The Official Patient's Sourcebook on Lupus (7 page)

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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,

Trials 35

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