CLINICAL TRIALS PROFILE FOR FANSIDAR

All Clinical Trials for Fansidar

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00013689 ↗	Pyrimethamine and Sulfadoxine for Treatment of Autoimmune Lymphoproliferative Syndrome	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 1	2001-03-01	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.
NCT00065390 ↗	Pyrimethamine to Treat Autoimmune Lymphoproliferative Syndrome	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 1	2003-07-01	This study will examine whether the drug pyrimethamine can shrink lymph nodes and spleen in patients with autoimmune lymphoproliferative syndrome (ALPS). In this disease, lymphocytes (white blood cells) do not die as they normally would. As a result, patients have enlarged lymph glands, spleen, or liver, and other problems that may involve blood cell counts and autoimmune disease (overactivity of the immune system). Pyrimethamine is an orally administered antibiotic that has been used to treat or prevent malaria and toxoplasma, and may be effective in shrinking lymph nodes and spleen. Patients with ALPS who are between 2 and 70 years of age and have had lymph gland enlargement for at least 1 year may be eligible for this study. Candidates will be screened with a medical history and physical examination, blood tests, and possibly a bone marrow test. Females of reproductive age will be screened with a urine pregnancy test. Women who are capable of becoming pregnant must use an effective method of birth control during the entire study period, because, taken during early months of pregnancy, pyrimethamine can cause birth defects in the fetus. Women who are pregnant or nursing are excluded from the study. Participants will undergo the following tests and procedures: - CT scan: For this test, the patient lies still in the CT scanner while images are taken of the neck, chest, and stomach area. A contrast dye is injected into a vein to brighten the CT images. Very young children will be evaluated on a case by case basis to determine whether a CT scan will be performed. - Bone marrow biopsy: Participants undergo this test to rule out underlying bone marrow disease if they have not had a bone marrow test done in the last six months prior to enrolling in pyrimethamine study, as pyrimethamine can affect bone marrow function. Under local anesthesia, a needle is inserted into the back part of the hipbone and a small amount of marrow is removed. (Children are sedated for this test.) - Leukapheresis: This is a procedure for collecting a small proportion of circulating white blood cells while conserving the majority of blood cells. Specifically, blood is drawn from a needle placed in an arm vein and is directed into a cell separator machine, which separates the blood cells by spinning. A small proportion of circulating white cells are removed, and the red cells, platelets, plasma and majority of white cells are returned to the patient's blood circulation. Only patients who are 7 years of age or older and weigh at least 55 pounds undergo this procedure. Other participants who choose not to have apheresis will have about 3 tablespoons of blood drawn instead. - Pyrimethamine administration: When the above tests are completed, participants begin taking pyrimethamine. The dose is determined according to the individual's weight and is gradually increased during the study period. Patients take the drug twice a week for a total of 12 weeks. - Blood tests: Blood samples are collected during weeks 2, 4, 6, 8, and 10 after beginning treatment, and 2 weeks after the last dose of pyrimethamine. The purpose of these blood tests is to check for possible drug-related side effects. Patients who develop a skin rash, mouth sores or other side effects may have one or more doses of the treatment drug withheld. When indicated, the patient will be directed to stop taking the study drug. If needed, drug side effects will be treated with a vitamin supplement, folinic acid, taken by mouth, 3 times weekly. - Evaluations at the NIH Clinical Center will comprise of a pretreatment visit, one end of treatment visit at the end of 12 weeks and an optional post-treatment visit 3months after stopping pyrimethamine therapy. Patients who respond well to treatment may be asked to return to NIH for additional visits at 3, 6, and 12 months after the treatment has ended for repeat evaluations. If their lymph glands or spleen become much larger after stopping pyrimethamine, they will be offered treatment for another 12 weeks. If they respond to the second course of treatment, they will return to NIH again after 3, 6, and 12 months. If the symptoms return again, patients will be asked to resume treatment for an additional 6 months or more. They will have blood drawn periodically by their private physician and will return to NIH for evaluation every 12 weeks.
NCT00146718 ↗	Anti-Malarial Drug Resistance in Cameroon	Completed	University of Yaounde	Phase 2/Phase 3	2003-08-01	The project is a three-armed study designed to evaluate the efficacy of amodiaquine(AQ), sulphadoxine-pyrimethamine(SP) and(AQ+SP) in three sites in Cameroon that differ in their baseline characteristics for malaria. In addition, drug resistance will be determined by measurement of blood drug levels,and identification of molecular markers of resistance.
NCT00146718 ↗	Anti-Malarial Drug Resistance in Cameroon	Completed	London School of Hygiene and Tropical Medicine	Phase 2/Phase 3	2003-08-01	The project is a three-armed study designed to evaluate the efficacy of amodiaquine(AQ), sulphadoxine-pyrimethamine(SP) and(AQ+SP) in three sites in Cameroon that differ in their baseline characteristics for malaria. In addition, drug resistance will be determined by measurement of blood drug levels,and identification of molecular markers of resistance.
