EDIT-301 to see how safe it is to use in people and if it will help in the treatment of severe sickle cell disease (SCD). Investigational means that the study drug being tested has not been approved by the FDA or any other regulatory authority. This is a First-In-Human (FIH) research study, which means that the study drug has not been used in people before. This study is being sponsored by Editas Medicine, Inc. The investigational drug referred to as EDIT-301 is an abbreviated name for Autologous Clustered Regularly Interspaced Short Palindromic Repeats Gene-edited CD34+ Human Hematopoietic Stem and Progenitor Cells.

Age & Gender

12 years ~ 50 years
Male, Female, Gender Inclusive

Visit Availability

Standard business hours (M-F, 8-5)
Visits can be combined with regular clinical appointments

Location

United States (Nationwide)

What will be asked of you

•Pre-Transplant (Before Study Treatment) - Screening: 1 to 2 months - Mobilization and Apheresis: approximately 3 days. During this time, you will be given a drug to help increase the number of blood-forming cells in your bone marrow and to help those cells move from your bone marrow into your blood circulation (mobilization). The blood stem cells are then collected from your blood by a process called apheresis. One procedure of mobilization and apheresis will take about 3 days. You may require more than one procedure or mobilization and apheresis. • Conditioning and Transplant with EDIT-301 - Conditioning: This starts 7 days before infusion of study treatment EDIT-301. A drug called busulfan will be administered by IV to help make room in your bone marrow for EDIT-301 (your modified blood stem cells) - Transplant: This is when you receive the infusion of study treatment, EDIT-301. This is given as a single study treatment in 1 day. • Post-Transplant (After Study Treatment) - Post-Infusion Follow-Up: up to 24 months after study treatment with EDIT-301 for 17 visits at the site or more if your study doctor wants to see you more frequently for safety assessments. Other study procedures include: Bone marrow biopsy, 12-lead ECG, COVID-19 test, pregnancy test, blood and urine tests, Liver MRI, imaging for heart function, kidney function assessment, and lung function tests.

Incentives

Compensation for study visits and reimbursement of reasonable expenses (food, travel, parking, etc.)

In-person visits:
Variable - based on disease and side effects

Phone or online visits:
Variable - based on worsened disease

Total length of participation:
Up to 30 months - this includes pre-transplant screening period, one-time treatment and follow-up. There is an optional follow-up period lasting 13 years.

Looking for Specific Volunteers

Able to participate:

You have a diagnosis of severe SCD genotype (βS/βS, βS/β0, or βS/β+) or a history of at least 2 severe vaso-occlusive events per year
You have adequate bone marrow function (ANC >1000/uL and platelet count >120,000/uL)
You are willing to stay close to UNC hospital systems for monitoring after treatment

Not eligible if:

You have an available 10/10 HLA-matched related donor
You have had a prior HSCT or contradiction to autologous HSCT
You have any contradictions to use of plerixafor or busulfan
You are unable to receive red blood cell (RBC) transfusions
You have any prior or current malignancy or immunodeficiency disorder

Contact the Team

Visit Location

Contact & Visit Location

Primary Contact

Immunotherapy Team
UNCImmunotherapy@med.unc.edu

Visit Study Website

Primary Visit Location

NC Cancer Hospital (Basnight)
UNC Hospitals / UNC Medical Center
101 Manning Dr, Chapel Hill, NC 27514, USA

Additional Study Information

Principal Investigator

Kimberly Kasow
LCCC - Clinical Trials

Study Type

Clinical or Medical
Interventional

Study Topics

Blood Conditions
Chronic Conditions
Genetics and Genetic Disorders
Transplant

IRB Number

21-0175

ClinicalTrials.gov

NCT04853576