Lymphoproliferative Disorder Clinical Trial
Official title:
Pilot Study of Pyrimethamine and Sulfadoxine (Fansidar) for the Treatment of Individuals With the Autoimmune Lymphoproliferative Syndrome (ALPS)
This study will evaluate the safety and effectiveness of an antibiotic called Fansidar on
autoimmune lymphoproliferative syndrome (ALPS). Patients with ALPS have enlarged lymph
glands, spleen and/or liver, abnormal blood cell counts and overactive immune function.
Current treatments are aimed at suppressing the immune system and improving symptoms, such
as anemia (low red blood cell count) and low white blood cell and platelet counts. These
treatments, however, are only partially effective and may have complications. Fansidar is a
combination of two drugs, sulfadoxine and pyrimethamine, that is used to treat or prevent
parasitic infections such as malaria. Recently a child with ALPS who was treated with
Fansidar for a different illness had a marked shrinkage of the lymph organs. This study will
examine whether Fansidar can shrink the lymph glands or spleen in patients with ALPS.
Patients with ALPS between the ages of 4 and 70 years who have had lymph gland enlargement
for at least 1 year and are not allergic to sulfa drugs may be eligible for this study.
Candidates will be screened with a medical history and physical examination and blood tests.
Females of reproductive age will have a urine pregnancy test.
Participants will be evaluated at the NIH Clinical Center in Bethesda, MD, with blood tests
and a computed tomography (CT) scan of the lymph nodes. For the CT scan, the patient lies on
a table during an X-ray scan of the neck, part of the chest, and, if the spleen has not been
removed, the stomach area.
When these baseline tests are completed, patients will be given Fansidar pills to take once
a week for 12 weeks. The dosage will be increased after 2 weeks and again after 4 weeks. At
2, 4, 6, 8 and 10 weeks after starting the treatment and 2 weeks after the last dose,
patients will have blood drawn to check for possible side effects of therapy. Women will
have a repeat urine pregnancy test at week 6 of treatment.
Within a week before completing treatment or after completing treatment, patients will
return to NIH for a history, physical examination, blood tests and CT scan. Patients who
responded well to treatment will be offered to return to NIH again 3, 6 and 12 months later
to repeat the evaluations. If ALPS symptoms recur during this time, patients will be offered
another 12-week course of Fansidar and the procedure, including the 3, 6 and 12-month
evaluations will be repeated again. If symptoms recur again, patients will be asked to
resume Fansidar for 6 months or longer, with doses adjusted as needed. During this time,
patients will be seen at NIH every 12 weeks for evaluation and blood will be drawn by the
patient's private physician every 6 weeks or 2 and 4 weeks after the dose is increased to
check for side effects.
The Autoimmune Lymphoproliferative Syndrome is an inherited disease associated with a defect
of lymphocyte apoptosis, lymphoproliferation and autoimmunity. Although, there are
treatments for many of its autoimmune complications, there currently is no safe and
effective therapy for this syndrome itself. Recently investigators in Europe serendipitously
found that a child with ALPS type I had significant clinical improvement while on
pyrimethamine/sulfadoxine (Fansidar) for Pneumocystis carinii prophylaxis.
Based on this finding, we propose to conduct a pilot study to obtain information on safety
and initial data on efficacy of the drug combination Fansidar for the treatment of ALPS. Six
to 8 individuals, with ALPS who report no allergy to sulfa drugs will be treated for up to 3
months with weekly Fansidar at escalating doses adjusted by weight. The effect of Fansidar
treatment on lymph node and/or spleen size will be assessed by CT scanning. The effect of
treatment on other laboratory features of ALPS will also be assessed. Evaluating the effects
of Fansidar on these clinical and laboratory parameters will allow us to determine if this
drug demonstrates sufficient activity to warrant further study in a larger randomized
controlled trial.
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Endpoint Classification: Safety Study, Primary Purpose: Treatment
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