Pediatric Genetics Clinical Trials

Our Pediatric Genetics clinical trials team at Vanderbilt includes medical doctors, nurse practitioners, research nurse specialists and clinical trial associates. Our team's goal is to unite our resources as advocates for your child and their health.

We are currently involved in clinical trials for achondroplasia, phenylketonuria, fatty acid oxidation disorders (to include CPT2, CACT, TFP, LCHAD and VLCAD), Angelman syndrome and lysosomal storage diseases.

What are clinical trials?

A clinical trial is a research study that uses human volunteers to try to answer a specific question. Sometimes when a new drug or cancer treatment becomes available, a clinical trial is an important step in the approval process. Some clinical trials study an investigational product (a non-FDA-approved study drug), and some are observational (without a study drug).

Clinical trials are held for different reasons, says the National Institutes of Health (NIH):

  • Treatment trials test experimental treatments, new combinations of drugs or new types of surgery or radiation therapy.
  • Prevention trials test new ways to prevent certain diseases or prevent a disease from returning.
  • Diagnostic trials look at new tests or procedures that diagnose disease.
  • Screening trials test new methods for finding disease.
  • Quality of life trials look at new ways to improve quality of life for people with chronic illness.

Are clinical trials safe?

Yes. Most clinical trials test treatments that already have shown some promise of being more effective than existing therapies. All clinical trials that take place in the U.S. must be overseen by an institutional review board (IRB) at each site participating in the research. The IRB helps make sure of low risks and proper trial procedures.

At Vanderbilt we place your child's safety first. We work closely with the Vanderbilt University IRB to ensure we honor patients’ rights.

All clinical trials have guidelines that describe the criteria for participants. To ensure that a trial's results are reliable, you may be included or excluded from the trial according to these criteria. In most trials, one group of patients is given a standard treatment. Another group receives the therapy being tested. Neither the patient nor the clinical healthcare provider knows which treatment each individual person receives.

At Vanderbilt we inform all participants about the risk involved in every research study prior to involvement. Each study is different and has its own risks. It is very important that you take the time to read the informed consent and understand all aspects of the study prior to taking part.

All clinical trials are on a volunteer basis. You and your child may withdraw at any time. This decision will not affect your care here at Vanderbilt or anywhere else.

How much does it cost to be part of a clinical trial?

This can vary from study to study. Don't assume you cannot be part of a study due to cost. Ask the study site about the cost, and if they will reimburse families for the research visits. Many studies do cover cost for your visits and your time.

How can I learn more about the clinical trials offered at Vanderbilt?

If your child has one of the diseases listed above, contact our study team at (615) 343-6761.

If you want to learn about all the clinical trials available for all diseases, visit Vanderbilt's Research Match website and enter the disease in the search box at the bottom of the screen.