Clinical Trials

Clinical trials are the keystone for advancement towards a cure

Research to discover improved therapies and, ultimately, a cure forms the basis for all that is done at the Scleroderma Research Foundation. One of the most important components of medical research is the clinical trial.

 

Interested in clinical trials that are currently recruiting?

If you would like to participate in or learn about clinical trials that are currently recruiting, follow the links below to ClinicalTrials.gov, a service of the National Institutes of Health (NIH).

Click below to learn more about active clinical trials for these illnesses:

For more resources on clinical trials visit the sites below:

 

What is a clinical trial?

A clinical trial is a research study designed to test potential disease treatments that have already shown promise in the laboratory or in a limited number of patients through a small pilot study. The goal of a clinical trial is to determine the efficacy and safety of a treatment with human patients.

Clinical trials are often sponsored by the federal government (through the NIH and other agencies), by private industry (pharmaceutical and biotechnology companies) and by medical institutions, foundations and other groups.

How does a clinical trial work?

Before a trial can begin, the U.S. Food and Drug Administration (FDA) must decide whether to grant approval to test a new drug or treatment approach on human subjects. The FDA bases its decision on data collected from extensive laboratory and preclinical research, which may include animal subjects and experiments using human tissue samples. The data is submitted as part of an Investigational New Drug application (IND). With FDA approval of this application, a clinical trial can begin.

What happens during a clinical trial?

The clinical trial process will depend on the kind of trial being conducted. The clinical trial team includes doctors and nurses as well as social workers and other health care professionals. They check the health of the participant at the beginning of the trial, give specific instructions for participation, monitor the participant carefully and keep in contact after the trial is completed.

Some clinical trials involve more tests and doctor visits than the participant would normally have for an illness or condition. For all types of trials, the participant works with a research team. Clinical trial participation is most successful when the protocol is carefully followed and there is frequent contact with the research staff.

In general, clinical trials are conducted in phases. Each phase serves a different purpose:

Phase I evaluates the safety of the treatment on a small number of participants (usually 20-80 healthy people), establishes the correct dosage and identifies side effects. The experimental treatment must pass the Phase I safety test before the study can proceed to the next phase.

Phase II tests the effectiveness of the experimental drug or treatment on a larger group (100-300 patients). The goal is to answer the question: Does the drug work the way it is intended to? Phase II can last for several months to two years.

This phase is often a randomized trial, which means that one group of patients will receive the experimental drug while the other group—the control group—receives a placebo (a harmless substance with no active ingredients). The trial is often conducted as a double blind trial, meaning that neither the participants nor physician or researcher administering the drug and monitoring the patients know who is getting the experimental drug and who is getting the placebo.

Phase III provides more information on the benefits and drawbacks of the treatment, which is tested on a much larger group of people (several hundred to several thousand) and can last for several years. Phase III is usually a randomized, double blind, placebo controlled trial comparing the experimental treatment to already established treatments to see which is most effective and safe, and has fewer side effects.

Once Phase III is successfully completed, the pharmaceutical company will request FDA approval to put the drug on the market and make it available to patients.

Phase IV takes place after the drug has been approved by the FDA, and is not always required. This phase may be conducted to follow up on long-term effectiveness and side effects of the drug and its impact on the patient’s quality of life. Sometimes a pharmaceutical company may choose to conduct a Phase IV trial to compare its drug to competing products.

Is participation in a clinical trial right for you?

Participation in a clinical trial is a personal decision as well as one that should be made with as much information as possible. Since there can be serious risks involved when entering into a clinical trial, you should have extensive conversations with the research team, your own medical care provider and your loved ones to determine if this is the right path for you. Below is a list of questions that you may want to ask the researchers.

Asking questions and gathering information will help you make an informed decision.

