Early Drug Development in Pediatric Cancer


The Children's Center for Cancer and Blood Disorders has developed a comprehensive Early Drug Development Program for patients who have no good treatment options or have not responded to standard therapy. 

Phase I and II trials are available through this program, representing the early development of some of the most promising agents available.

These trials give our patients access to these newest treatment agents, without having to travel to other centers within Arizona or out-of state. Phoenix Children's is the only center in the Southwest tri-state region (Arizona, New Mexico, and Nevada) to offer phase I pediatric oncology trials. Families from outside the state and country have been referred to our program to receive these treatments.

Early Drug Development Collaboration 

The Early Drug Development Program is made up of a seven-member team including two research nurses and five physician-investigators representing the Liquid Tumor, Solid Tumor, and Neuro-oncology teams.

The program’s clinical trials come from many different sources.

Phoenix Children's is a member of the Pediatric Oncology Experimental Therapeutics Investigators’ Consortium (POETIC), an elite consortium made up of nine major pediatric oncology centers in the U.S. and one in Canada.  POETIC is devoted to cutting-edge phase I and II clinical trials with incorporated, correlative science.

Phoenix Children's is also a member of the Neuroblastoma and Medulloblastoma Translational Research Consortium (NMTRC), a group of 15 institutions devoted to phase I clinical trials, particularly in neuroblastoma and medulloblastoma.

In addition to these consortiums, investigators at Phoenix Children's have also develop and enroll in patients clinical trials with various other academic and industry partners, to provide the newest agents to their patients. 


What are the phases of a clinical trial?

Most clinical research progresses in an orderly series of steps or phases:

  • Phase 0 trials. These are the earliest steps in testing new treatments in humans. A small number of people are studied and the focus is on learning how the agent is processed by the body and how the agent affects the body. No information is gathered on safety or effectiveness in treating cancer.

  • Phase I trials. These are the first studies to evaluate how a new therapy should be administered — how it should be given, how often, and in what dosage. The focus is to determine safety (drug dose, device safety, or other therapy's safety) for the next phase of testing.

  • Phase II trials. These provide preliminary information about how well the new therapy works, and generates more information about its safety and benefits. A Phase II study usually focuses on a particular type of cancer.

  • Phase III trials. These compare a promising new therapy, combination of treatments, or procedure with a current standard of treatment. These trials often enroll large numbers of people and may be done at may doctors' offices, clinics, and cancer centers nationwide, or even worldwide.

  • Phase IV trials. These include the continuing evaluation that takes place after FDA approval, when the therapy is already available for general use.

What is a clinical trial protocol?

Clinical trial protocols are the action plans or guidelines that must be followed during the course of any study. Guidelines may cover:

  • The design of the study — what will be studied and how

  • Who may participate — criteria for patients who enter the study

  • What treatments and medical tests will be used to follow the participants

  • Specific research questions

  • What information will be collected

Several research centers may be involved in the study, and each uses the same protocols to ensure that information from all centers can be combined and compared.

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