Clinical Trial Goal
To find out:
- The highest dose of CRISPR_SCD001 that's safe to give
- If CRISPR_SCD001 is safe and works well to treat SCD in children and adults
You may be able to join this trial if you:
- Are 12 - 35 years old
- Have SCD
- Had 4 or more vaso-occlusive events in the last 2 years. This may include pain crises, acute chest syndrome, and other complications
- Do not have a brother or sister who matches you and can be your donor for an allogeneic (cells from a donor) blood or marrow transplant (BMT)
- Have not had BMT
- Have not had a stroke
- Do not get blood transfusions to prevent stroke
- Agree to have other standard tests done to see if you can be in the clinical trial
Trial Details
Everyone in this trial will get standard drugs to treat pain if needed.
First, you’ll have apheresis, a process to collect, separate and save your blood-forming cells.
Doctors will treat your blood-forming cells in a lab with CRISPR_SCD001. The therapy will edit the blood-forming cells to help your body make healthy red blood cells.
You’ll get standard chemotherapy (chemo) with busulfan to make room for the new blood-forming cells.
Then, your treated blood-forming cells (CRISPR_SCD001) are given back to you through an intravenous (IV) infusion.
You'll have blood tests to see how well the treatment is working. The clinical trial doctors will check your health for 2 years.
The Food and Drug Administration (FDA) has approved not yet approved CRISPR_SCD001.
Watch a video about gene therapy
Doctors will treat your blood-forming cells in a lab with CRISPR_SCD001. The therapy will edit the blood-forming cells to help your body make healthy red blood cells.
You’ll get standard chemotherapy (chemo) with busulfan to make room for the new blood-forming cells.
Then, your treated blood-forming cells (CRISPR_SCD001) are given back to you through an intravenous (IV) infusion.
You'll have blood tests to see how well the treatment is working. The clinical trial doctors will check your health for 2 years.
The Food and Drug Administration (FDA) has approved not yet approved CRISPR_SCD001.
Watch a video about gene therapy
Contacts
Mark Walters, MD, (510) 428-3374, Mark.Walters@ucsf.edu
Christina Chun, MPH, (415) 502-2558, Christina.Chun@ucsf.edu
Locations
UCSF Benioff Children's HospitalRECRUITING
Oakland, California
Contacts:
- Marci Moriarty, RN BSN, (510) 428-3885, marci.moriarty@ucsf.edu
- Cyrus Bascon, (510) 428-3885, cyrus.bascon@ucsf.edu
- Mark Walters, MD
University of California, Los AngelesRECRUITING
Los Angeles, California
Contacts:
- Augustine Fernandes, PhD, afernandes@mednet.ucla.edu
- Vladimir Kustanovich, PhD, VKustanovich@mednet.ucla.edu
- Gary J Schiller, MD
Sponsors
collaborator: University of California, Los Angeles, collaborator: University of California, Berkeley, lead: Mark Walters, MD
