US Tax Deductible EIN: 26-0256621

- (562) 212-1071

- 2409 E. Luke Avenue Phoenix AZ 85016

US Tax Deductible EIN: 26-0256621

- (562) 212-1071

- 2409 E. Luke Avenue Phoenix AZ 85016

What is a Clinical Trial???

This week, the Board of the CTSF hopes to share some information with you on the progress that has been made in moving forward with Gene Therapy clinical trials for our kids......... we call it "Traveling Towards Treatment". That involves clinical trials, something that so many of you have helped us fund in our 11 year existence. So, it dawned on me that not everyone may know exactly what a clinical trial is. Never fear, for there are no dumb questions on this FB page!

So, What is a Clinical Trial???

New tests and treatments aren't offered to the public as soon as they're made. They need to be studied. A clinical trial is a type of research that studies a test or treatment given to people. Clinical trials study how safe and helpful tests and treatments are. When found to be safe and helpful, they may become tomorrow's standard of care. Clinical trials can study many things, such as:

  • New drugs not yet approved by the U.S. FDA (Food and Drug Administration)
  • New uses of drugs already approved by the FDA
  • New ways to give drugs, such as in pill form
  • Use of alternative medicines, such as herbs and vitamins
  • New tests to find and track disease, and
  • Drugs or procedures that relieve symptoms

Clinical trials are carefully designed, reviewed, and completed. The principal investigator is the person in charge of the trial. He or she is a scientist who's an expert in what the clinical trial is about. The principal investigator takes the lead in designing the clinical trial, choosing the research team, and carrying out the study. Often, other scientists, called investigators, are part of the research team.

Clinical trials need to be approved before they can start. Clinical trials need sponsors since they can't be done without money or other support. Sponsors often are government agencies, pharmaceutical (drug) companies, and non-profit organizations. Sponsors of the clinical trial review the research plan (also called protocol). Funding or other support is given by the sponsor once the research plan is approved.

Clinical trials also need approval from an IRB (Institutional Review Board). An IRB is a group of people chosen by the health care center where patients will be enrolled into a clinical trial. Each IRB include five members. These members must include a scientist, someone who's not a scientist, and someone who's not from the health care center. The IRB reviews the research plan to make sure the rights and welfare of patients are protected. After the trial has started, the IRB reviews its progress at least every year.

What each clinical trial has to offer to patients differs. However, there are four general benefits. First, you'll have access to the most current care. Second, you will be treated by experts. Third, the results of your treatment-both good and bad-are carefully tracked. Fourth, you may help other patients with Tay-Sachs.

Clinical trials have risks too. Like any test or treatment, there may be side effects. Also, new tests or treatments may not work as good as or better than the one now in use. Trips to the treating hospital may be frequent and expensive.

Stay tuned for specific information that applies to the work you have been helping us fund..... and be excited for the possibilities!

Rick and the CTSF Board of Directors