Clinical Trial Goal
To find out:
- The highest dose of APR-2020 that's safe to give
- If APR-2020 is safe and works well to treat DBA in children and teens with RPS19 deficiency
You may be able to join this trial if you:
Your child:
- Is 2 - 18 years old
- Has DBA
- Has RPS19 deficiency
- Is transfusion-dependent
- Is eligible for allogeneic (cells from a donor) blood or marrow transplant (BMT). Your child's doctor can tell you this
- Does not have a fully matched sibling donor
- Has not been treated with allogeneic BMT or gene therapy
- You agree to have other standard tests done to see if your child can be in the clinical trial
Trial Details
APR‑2020 is a type of gene therapy that uses a harmless, modified virus (called a lentiviral vector) to deliver a healthy copy of the gene your body is missing. By giving your stem cells the correct instructions, doctors hope this treatment will help your body make healthy red blood cells and reduce or eliminate the need for transfusions.
First, your child will have apheresis, a process to collect, separate and save their blood-forming cells (CD34+ cells).
Doctors will treat your child's blood-forming cells in a lab with APR-2020. The therapy will add a healthy copy of the RPS19 gene to these stem cells to replace the gene that causes Diamond‑Blackfan anemia.
Then, your child’s treated blood‑forming cells are given back to them through an intravenous (IV) infusion.
Your child will have blood tests to see how well the treatment is working. The clinical trial doctors will check your child's health for up to 2 years.
The Food and Drug Administration (FDA) has not yet approved APR-2020.
Watch a video about gene therapy
Doctors will treat your child's blood-forming cells in a lab with APR-2020. The therapy will add a healthy copy of the RPS19 gene to these stem cells to replace the gene that causes Diamond‑Blackfan anemia.
Then, your child’s treated blood‑forming cells are given back to them through an intravenous (IV) infusion.
Your child will have blood tests to see how well the treatment is working. The clinical trial doctors will check your child's health for up to 2 years.
The Food and Drug Administration (FDA) has not yet approved APR-2020.
Watch a video about gene therapy
Locations
Stanford University - Stanford Children's HealthRECRUITING
Stanford, California
Research Nurse, 650-724-6055, scgt_clinical_trials_office@lists.stanford.edu
Boston Children's HospitalRECRUITING
Boston, Massachusetts
Emily Morris, 617-632-1954, gene.therapy@childrens.harvard.edu
Sponsors
lead: Apriligen, Inc.
