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In Pain, Out of Work and Can't Pay the Bills
A Resource Directory for People With CRPS

Clinical Trials

Long before a new medication or modality is brought to market, a series of research studies or clinical trials are conducted to make sure the therapy is safe for patients and effective for the disease state it is designed to treat. Clinical trials are organized tests of medicines and new treatment options and rely on patient volunteers who try the new or changed treatment. Clinical trials can study medicines that have not been approved by the US Food and Drug Administration (FDA) for sale in the US, or drugs that are on the market but are being tested for different dosages or even a new disease state. Generally, pharmaceutical or biotechnology companies sponsor clinical trials, although some health-related government agencies can also provide funding and other resources.

Why Participate in a Clinical Trial?

The process of collecting information in the study will allow your physician to find out more about your disease and the effects it has on you. You may benefit from better treatment after the trial. You may receive free laboratory and medical testing. Even if the trial may not benefit you directly, the information gathered may be of help to other patients with the same condition. Many patients want to be part of the effort to potentially reduce the suffering of others.

Are Clinical Trials Safe?

Clinical trials are conducted in a healthcare setting (a hospital or clinic) and are typically monitored by a trained healthcare professional. The FDA develops strict policies and guidelines for all medical research, regardless of manufacturer, study phase, or drug type, and also reviews clinical trials. Independent Institutional Review Boards (IRB), a committee made up of physicians, ethicists, and members of the general public and administrators, also review and approve all study-related documents, such as protocols, informed consent forms, physician credentials and eligibility, and patient recruitment materials, including advertisements and public service announcements. The IRB is held responsible for determining that a trial does not represent an unreasonable risk to patients who are participating.

Are You Eligible?

Your personal physician is responsible for your healthcare and is the best person to help you decide whether or not you are eligible to participate in a trial.

The Clinical Trial Process

A drug under investigation must pass through three phases of study before it can be approved by the FDA and possibly a fourth phase after being approved.

Phase I: The manufacturer wants to find out how the drug works in healthy study participants. Phase I studies are primarily concerned with assessing the drug’s safety. This initial Phase of testing in humans is done in a small number of healthy volunteers (20 to 100), who are usually paid for participating in the study. The study is designed to determine what happens to the drug in the human body (how it is absorbed, metabolized, and excreted). A Phase I study will investigate side effects that occur as dosage levels are increased. This initial Phase of testing typically takes several months; about 70% of experimental drugs pass this initial Phase of testing.

Phase II: Once a drug has been shown to be safe, it must be tested for effectiveness. This second phase of testing may last from several months to two years and involves up to several hundred patients. Most Phase II studies are randomized trials. One group of patients will receive the experimental drug while a second “control” group will receive a standard treatment or placebo. Often these studies are “blinded”—neither the patients nor the researchers know who is getting the experimental drug. In this manner, the study can provide the pharmaceutical company and the FDA with comparative information about the safety and effectiveness of the new drug. Only about one-third of experimental drugs successfully complete both Phase I and Phase II studies. Safety continues to be evaluated. Now the study group is even larger and the trial uses advanced rating scales and clinical measures.

Phase III: A drug is tested in several hundred to several thousand patients. This large-scale testing provides the pharmaceutical company and the FDA with a more thorough understanding of the drug’s safety, effectiveness, benefits, and the range of possible adverse reactions. In recent years, there has been a growing industry trend to not only measure clinical effectiveness at this phase, but also measure quality-of-life results. Most Phase III studies are randomized and blinded trials and typically last several years. Between 70% and 90% of drugs in Phase III studies successfully complete this phase of testing and move to the FDA for marketing approval. Most drugs that reach Phase III will at least be considered for approval by an FDA advisory board.

Phase IV: These studies identify an additional use for an already approved drug, or gather additional safety information from a larger group of patients. In some cases, Phase IV studies are implemented to establish effectiveness in a subgroup of patients, for example, patients over age 65.

After the Study

When the clinical trials are complete, the FDA rules whether to continue or halt the drug’s development. If it remains in development, the manufacturer may build in a “compassionate use” (also called “expanded access”) extension for the study. This allows patients to receive drugs that have not yet been approved by the FDA.

Usually an investigational drug can be considered for compassionate use when the patient will benefit from the use of the drug; there is no drug commercially available to successfully treat the patient’s disease state; the patient currently does not meet eligibility requirements to be treated on an investigational protocol using this drug; no investigational protocol exists; or the patient does not have access to a physician participating in such a protocol. Often, patients requesting compassionate access may include those who are too sick to participate in the controlled studies.

