Clinical Trials and Raynauds Phenomenon

Overview

Very few medical conditions have a single treatment. The basic treatment guidelines that your physician has discussed with you, or those that you have found using the techniques discussed in Chapter 1, may provide you with all that you will require. For some patients, current treatments can be enhanced with new or innovative techniques currently under investigation. In this chapter, we will describe how clinical trials work and show you how to keep informed of trials concerning Raynaud's phenomenon.

What Is a Clinical Trial?15

Clinical trials involve the participation of people in medical research. Most medical research begins with studies in test tubes and on animals. Treatments that show promise in these early studies may then be tried with people. The only sure way to find out whether a new treatment is safe, effective, and better than other treatments for Raynaud's phenomenon is to try it on patients in a clinical trial.

15 The discussion in this chapter has been adapted from the NIH and the NEI: www.nei.nih.gov/netrials/ctivr.htm.

What Kinds of Clinical Trials Are There?

Clinical trials are carried out in three phases:

• Phase I. Researchers first conduct Phase I trials with small numbers of patients and healthy volunteers. If the new treatment is a medication, researchers also try to determine how much of it can be given safely.

• Phase II. Researchers conduct Phase II trials in small numbers of patients to find out the effect of a new treatment on Raynaud's phenomenon.

• Phase III. Finally, researchers conduct Phase III trials to find out how new treatments for Raynaud's phenomenon compare with standard treatments already being used. Phase III trials also help to determine if new treatments have any side effects. These trials—which may involve hundreds, perhaps thousands, of people--can also compare new treatments with no treatment.

How Is a Clinical Trial Conducted?

Various organizations support clinical trials at medical centers, hospitals, universities, and doctors' offices across the United States. The "principal investigator" is the researcher in charge of the study at each facility participating in the clinical trial. Most clinical trial researchers are medical doctors, academic researchers, and specialists. The "clinic coordinator" knows all about how the study works and makes all the arrangements for your visits.

All doctors and researchers who take part in the study on Raynaud's phenomenon carefully follow a detailed treatment plan called a protocol. This plan fully explains how the doctors will treat you in the study. The "protocol" ensures that all patients are treated in the same way, no matter where they receive care.

Clinical trials are controlled. This means that researchers compare the effects of the new treatment with those of the standard treatment. in some cases, when no standard treatment exists, the new treatment is compared with no treatment. Patients who receive the new treatment are in the treatment group. Patients who receive a standard treatment or no treatment are in the "control" group. In some clinical trials, patients in the treatment group get a new medication while those in the control group get a placebo. A placebo is a harmless substance, a "dummy" pill, that has no effect on Raynaud's phenomenon. in other clinical trials, where a new surgery or device (not a medicine) is being tested, patients in the control group may receive a "sham treatment." This treatment, like a placebo, has no effect on Raynaud's phenomenon and does not harm patients.

Researchers assign patients "randomly" to the treatment or control group. This is like flipping a coin to decide which patients are in each group. If you choose to participate in a clinical trial, you will not know which group you will be appointed to. The chance of any patient getting the new treatment is about 50 percent. You cannot request to receive the new treatment instead of the placebo or sham treatment. Often, you will not know until the study is over whether you have been in the treatment group or the control group. This is called a "masked" study. In some trials, neither doctors nor patients know who is getting which treatment. This is called a "double masked" study. These types of trials help to ensure that the perceptions of the patients or doctors will not affect the study results.

Natural History Studies unlike clinical trials in which patient volunteers may receive new treatments, natural history studies provide important information to researchers on how Raynaud's phenomenon develops over time. A natural history study follows patient volunteers to see how factors such as age, sex, race, or family history might make some people more or less at risk for Raynaud's phenomenon. A natural history study may also tell researchers if diet, lifestyle, or occupation affects how a disease or disorder develops and progresses. Results from these studies provide information that helps answer questions such as: How fast will a disease or disorder usually progress? How bad will the condition become? Will treatment be needed?

What Is Expected of Patients in a Clinical Trial?

Not everyone can take part in a clinical trial for a specific disease or disorder. Each study enrolls patients with certain features or eligibility criteria. These criteria may include the type and stage of disease or disorder, as well as, the age and previous treatment history of the patient. You or your doctor can contact the sponsoring organization to find out more about specific clinical trials and their eligibility criteria. If you are interested in joining a clinical trial, your doctor must contact one of the trial's investigators and provide details about your diagnosis and medical history.

If you participate in a clinical trial, you may be required to have a number of medical tests. You may also need to take medications and/ or undergo surgery. Depending upon the treatment and the examination procedure, you may be required to receive inpatient hospital care. Or, you may have to return to the medical facility for follow-up examinations. These exams help find out how well the treatment is working. Follow-up studies can take months or years. However, the success of the clinical trial often depends on learning what happens to patients over a long period of time. Only patients who continue to return for follow-up examinations can provide this important long-term information.