NCT00158574 ↗	Kilimanjaro IPTi Drug Options Trial	Completed	Kilimanjaro Christian Medical Centre, Tanzania	Phase 2/Phase 3	2005-01-01	Malaria and anaemia are major causes of morbidity and mortality in children in sub-Saharan Africa. Administration of three courses of sulfadoxine/pyrimethamine (SP) as intermittent preventive treatment (IPTi) to infants when they receive EPI vaccines reduced the incidence of malaria and anaemia in infants in an area with low SP resistance, low transmission pressure and high bednet use. However, it is not clear whether this observation can be generalised to areas with high transmission and high SP resistance. The mechanism of the protective effect of IPTi is unclear. There is an urgent need to identify other anti-malarial drugs that could be used for IPTi instead of SP. This study objectives are: 1. Identification of a drug that could be used safely and effectively for IPTi instead of SP in areas, such as north eastern Tanzania, where there is a high level of resistance to SP and amodiaquine. 2. Determination of whether a short acting antimalarial drug (Lapdap) is as effective as a long acting drug (mefloquine) when used for IPTi. 3. Investigation of the effect of the intensity of transmission on the requirements for a long or short acting drug for IPTi. 4. Assessment of the effect of IPTi on the development of clinical immunity in children in low and high transmission areas. A randomised trial with four treatment regimes is proposed which will be conducted in two different transmission settings. The four treatment regimens are as follows: (1) placebo; (2) mefloquine; (3) Lapdap; (4) SP. All medications will be given at the time of immunisation with DPT/polio 2, DPT/polio 3, and measles vaccines. The study will involve 1280 infants in a high endemic area and 2440 infants in a low endemic area, in Tanzania.The primary outcome is the incidence of clinical malaria.
NCT00158574 ↗	Kilimanjaro IPTi Drug Options Trial	Completed	National Institute for Medical Research, Tanzania	Phase 2/Phase 3	2005-01-01	Malaria and anaemia are major causes of morbidity and mortality in children in sub-Saharan Africa. Administration of three courses of sulfadoxine/pyrimethamine (SP) as intermittent preventive treatment (IPTi) to infants when they receive EPI vaccines reduced the incidence of malaria and anaemia in infants in an area with low SP resistance, low transmission pressure and high bednet use. However, it is not clear whether this observation can be generalised to areas with high transmission and high SP resistance. The mechanism of the protective effect of IPTi is unclear. There is an urgent need to identify other anti-malarial drugs that could be used for IPTi instead of SP. This study objectives are: 1. Identification of a drug that could be used safely and effectively for IPTi instead of SP in areas, such as north eastern Tanzania, where there is a high level of resistance to SP and amodiaquine. 2. Determination of whether a short acting antimalarial drug (Lapdap) is as effective as a long acting drug (mefloquine) when used for IPTi. 3. Investigation of the effect of the intensity of transmission on the requirements for a long or short acting drug for IPTi. 4. Assessment of the effect of IPTi on the development of clinical immunity in children in low and high transmission areas. A randomised trial with four treatment regimes is proposed which will be conducted in two different transmission settings. The four treatment regimens are as follows: (1) placebo; (2) mefloquine; (3) Lapdap; (4) SP. All medications will be given at the time of immunisation with DPT/polio 2, DPT/polio 3, and measles vaccines. The study will involve 1280 infants in a high endemic area and 2440 infants in a low endemic area, in Tanzania.The primary outcome is the incidence of clinical malaria.
NCT00158574 ↗	Kilimanjaro IPTi Drug Options Trial	Completed	University of Copenhagen	Phase 2/Phase 3	2005-01-01	Malaria and anaemia are major causes of morbidity and mortality in children in sub-Saharan Africa. Administration of three courses of sulfadoxine/pyrimethamine (SP) as intermittent preventive treatment (IPTi) to infants when they receive EPI vaccines reduced the incidence of malaria and anaemia in infants in an area with low SP resistance, low transmission pressure and high bednet use. However, it is not clear whether this observation can be generalised to areas with high transmission and high SP resistance. The mechanism of the protective effect of IPTi is unclear. There is an urgent need to identify other anti-malarial drugs that could be used for IPTi instead of SP. This study objectives are: 1. Identification of a drug that could be used safely and effectively for IPTi instead of SP in areas, such as north eastern Tanzania, where there is a high level of resistance to SP and amodiaquine. 2. Determination of whether a short acting antimalarial drug (Lapdap) is as effective as a long acting drug (mefloquine) when used for IPTi. 3. Investigation of the effect of the intensity of transmission on the requirements for a long or short acting drug for IPTi. 4. Assessment of the effect of IPTi on the development of clinical immunity in children in low and high transmission areas. A randomised trial with four treatment regimes is proposed which will be conducted in two different transmission settings. The four treatment regimens are as follows: (1) placebo; (2) mefloquine; (3) Lapdap; (4) SP. All medications will be given at the time of immunisation with DPT/polio 2, DPT/polio 3, and measles vaccines. The study will involve 1280 infants in a high endemic area and 2440 infants in a low endemic area, in Tanzania.The primary outcome is the incidence of clinical malaria.

