A Phase I Study of AMG 191 Given Before Transplant to Patients with Severe Combined Immunodeficiency (SCID)

Full Title

A Phase 1/2 Study to evaluate the Safety, Tolerability, and Efficacy of JSP191 for Hematopoietic Cell Transplantation Conditioning to Achieve Engraftment and Immune Reconstitution in Subjects with SCID

Purpose

Hematopoietic stem cell transplantation (HCT) is the only proven cure for severe combined immunodeficiency (SCID), a rare disorder in which patients do not have functional lymphocytes (white blood cells that work properly). Recent studies suggest that for infants with SCID who do not have the option of a transplant from a matched sibling, the best outcomes (in terms of restoring immune function) are achieved when chemotherapy is given before the transplant. However, this chemotherapy can have short-term and long-term side effects, and patients with SCID are especially vulnerable to these effects.

The study will assess a treatment that is expected to cause fewer side effects than standard chemotherapy. Before transplant, a single dose of a protein called a monoclonal antibody is given intravenously (by vein). This antibody, called AMG 191, binds to a specific molecule on the surface of cells called c-kit or CD117. The researchers of this study want to know if AMG 191 can be used instead of chemotherapy to help stem cells from the donor take hold (engraft) in the patient. In this study, they will see which dose of AMG 191 is best.

There are two types of patients who may be eligible for this trial: those who already had a transplant and have poor immune function with no stem cells from the donor, and those who have never had a transplant.

Who Can Join

To be eligible for this study, patients must meet several criteria, including but not limited to the following:

• Patients in this study must have SCID and be scheduled to receive a first or second stem cell transplant.
• Patients must have a stem cell donor.
• Patients who have already had a stem cell transplant must be age 6 months or older. Those having a transplant for the first time should be age 3 months or older.

For more information about this study and to inquire about eligibility, please contact Dr. Susan Prockop at 212-639-3267.