Recent Trials on Raynaud's Phenomenon

The National Institutes of Health and other organizations sponsor trials on various diseases and disorders. Because funding for research goes to the medical areas that show promising research opportunities, it is not possible for the NIH or others to sponsor clinical trials for every disease and disorder at all times. The following lists recent trials dedicated to Raynaud's phenomenon.16 If the trial listed by the NIH is still recruiting, you may be eligible. If it is no longer recruiting or has been completed, then you can contact the sponsors to learn more about the study and, if published, the results. Further information on the trial is available at the Web site indicated. Please note that some trials may no longer be recruiting patients or are otherwise closed. Before contacting sponsors of a clinical trial, consult with your physician who can help you determine if you might benefit from participation.

• Safety and efficacy of Pletal(cilostazol) for the treatment of juvenile primary and secondary Raynaud's phenomenon

Condition(s): Secondary Raynaud's

Study Status: This study is currently recruiting patients.

Sponsor(s): Otsuka America Pharmaceutical

Purpose - Excerpt: Juvenile secondary Raynaud's (ray-knows) Phenomenon is a disorder of the blood vessels in the fingers and sometimes can affect the toes, nose, or ears. Children with secondary Raynaud's phenomenon have an underlying condition such as systemic lupus, scleroderma, or mixed connective tissue disease. When children with secondary Raynaud's are exposed to chilly or cold conditions from weather, cold temperatures, or even holding cold items from the refrigerator, their fingers may become cold, numb, hurt, and/or turn purple or white. The investigational drug, Pletal(cilostazol), which has been approved for other conditions, inhibits the ability of one type of

16 These are listed at www.ClinicalTrials.gov.

blood cell, platelets, to form blood clots, and also widens narrowed blood vessels. It has been used in a variety of other conditions in which blood flow is decreased. This study will test the safety and effectiveness Pletal(cilostazol) to lessen the severity of the symptoms and decrease the number of secondary Raynaud's episodes in juvenile patients.

Phase(s): Phase IV; MEDLINEplus consumer health information

Study Type: Interventional

Contact(s): see Web site below

Web Site:

http://clinicaltrials.gov/ct/gui/show/NCT00048776;jsessionid=C421D4 379F7646F759378AE8A0CFD2E5

• Phase II Study of Recombinant Relaxin for Progressive Systemic Sclerosis

Condition(s): Systemic Sclerosis Study Status: This study is completed.

Sponsor(s): National Center for Research Resources (NCRR); National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS); Stanford University

Purpose - Excerpt: Objectives: I. Determine whether parenteral relaxin improves skin tightness, Raynaud's phenomenon, digital morbidity, and digital ulcers in a patient with progressive systemic sclerosis (scleroderma). II. Determine whether relaxin decreases collagen production by fibroblasts in vivo and cultured from skin biopsies.

Phase(s): Phase II

Study Type: Interventional

Contact(s):. Study chairs or principal investigators: G. Scott Herron, Study Chair; Stanford University

Web Site:

http://clinicaltrials.gov/ct/gui/show/NCT00004380;jsessionid=C421D4 379F7646F759378AE8A0CFD2E5

• Phase III Randomized, Double-Blind, Placebo-Controlled Study of Oral Iloprost for Raynaud's phenomenon Secondary to Systemic Sclerosis

Condition(s): Systemic Sclerosis; Raynaud Disease Study Status: This study is completed.

Sponsor (s): National Center for Research Resources (NCRR); University of pittsburgh purpose - Excerpt: objectives: i. Evaluate the safety and efficacy of oral iloprost, a prostacyclin analog, in patients with Raynaud's phenomenon secondary to systemic sclerosis.

Phase(s): Phase III

Study Type: Interventional

Contact(s):. Study chairs or principal investigators: Thomas A. Medsger, Jr., Study Chair; University of Pittsburgh

Web Site:

http://clinicaltrials.gov/ct/gui/show/NCT00004786;jsessionid=C421D4 379F7646F759378AE8A0CFD2E5

• Raynaud's Treatment Study (RTS)

Condition(s): Cardiovascular Diseases; Heart Diseases; Raynaud's Disease; Vascular Diseases

Study Status: This study is completed.

Sponsor(s): National Heart, Lung, and Blood institute (NHLBi)

Purpose - Excerpt: To determine the relative efficacy of usual medical care and a course of treatment by thermal biofeedback in reducing vasospastic attacks characteristic of Raynaud's syndrome. Also, to confirm the frequency and severity of attacks, examine the role of psychophysiological factors in precipitating attacks, and assess the influence of treatment on health quality of life.

