Understanding Clinical Trials

What Is a Clinical Trial?

A clinical trial is the scientific term for the step-by-step process that studies or tests a new procedure, drug, vaccine or device for prevention, treatment, screening or improving quality of life. The majority of clinical trials in the United States tests new drugs. Many trials are “controlled” meaning some participants will be randomly selected to receive the actual test medicine and others will receive essentially sugar water, called a placebo. The purpose is to see whether the real drug actually works since, in some cases, people’s health improves simply by taking “something”. Doctors need to know for sure that it is the drug causing the effect (either positive or negative) and not some other reason tied to emotional “feelings” about participating in the trial itself.

Types of Clinical Trials: Trials can be classified into the following categories

  • Prevention trials to test new drugs, treatments, or lifestyle changes that may help prevent disease.
  • Diagnostic trials to find new ways of detecting or finding out more about a disease.
  • Treatment trials may monitor new drugs or evaluate new combinations of established treatments to determine their effectiveness and safety.
  • Screening trials seek to find out if a screening test or tool is useful in detecting the disease at an early stage, and as a result, reduce the number of deaths from the disease.
  • Quality of life trials may study the psychological impact of the disease and ways to improve a persons comfort and quality of life.

Trial Phases:

  • Phase I trials test a potential therapy, procedure or drug for the first time in human beings. The most important goal at Phase I is to establish that a potential new treatment is safe for humans. For this reason, Phase I trials are typically conducted in very small groups of people (10-80). In addition to safety, Phase I research yields important information on side effects, effectiveness of a chosen delivery method (e.g., by mouth, injection, etc.) and appropriate dosage levels.
  • Phase II trials more comprehensively evaluate a treatment’s safety and effectiveness in a larger group of participants (usually 100 to 300).
  • Phase III trials confirm a therapy’s effectiveness, monitor side effects and compare it against the current standard treatments in a large group of people (around 1,000 to 3,000). Phase III trials last longer, are usually conducted at multiple centers and evaluate whether a treatment will benefit people with Parkinson’s — and if the benefits associated with a therapy outweigh the risks.
  • Phase IV trials are conducted after a treatment has received FDA approval and been brought to market. These trials help further evaluate long-term side effects and potential new uses for other conditions
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Why are clinical trials important?

Everyone who has ever been sick and taken a pill, even an over the counter pill, has benefited from medical research and from someone or a group of people willing to volunteer to test that pill’s safety and efficacy. Medical research is our only hope for a cure for disease. It’s the way we ensure treatments are tested and proven before they are made available to the general public. What makes clinical trials possible are the people who volunteer to help. While we need money to fund research, money won’t help us bring a drug or treatment to market. We need volunteers for that, maybe even you!

The process of developing new drugs and treatments is long, expensive and difficult. It is an expensive learning process, taking many years and often costing millions of dollars. Unfortunately, most drugs fail not because they aren’t safe, but because not enough people volunteer to test them. Eighty percent of clinical trials finish late due to difficulties enrolling participants. As a result, many drugs never make it into Phase III to determine the drug’s effectiveness. Today, at least 50,000 volunteers, both with and without Alzheimer’s are urgently needed to participate in clinical trials and studies across the United States.

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Why should African Americans and other minorities participate in clinical trials?

Minorities in the United States live sicker and die younger and yet are the most reluctant to participate in clinical research to find better treatments and cures for the diseases that impact us the most. African Americans make up more than 13 percent of the overall US population account for only 5% of clinical trial participants and Hispanics, who make up more than 16%, account for only 1% of trial participants. Increasing minority participation in clinical trials is important because there is growing evidence that race, gender and ethnicity play important roles in healthcare outcomes and also in how patients respond to drugs.

By being part of a clinical trial you can help move research forward to find an effective treatment or cure for Alzheimer’s. In the past, most drugs were tested on white men. Groups such as African Americans, Hispanic Americans, Native Americans, Asian Americans, and women, had generally not been a part of drug clinical trials. Studies have shown that sometimes drugs work differently in people in these groups. It is important to make sure we understand how to use drugs to get the best results in all people. African Americans make up over 13 percent of the population but are only 5 percent of clinical trials participants. And this number is even lower for older African Americans. We need to ban together to increase minority, and African American in particular, participation in clinical trials. It’s the only way to ensure that new drugs, treatments and therapies are both safe and effective African Americans.

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What types of volunteers do clinical trials need?

Because clinical trials vary, the needs for volunteers also vary. Researchers design clinical studies with certain eligibility criteria in mind based on the purpose of the trial and what they are testing. Men, women, young, old, people with Alzheimer’s, their relatives, and healthy volunteers may all be needed for various trials. One thing that is important to researchers is that their volunteer group must be large enough to be able to reach statistically valid conclusions.

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What are the benefits of participating in clinical trials?

There are many benefits of volunteering for clinical research. One important benefit is that you will be helping others, including perhaps members of your own family and future generations who may be at risk for developing Alzheimer’s. In addition, participating in research provides:

  • Possible opportunity to get a new drug or treatment before it is available to the general population.
  • Special care and close oversight by specialists conducting the trial
  • Possibility of receiving free health screenings and exams
  • Opportunity to contribute to medical and scientific knowledge, possibly for your good, the good of family and friends, and/or the good of future generations.
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How safe are clinical trials?

When researchers design clinical trials, they pay careful attention to making sure they are as safe as possible. They start with the lowest possible dose of the medicine and closely monitor any adverse effects. In the United States, trials involving people must be approved by an Institutional Review Board (IRB) which is a committee of physicians, scientists, community members and others who are not involved in the trial. Their job is to make sure the study does not impose unnecessary risk on volunteers and to review reports of adverse effects, if any, throughout the trial.

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What are my rights as a trial participant?

There are now federal laws in place to protect the volunteer from being taken advantage of in medical research. Today, you must be told all about a clinical trial before you are asked to sign up. This is called informed consent. You also have the right:

  • To be told the purpose of the clinical trial
  • To be told about all the risks, side effects, or discomforts that might be reasonably expected
  • To be told of any benefits that can be reasonably expected
  • To be told what will happen during the trial and whether any procedures, drugs, or devices are different than those that are used as standard medical treatment
  • To be told about available options and how they may be better or worse than what is being studied in the clinical trial
  • To be allowed to ask any questions about the trial before giving consent and at any time during the course of the trial
  • To be allowed ample time, without pressure, to decide whether to consent to participate
  • To refuse to participate, for any reason, before and after the trial has started
  • To receive a signed and dated copy of the Informed Consent form
  • To be told of any medical treatments available if complications occur during the trial

Adapted from: The Center for Information and Study on Clinical Research Participation

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What are the risks?

Almost no medical treatment, no matter how routine, is completely safe. However, clinical trials are designed with safety as a priority and risks at a minimum. There is the possibility that:

  • The new treatment may not work well for you
  • There may be known or unknown side effects
  • You may receive a placebo – i.e. an inactive pill, liquid or powder that has no treatment value.
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Who is protecting my safety?

Several agencies are specifically looking out for you and your interests.

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