Clinical Trials

The LGDA reviews the medical literature for current information concerning research into the causes and/or treatments of lymphangiomatosis and Gorham’s disease and reports this information on this web site and in our newsletter, Channels.

 

The LGDA provides this information as a resource only and receives no support or compensation for the listing of research programs. The information found on web sites to which LGDA provides links is beyond the control of the LGDA. The LGDA, its Board of Directors, and associates recommend you seek the guidance of your personal physician when making decisions regarding research or entering a clinical trial.

 

Frequently Asked Questions about Clinical Trials

What is a clinical trial?

A clinical trial is a research study conducted in people to determine whether a given treatment is safe and effective. Clinical trials are the best way for researchers to find new ways to diagnose, treat, and hopefully someday cure lymphangiomatosis and Gorham’s disease.

What are the types of clinical trials?

Interventional: A trial or study in which research subjects (patients) are assigned to receive a particular treatment (intervention) with response being documented by the researcher at specified intervals for a predetermined length of time, after which the results are compared to other subjects, usually patients who received a different intervention or no intervention at all. This type of trial may or may not be randomized. A randomized study is one in which subjects are assigned to one of two groups, the group receiving the intervention (usually a drug) and the group receiving a placebo (sugar pill) in random manner so that neither the subjects not the researchers knows to which group any given subject belongs.

Observational: studies in which health outcomes are assessed in pre-defined groups of individuals. Subjects in this type study may receive diagnostic, therapeutic, or other interventions, but the investigator does not assign specific interventions to the subjects of the study and has no control over the behavior of the subjects. Such studies are often undertaken to determine risk factors for certain illnesses and behaviors in groups of individuals.

What are the Phases of a Clinical Trial?

Preclinical: laboratories establish a scientific basis that a drug is reasonably safe and may be effective.

Phase I: The first stage of human testing. Typically involved fewer than 100 healthy volunteers. This phase look at the risks and side effects of a drug.

Phase II:  Up to a few hundred volunteers who have the condition the drug is designed to treat are enrolled. This phase provides further information about safety and help to determine the best dosage of a drug. Phase II trials are generally too small to provide clear evidence about a treatment’s benefit.

Phase III: Several hundred to thousands of volunteers are enrolled, often at multiple study sites worldwide. This phase provides the chief evidence for safety and effectiveness that the FDA will consider in deciding whether to approve a drug.

Phase IV: This phase, also called the post-marketing phase, is often required by the FDA after a drug is approved. During this phase, researchers continue to monitor the health of people taking the medication to gain further insight into its long-term safety and effectiveness.

What is informed consent?

The process of learning the key facts about a clinical trial before agreeing to participate is called informed consent. The FDA requires that potential research subjects (or their parent/legal guardian in the case of a child) be given complete information about the trial in writing. Part of this process includes a meeting with the researcher(s) during which the possible risks and benefits are explained and any questions are answered. If you decided to enter the trial you will be asked to sign a consent form. The form will also include the signature(s) of the person(s) who obtained your consent. You will receive a copy of the signed consent forms.

Anyone considering a clinical trial should feel free to ask any questions or bring up any issues concerning the trial at any time. The following suggestions* may give you some ideas as you think about your own questions.

The Study

  1. What is the purpose of the study?
  2. Why do researchers think the approach may be effective?
  3. Who will sponsor the study?
  4. Who has reviewed and approved the study?
  5. How are study results and safety of participants being checked?
  6. How long will the study last?
  7. What will my responsibilities be if I participate?

Possible Risks and Benefits

  1. What are my possible short-term benefits?
  2. What are my possible long-term benefits?
  3. What are my short-term risks, such as side effects?
  4. What are my possible long-term risks?
  5. What other options do people with my risk of cancer or type of cancer have?
  6. How do the possible risks and benefits of this trial compare with those options?

Participation and Care

  1. What kinds of therapies, procedures and /or tests will I have during the trial?
  2. Will they hurt, and if so, for how long?
  3. How do the tests in the study compare with those I would have outside of the trial?
  4. Will I be able to take my regular medications while in the clinical trial?
  5. Where will I have my medical care?
  6. Who will be in charge of my care?

Personal Issues

  1. How could being in this study affect my daily life?
  2. Can I talk to other people in the study?

Cost Issues

  1. Will I have to pay for any part of the trial such as tests or the study drug?
  2. If so, what will the charges likely be?
  3. What is my health insurance likely to cover?
  4. Who can help answer any questions from my insurance company or health plan?
  5. Will there be any travel or child care costs that I need to consider while I am in the trial?

Tips for Asking your Doctor About Trials

When you talk with your doctor or members of the research team:

  1. Consider taking a family member or friend along, for support and for help in asking questions or recording answers.
  2. Plan ahead what to ask–but don’t hesitate to ask any new questions you think of while you’re there.
  3. Write down your questions in advance, to make sure you remember to ask them all.
  4. Write down the answers, so that you can review them whenever you want.
  5. Consider bringing a tape recorder to make a taped record of what’s said (even if you write down answers).

*From  A Guide to Understanding Informed Consent from the National Cancer Institute at the National Institutes of Health.

Why should I (or my child) participate in a clinical trial?

Participating in clinical trial has the potential to help both the individual participant and other individuals suffering with lymphangiomatosis and Gorham’s disease. Participation in clinical trials also allows you to

  • play a more active role in your care;
  • gain access to potential treatments before they are widely available;
  • receive expert medical care at leading healthcare facilities — often free of cost — while participating in important medical research;
  • help future generations by contributing to lymphangiomatosis and Gorham’s disease research.

What is a multicenter research trial?

A multicenter research trial is a clinical trial conducted at more than one medical center or clinic. Most large clinical trials, particularly Phase III trials, are conducted at several institutions. There are many scientific benefits of multicenter trials, but for the patient wishing to participate in a clinical trial the most important likely is ease of access. The closer you live to a center involved in the trial, the easier it is for you to join the trial and gain access to the new treatment.

More information about clinical trials from Wikipedia
More information about clinical trials from ClinicalTrials.gov

Trials listed at ClinicalTrials.gov in which lymphangiomatosis & Gorham’s disease patients may be eligible to participate:

Safety and Efficacy Study of Sirolimus in Complicated Vascular Anomalies
Characterization of the Pathogenesis of Primary and Secondary Lymphatic Disorders
 Lymphatic Anomalies Registry