Phase(s): Phase III

Study Type: interventional

Contact(s):. Study chairs or principal investigators: Leonard Bielory; University of Medicine and Dentistry of New Jersey; Robert Freedman; Wayne State University; Rolf Jacob; University of Pittsburgh; Edwin Smith; Medical University of South Carolina; Frederick Wigley; Johns Hopkins University

Web Site:

http://clinicaltrials.gov/ct/gui/show/NCT00000530;jsessionid=C421D4 379F7646F759378AE8A0CFD2E5

Benefits and Risks17

What Are the Benefits of Participating in a Clinical Trial?

If you are interested in a clinical trial, it is important to realize that your participation can bring many benefits to you and society at large:

• A new treatment could be more effective than the current treatment for Raynaud's phenomenon. Although only half of the participants in a clinical trial receive the experimental treatment, if the new treatment is proved to be more effective and safer than the current treatment, then those patients who did not receive the new treatment during the clinical trial may be among the first to benefit from it when the study is over.

• If the treatment is effective, then it may improve health or prevent diseases or disorders.

• Clinical trial patients receive the highest quality of medical care. Experts watch them closely during the study and may continue to follow them after the study is over.

• People who take part in trials contribute to scientific discoveries that may help other people with Raynaud's phenomenon. In cases where certain diseases or disorders run in families, your participation may lead to better care or prevention for your family members.

The Informed Consent

Once you agree to take part in a clinical trial, you will be asked to sign an "informed consent." This document explains a clinical trial's risks and benefits, the researcher's expectations of you, and your rights as a patient.

What Are the Risks?

Clinical trials may involve risks as well as benefits. Whether or not a new treatment will work cannot be known ahead of time. There is always a chance that a new treatment may not work better than a standard treatment. There is also the possibility that it may be harmful. The treatment you receive may cause side effects that are serious enough to require medical attention.

17 This section has been adapted from ClinicalTrials.gov, a service of the National Institutes of Health:

http;//www.clinicaltrials.gov/ct/gui/c/a1r/info/whatis?JServSessionIdzone_ct=9jmun6f291.

How Is Patient Safety Protected?

Clinical trials can raise fears of the unknown. Understanding the safeguards that protect patients can ease some of these fears. Before a clinical trial begins, researchers must get approval from their hospital's Institutional Review Board (IRB), an advisory group that makes sure a clinical trial is designed to protect patient safety. During a clinical trial, doctors will closely watch you to see if the treatment is working and if you are experiencing any side effects. All the results are carefully recorded and reviewed. In many cases, experts from the Data and Safety Monitoring Committee carefully monitor each clinical trial and can recommend that a study be stopped at any time. You will only be asked to take part in a clinical trial as a volunteer giving informed consent.

What Are a Patient's Rights in a Clinical Trial?

If you are eligible for a clinical trial, you will be given information to help you decide whether or not you want to participate. As a patient, you have the right to:

• Information on all known risks and benefits of the treatments in the study.

• Know how the researchers plan to carry out the study, for how long, and where.

• Know any costs involved for you or your insurance provider.

• Know before any of your medical or personal information is shared with other researchers involved in the clinical trial.

• Talk openly with doctors and ask any questions. After you join a clinical trial, you have the right to:

• Leave the study at any time. Participation is strictly voluntary. However, you should not enroll if you do not plan to complete the study.

• Receive any new information about the new treatment.

• Continue to ask questions and get answers.

• Maintain your privacy. Your name will not appear in any reports based on the study.

• Know whether you participated in the treatment group or the control group (once the study has been completed).

What about Costs?

In some clinical trials, the research facility pays for treatment costs and other associated expenses. You or your insurance provider may have to pay for costs that are considered standard care. These things may include inpatient hospital care, laboratory and other tests, and medical procedures. You also may need to pay for travel between your home and the clinic. You should find out about costs before committing to participation in the trial. If you have health insurance, find out exactly what it will cover. If you don't have health insurance, or if your insurance company will not cover your costs, talk to the clinic staff about other options for covering the cost of your care.

What Questions Should You Ask before Deciding to Join a Clinical Trial?

Questions you should ask when thinking about joining a clinical trial include the following:

• What is the purpose of the clinical trial?

• What are the standard treatments for Raynaud's phenomenon? Why do researchers think the new treatment may be better? What is likely to happen to me with or without the new treatment?

• What tests and treatments will I need? Will I need surgery? Medication? Hospitalization?

• How long will the treatment last? How often will I have to come back for follow-up exams?

• What are the treatment's possible benefits to my condition? What are the short- and long-term risks? What are the possible side effects?

• Will the treatment be uncomfortable? Will it make me feel sick? If so, for how long?

• How will my health be monitored?

• Where will I need to go for the clinical trial? How will I get there?