The FDA has no formal regulations covering such programs. However, for each compassionate access program, companies make a proposal to the FDA showing that the program fulfills minimum requirements for scientific and ethical merit and targets a population with the disease that was included in previous trials. At their own expense, drug companies provide drugs free to patients and analyze and present data on their treatment to the FDA. The drugs involved are usually in Phase III of the drug approval process.

Questions to Ask Before You Enter a Clinical Trial

  • How long will the trial last?
  • Will medications prescribed by my regular physician interfere with the study?
  • Are there any tests requiring painful shots or blood samples? What do I do if I want a friend/relative to join a study?
  • Will I be paid?
  • Will I be reimbursed for transportation expenses?
  • What kinds of medical problems would prevent participation in the clinical trial?
  • Do I have to talk to my health insurance company before enrolling in a study?
  • What if I miss a dose?
  • Can I drop out at any time?
  • Are there placebos involved?

Terms Used in Clinical Trials

Baseline: A patient’s initial vital signs and clinical symptoms are documented. The patient’s response is compared back to baseline over time.

Clinical Trial Site: The facility where the trial is being conducted. It can be a physician’s private practice, a center dedicated solely to clinical research, or a hospital setting.

Contract Research Organization (CRO): A clinical services company involved with various steps in the clinical trial process from study design to trial execution and data management.

Double-blind Trial: Many clinical trials are double blind in design. This means that neither patients nor investigators/doctors know whether an individual participant is on an active drug or a placebo.

FDA (US Food and Drug Administration): The FDA is the federal agency responsible for ensuring that drugs, biological products, and medical devices are safe and effective for the general public. The FDA reviews and oversees the manufacturing specifications of these products, as well as the execution of clinical trials in which these products are evaluated. The agency also ensures that these products, once made commercially available, are represented to the public in an accurate and informative manner.

Inclusion/Exclusion Criteria: Inclusion/Exclusion Criteria is an actual section of a clinical trial protocol that lists specific medications, conditions, and relevant medical information that may, or may not, be allowable during a trial. Age, blood pressure, and previously taken medications are a few examples of patient-specific information that would be compared to the inclusion/exclusion criteria of a protocol to determine a patient’s eligibility.

Informed Consent Form: A form that outlines a patient’s rights during participation in a clinical trial. It also discussesthe potential risks and benefits associated with participation. An Informed Consent form must be signed by the patient or authorized caregiver before entrance into a study.

Institutional Review Board (IRB): An IRB is an independent committee, comprised of individuals with diverse medical and non-medical backgrounds, that reviews and approves all study-related documents. An IRB approves protocols, Informed Consent forms, physician credentials and eligibility, and patient recruitment materials, such as print advertisements and public service announcements. IRBs work closely with the FDA to ensure patient safety in a clinical trial.

Open-label Trial: Later-phase studies (Phases III to IV) often have an open-label design, meaning that all patients receive active study medication— no placebo is dispensed.

Placebo: A substance containing no active ingredients and having no pharmacological effects. Clinical trials often compare an investigational medication to a placebo as a way to differentiate patient response.

Principal Investigator (PI): The lead doctor in charge of the clinical management of a clinical trial. As part of the site selection process, the PI’s academic credentials and area(s) of expertise are evaluated.

Protocol: A set of guidelines that must be followed during a clinical trial. This includes information such as patient eligibility, data to be collected during patient visits, and included and excluded medical conditions and medications.

Randomization: The assignment of individuals to treatment groups in such a way that each individual in a clinical trial has an equal chance to be assigned to each treatment group. (Also see Double-Blind Trial)

Study Coordinator: A key member of the clinical team who works directly for the principal investigator. Responsibilities usually include maintaining accurate patient documentation, dispensing medication, and corresponding with patients.

For information on clinical trials, visit www.clinicaltrials.gov

Clinical trials for Complex regional pain syndrome on NHS Choices
Information provided by WHO International Clinical Trials Registry

ResearchMatch is a nonprofit effort that brings together researchers and people who are willing to learn more about research studies via a secure and convenient online web portal.

ResearchMatch has a simple goal—to bring together two groups of people who are looking for one another: (1) people who are trying to find research studies, and (2) researchers who are looking for people to participate in their studies.  It is a free and secure registry that has been developed by major academic institutions across the country who want to involve you in the mission of helping today's studies make a real difference for everyone's health in the future.

 

 

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Updated October 26, 2010

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