Read The Official Patient's Sourcebook on Lupus Online
Authors: MD James N. Parker,PH.D Philip M. Parker
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Phase III.
Finally, researchers conduct Phase III trials to find out how new treatments for lupus nephritis 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 lupus nephritis
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 lupus nephritis.
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.”
Trials 31
This treatment, like a placebo, has no effect on lupus nephritis 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 lupus nephritis 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 lupus
nephritis. 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
32 Lupus Nephritis
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 Lupus Nephritis
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 lupus nephritis
.18 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.
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Immune System Related Kidney Disease
Condition(s): Glomerulonephritis; Lupus Nephritis; Membranous
Glomerulonephritis; Nephritis; Nephrotic Syndrome
Study Status: This study is currently recruiting patients.
Sponsor(s): National Institute of Diabetes and Digestive and Kidney
Diseases (NIDDK)
Purpose - Excerpt: Kidney diseases related to the immune system
include, nephrotic syndrome, glomerulonephritis, membranous
nephropathy, lupus nephritis, and nephritis associated with connective
tissue disorders. This study will allow researchers to admit and follow
patients suffering from autoimmune diseases of the kidney. It will
attempt to provide information about the causes and specific
abnormalities associated with autoimmune kidney disease. Patients with
kidney disease as a result of their immune system, and patients with
diseases of the immune system who may later develop kidney disease,
will be potential subjects for this study. Patients will undergo a history
18 These are listed at
www.ClinicalTrials.gov
.
Trials 33
and physical examination, and standard laboratory test to more closely
understand the causes, signs, symptoms, and responses to medication of
these diseases. Based on these evaluations the patients may qualify as
candidates for other experimental studies. At any time these patients may
be asked to submit blood or urine samples for further research.
Study Type: Observational
Contact(s): Maryland; National Institute of Diabetes and Digestive and
Kidney Diseases (NIDDK), 9000 Rockville Pike Bethesda, Maryland,
20892, United States; Recruiting; Patient Recruitment and Public Liaison
Office 1-800-411-1222 [email protected]; TTY 1-866-411-1010
Web Site: http://clinicaltrials.gov/ct/gui/show/NCT00001979
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Safety and Efficacy Study of LJP 394 (abetimus sodium) to treat lupus
kidney disease
Condition(s): Immunologic Diseases; Autoimmune Diseases; Systemic
Lupus Erythematosus; Lupus Nephritis; Lupus Glomerulonephritis
Study Status: This study is currently recruiting patients.
Sponsor(s): La Jolla Pharmaceutical Company
Purpose - Excerpt: The purpose of this study is to determine whether LJP
394 (abetimus sodium) is safe and effective in delaying and reducing
renal flares in patients with lupus nephritis.
Phase(s): Phase III
Study Type: Interventional
Contact(s): see Web site below
Web Site: http://clinicaltrials.gov/ct/gui/show/NCT00035308
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Study of Systemic Lupus Erythematosus
Condition(s): Lupus Nephritis; Systemic Lupus Erythematosus
Study Status: This study is currently recruiting patients.
Sponsor(s): National Institute of Arthritis and Musculoskeletal and Skin
Diseases (NIAMS)
Purpose - Excerpt: This protocol will evaluate patients with systemic
lupus erythematosus (SLE) and their relatives to learn more about how
the disease develops and changes over time. It will also study genetic
factors that make a person susceptible to SLE. Patients 10 years of age
and older with known or suspected SLE and their relatives may be
eligible for this study. Patients will be evaluated with a medical history
and physical examination, blood and urine tests. Other procedures may
34 Lupus Nephritis
include: 1. Electrocardiogram 2. 24-hour urine collection 3. Imaging
studies, such as chest and joint X-rays, magnetic resonance imaging
(MRI) scans, bone scans, and bone densitometry. 4. Questionnaire about
the degree of disease activity, and survey of risk factors for disease
complications. 5. Apheresis-Collection of plasma (fluid portion of blood)
or blood cells for analysis. Whole blood is collected through a needle in
an arm vein. The blood circulates through a machine that separates it into
its components. The required component (plasma or cells) is removed
and the rest of the blood is returned to the body through the same needle
or through a second needle in the other arm. 6. Skin biopsy-Removal of a
small skin sample for microscopic analysis. An area of skin is numbed
with an anesthetic and a small circular portion (about 1/4 inch in
diameter) is removed, using a sharp cookie cutter-type instrument. 7.
Kidney, bone marrow or other organ biopsy-Removal of a small sample
of organ tissue. These biopsies are done only if they can provide
information useful in better understanding the disease or making
treatment decisions. 8. Genetic studies-Collection of a blood sample for
gene testing. Patients will be followed at least once a year with a brief
history and physical examination and routine blood and urine tests.
Some patients may be seen more often. Treatment recommendations will
be offered to patients' physicians, and patients who are eligible for other
research treatment studies will be invited to enroll. Participating relatives
of patients will fill out a brief medical history questionnaire and provide
a DNA sample (either a blood sample or tissue swab from the inside of
the cheek) for genetic testing.
Study Type: Observational
Contact(s): Maryland; National Institute of Arthritis and Musculoskeletal
and Skin Diseases (NIAMS), 9000 Rockville Pike Bethesda, Maryland,
20892, United States; Recruiting; Patient Recruitment and Public Liaison
Office 1-800-411-1222 [email protected]; TTY 1-866-411-1010
Web Site: http://clinicaltrials.gov/ct/gui/show/NCT00001372
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Cyclophosphamide and Fludarabine to Treat Lupus Nephritis
Condition(s): Glomerulonephritis; Lupus Nephritis; Systemic Lupus
Erythematosus
Study Status: This study is no longer recruiting patients.
Sponsor(s): National Institute of Arthritis and Musculoskeletal and Skin
Diseases (NIAMS)
Purpose - Excerpt: This study will test the safety and effectiveness of
combination therapy with cyclophosphamide (Cytoxan) and fludarabine
in treating lupus nephritis (kidney inflammation). This condition,
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common in patients with systemic lupus erythematosus, is caused by
abnormal action of immune cells called lymphocytes against the kidneys.
Left untreated, severe cases can result in loss of kidney function. The
current treatment of choice-intermittent high doses (pulses) of
cyclophosphamide-does not work in all patients and causes infertility in
many women. The rate of infertility in men is not known. This study will
examine whether fludarabine can safely be given with significantly lower
doses of cyclophosphamide, and if this combination controls kidney
inflammation. Patients 18 years of age and older with severe lupus
nephritis (called proliferative lupus nephritis) may be eligible for this
study. Candidates will have a history and physical examination; blood
and urine tests; chest X-ray; electrocardiogram; cancer screening that may
include a Pap smear, mammogram, rectal examination, PSA testing, and
sigmoidoscopy. Participants will be divided into one of the following
treatment groups: Group 1-Patients undergo three treatment cycles of
cyclophosphamide, taken by mouth, and fludarabine, injected
subcutaneously (under the skin). Patients receive both drugs on day 1 of
the cycle, and fludarabine alone on days 2 and 3. This regimen is