• How much will it cost to be in the study? What costs are covered by the study? How much will my health insurance cover?

• Will I be able to see my own doctor? Who will be in charge of my care?

• Will taking part in the study affect my daily life? Do I have time to participate?

• How do I feel about taking part in a clinical trial? Are there family members or friends who may benefit from my contributions to new medical knowledge?

Keeping Current on Clinical Trials

Various government agencies maintain databases on trials. The U.S. National institutes of Health, through the National Library of Medicine, has developed ClinicalTrials.gov to provide patients, family members, and physicians with current information about clinical research across the broadest number of diseases and conditions.

The site was launched in February 2000 and currently contains approximately 5,700 clinical studies in over 59,000 locations worldwide, with most studies being conducted in the united states. ClinicalTrials.gov receives about 2 million hits per month and hosts approximately 5,400 visitors daily. To access this database, simply go to their Web site (www.clinicaltrials.gov) and search by "Raynaud's phenomenon" (or synonyms).

While ClinicalTrials.gov is the most comprehensive listing of NIH-supported clinical trials available, not all trials are in the database. The database is updated regularly, so clinical trials are continually being added. The following is a list of specialty databases affiliated with the National institutes of Health that offer additional information on trials:

• For clinical studies at the Warren Grant Magnuson Clinical Center located in Bethesda, Maryland, visit their Web site: http://clinicalstudies.info.nih.gov/

• For clinical studies conducted at the Bayview Campus in Baltimore, Maryland, visit their Web site: http://www.jhbmc.jhu.edu/studies/index.html

• For trials on arthritis, musculoskeletal and skin diseases, visit newly revised site of the National Institute of Arthritis and Musculoskeletal and

Skin Diseases of the National Institutes of Health: http://www.niams.nih.gov/hi/studies/index.htm

General References

The following references describe clinical trials and experimental medical research. They have been selected to ensure that they are likely to be available from your local or online bookseller or university medical library. These references are usually written for healthcare professionals, so you may consider consulting with a librarian or bookseller who might recommend a particular reference. The following includes some of the most readily available references (sorted alphabetically by title; hyperlinks provide rankings, information and reviews at Amazon.com):

• A Guide to Patient Recruitment : Today's Best Practices & Proven Strategies by Diana L. Anderson; Paperback - 350 pages (2001), CenterWatch, Inc.; ISBN: 1930624115;

http://www.amazon.com/exec/obidos/ASIN/1930624115/icongroupinterna

• A Step-By-Step Guide to Clinical Trials by Marilyn Mulay, R.N., M.S., OCN; Spiral-bound - 143 pages Spiral edition (2001), Jones & Bartlett Pub; ISBN: 0763715697;

http://www.amazon.com/exec/obidos/ASIN/0763715697/icongroupinterna

• The CenterWatch Directory of Drugs in Clinical Trials by CenterWatch; Paperback - 656 pages (2000), CenterWatch, Inc.; ISBN: 0967302935; http://www.amazon.com/exec/obidos/ASIN/0967302935/icongroupinterna

• The Complete Guide to Informed Consent in Clinical Trials by Terry Hartnett (Editor); Paperback - 164 pages (2000), PharmSource Information Services, Inc.; ISBN: 0970153309;

http://www.amazon.com/exec/obidos/ASIN/0970153309/icongroupinterna

• Dictionary for Clinical Trials by Simon Day; Paperback - 228 pages (1999), John Wiley & Sons; ISBN: 0471985961;

http://www.amazon.com/exec/obidos/ASIN/047198596Vicongroupinterna

• Extending Medicare Reimbursement in Clinical Trials by Institute of Medicine Staff (Editor), et al; Paperback 1st edition (2000), National Academy Press; ISBN: 0309068886;

http://www.amazon.com/exec/obidos/ASIN/0309068886/icongroupinterna

• Handbook of Clinical Trials by Marcus Flather (Editor); Paperback (2001), Remedica Pub Ltd; ISBN: 1901346293;

http://www.amazon.com/exec/obidos/ASIN/1901346293/icongroupinterna

Vocabulary Builder

The following vocabulary builder gives definitions of words used in this chapter that have not been defined in previous chapters:

Cardiovascular: Pertaining to the heart and blood vessels. [eu]

Fibroblasts: Connective tissue cells which secrete an extracellular matrix rich in collagen and other macromolecules. [nih]

Parenteral: Not through the alimentary canal but rather by injection through some other route, as subcutaneous, intramuscular, intraorbital, intracapsular, intraspinal, intrasternal, intravenous, etc. [eu]

Recombinant: 1. a cell or an individual with a new combination of genes not found together in either parent; usually applied to linked genes. [eu]

Thermal: Pertaining to or characterized by heat. [eu]

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