Clinical Trial Conditions for Fansidar

Condition Name

Condition Name for Fansidar
Intervention	Trials
Malaria	15
Malaria in Pregnancy	5
Pregnancy	4
HIV Infections	2

Condition MeSH

Condition MeSH for Fansidar
Intervention	Trials
Malaria	26
Malaria, Falciparum	5
Lymphoproliferative Disorders	2
Autoimmune Lymphoproliferative Syndrome	2

Clinical Trial Locations for Fansidar

Trials by Country

Trials by Country for Fansidar
Location	Trials
Kenya	8
Tanzania	7
Uganda	3
Malawi	3
Benin	2

Trials by US State

Trials by US State for Fansidar
Location	Trials
Maryland	2

Clinical Trial Progress for Fansidar

Clinical Trial Phase

Clinical Trial Phase for Fansidar
Clinical Trial Phase	Trials
Phase 4	8
Phase 3	9
Phase 2/Phase 3	3

Clinical Trial Status

Clinical Trial Status for Fansidar
Clinical Trial Phase	Trials
Completed	22
Terminated	4
Unknown status	4

Clinical Trial Sponsors for Fansidar

Sponsor Name

Sponsor Name for Fansidar
Sponsor	Trials
London School of Hygiene and Tropical Medicine	15
Radboud University	6
Kilimanjaro Christian Medical Centre, Tanzania	5

Sponsor Type

Sponsor Type for Fansidar
Sponsor	Trials
Other	93
U.S. Fed	4
Industry	3