A Phase 1/2 Study of Afamitresgene Autoleucel SPEAR T Cell Therapy in Children and Young Adults With Solid Tumors


Full Title

A Phase 1/2, Open-Label, Basket Study to Assess the Safety, Tolerability, and Anti-Tumor Activity of Afamitresgene Autoleucel in Pediatric Subjects with MAGE-A4 Positive Tumors


Researchers are assessing a new type of therapy in young people with solid tumors that keep growing even after treatment. The therapy is called afamitresgene autoleucel. The people in this study have synovial sarcoma, malignant peripheral nerve sheath tumor (MPNST), neuroblastoma, or osteosarcoma. In addition, they have tested positive for the HLA-A*02 gene and their cancers make a protein called MAGE-A4. This protein plays a role in cancer growth.

Afamitresgene autoleucel is a cellular therapy made from your white blood cells called T cells. Some cancers block T cells from attacking cancer cells. If you join this study, we will first collect some of your T cells and add a small piece of DNA. This will help your T cells find and kill cancer cells that make MAGE-A4. These genetically changed T cells are called “specific peptide enhanced affinity receptor” (SPEAR) T cells. Treatments made from them are called SPEAR T cell therapies.

When it nears the time for you to get afamitresgene autoleucel, you will first get fludarabine and cyclophosphamide. These two chemotherapy drugs prepare your body to receive the cellular therapy. Afamitresgene autoleucel is then given intravenously (by vein).

Who Can Join

To join this study, there are a few conditions. You or your child must:

  • Have synovial sarcoma, MPNST, neuroblastoma, or osteosarcoma that keeps growing even with treatment.
  • Have the HLA-A*02 gene and a cancer that makes MAGE-A4.
  • Be well enough to walk and take care of yourself. You must be able to do activities such as office work or light housework.
  • Be age 2-21 years.


For more information and to see if you or your child can join this study, please call 1-833-MSK-KIDS.




Phase I/II (phases 1 and 2 combined)


ClinicalTrials.gov ID