Ignite Creation Date:
2024-05-05 @ 11:23 AM
Last Modification Date:
2024-10-26 @ 9:06 AM
Study NCT ID:
NCT00013689
Status:
COMPLETED
Last Update Posted:
2008-03-04
First Post:
2001-03-28
Brief Title:
Pyrimethamine and Sulfadoxine for Treatment of Autoimmune Lymphoproliferative Syndrome
Sponsor:
National Institute of Allergy and Infectious Diseases NIAID
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
Pilot Study of Pyrimethamine and Sulfadoxine Fansidar for the Treatment of Individuals With the Autoimmune Lymphoproliferative Syndrome ALPS
Status:
COMPLETED
Status Verified Date:
2003-03
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
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 andor 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 patients private physician every 6 weeks or 2 and 4 weeks after the dose is increased to check for side effects
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 patients private physician every 6 weeks or 2 and 4 weeks after the dose is increased to check for side effects
Detailed Description:
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 pyrimethaminesulfadoxine 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 andor 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
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 andor 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
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 01-I-0132 | None | None | None |