Questions you might consider:

  • What is the purpose of the trial?
  • What tests and treatments will be used?
  • What is known so far about the experimental treatment?
  • What are the benefits, risks and side effects compared to your current treatment?
  • Why do researchers believe the experimental treatment being tested may be effective? Has it been tested before?
  • How will my safety be protected?
  • How will the trial affect my daily life?
  • How long will the trial take?
  • Who will conduct the trial and what are their credentials?
  • Who is funding/sponsoring the trial?
  • Will I receive payment for participating and/or be reimbursed for expenses?
  • Who is included in the study?
  • Is hospitalization required?
  • Who will pay for the experimental treatment?
  • What type of long-term follow up care is part of this study?
  • How will I know that the experimental treatment is working? Will results of the trials be provided to me?
  • Who will be in charge of my care?

You may also want to:

  • Plan ahead and prepare the list of questions you want to ask.
  • Ask a friend or relative to come along for support, listen to responses to the questions and provide feedback.
  • Take detailed notes, ask for any relevant materials and/or bring a recording device so that you can review the discussion at a later time.

Benefits to participating

As a patient participating in a clinical trial, you can gain access to promising new research treatments in Phases II and III of the study, before they are available in the marketplace; play an active role in your own health care; receive expert medical care during the trial from a health care team; and benefit other patients by contributing to medical research.

Risks to participating

You may experience side effects from the experimental treatment that could range from unpleasant to serious or life-threatening; the treatment may not be effective; the protocol may be time-consuming and demanding, requiring more doctor visits and/or hospital stays, potential for complicated dosages and procedures and more detailed monitoring.

Who can participate?

Potential study participants must meet certain criteria to qualify. These requirements are called inclusion and exclusion criteria and are based on such considerations as age, gender, the type and progress of the disease, other medical conditions and more. These criteria are used to reduce risks to the participants while meeting the requirements of the research plan.

Protecting the safety of participants

Every clinical trial must first gain approval to go forward from an Institutional Review Board (IRB), which is usually comprised of physicians and lay people. The IRB reviews the trial protocol (the study plan) to insure that it does not present unnecessary risk to the participants and that the participants’ rights are protected. Clinical trials are subject to the same ethical and legal codes as medical practices, and most trials are federally regulated.

A team of health care professionals—doctors, nurses, sometimes a social worker and other health care experts—works with the participants throughout the trial. The team checks the subjects’ health at the beginning and throughout the trial, provides information and instructions about the protocol, and administers and monitors the treatment during the trial. Participants usually have more frequent contact with the health care team than they might normally have with their physician.

Note that results of clinical trials are typically published in medical journals and elsewhere. Although the trials are confidential, they are not always anonymous. Be sure to ask any questions regarding confidentiality before participating in a clinical trial.

What are side effects and adverse reactions?

Side effects are any undesired actions or effects of the experimental drug or treatment. Negative or adverse effects may include headache, nausea, hair loss, skin irritation or other reactions. All experimental treatments must be evaluated for both immediate and long-term side effects.

Informed Consent

Informed consent is the process of learning the key facts about a clinical trial before deciding whether or not to participate. It is also a continuing process throughout the study to provide information for participants. To help someone decide whether or not to participate, the doctors and nurses involved in the trial explain the details of the study. All participants are required to sign an Informed Consent form which acknowledges participation, potential risks and explains the details of the trial including: the purpose, duration, experimental treatment; possible risks and benefits, tests that may be given, as well as information on treatments already available for your condition. Informed consent is not a contract, and the participant may withdraw from the trial at any time.

Locating a clinical trial

If you are interested in participating in or learning about a clinical trial, this comprehensive website, which is a service of the NIH, provides details on trials currently underway, those that are recruiting and/or completed.

Click below to learn more about active clinical trials for these illnesses:

Helpful search tips

To assist in selecting the appropriate trials for viewing, try using one of these search terms: scleroderma, Raynaud’s, systemic sclerosis, GERD, pulmonary fibrosis or pulmonary arterial hypertension.

You may also refine the search by limiting it to states, or countries, to which you are willing to travel. For example, if you would like to search for trials relating to scleroderma in San Francisco, enter scleroderma AND San Francisco in the search terms. You can also further target your search by selecting "Refine Search" and provide further specifics such as: recruitment status, age group, gender and phase of trial.

Search for clinical trials.