A Phase I Dose Finding Study Evaluating Safety and Feasibility of BPX-701 in Patients with Relapsed or Refractory Myeloid Neoplasm

Principal Investigator

Rachel Cook

Study Purpose

The purpose of this study is to test the safety of a gene transfer agent called BPX-701. A gene transfer agent is produced with segments of genetic material. Genes are the units of DNA—the chemical structure carrying your genetic information—that determine many human characteristics such as the color of your eyes, your height, and whether you are male or female.

This study is one of the first steps in studying whether BPX-701 can be used to treat others with your disease in the future. This study tests different doses of the gene transfer agent to see which dose is safer in people.

Medical Condition(s)

Acute Myeloid Leukemia (AML) or Myelodysplastic Syndromes (MDS)

Eligibility Criteria

You may be eligible if you:
-Have AML or MDS and, after treatment, your disease has either relapsed (come back) or is refractory (has not responded to treatment)
-Are at least 18 years old

Age Range

18 - N/A

Healthy Volunteers Needed


Duration of Participation

You will receive the study T cells as an infusion by vein (intravenously, IV) at one study visit. You will stay in the hospital overnight for the infusion.

Long-term follow-up in gene transfer research allows for the collection of important information on the long-term safety and effects of the gene transfer intervention used in this study. The long-term follow-up planned for this study will occur at least every 6 months for 5 years, then yearly for 10 years in a potentially separate and long-term safety follow-up study (a total of 15 years of follow-up). It includes physical exams and blood draws.

Minors Included



Clinical Trials Information Line
Phone: 503-494-1080


Bellicum Pharmaceuticals

Recruitment End


Compensation Provided


Go Back