Published Studies Related to Daraprim (Pyrimethamine)
Effects of amodiaquine and artesunate on sulphadoxine-pyrimethamine pharmacokinetic parameters in children under five in Mali. [2011.09.21]
BACKGROUND: Sulphadoxine-pyrimethamine, in combination with artesunate or amodiaquine, is recommended for the treatment of uncomplicated malaria and is being evaluated for intermittent preventive treatment. Yet, limited data is available on pharmacokinetic interactions between these drugs... CONCLUSION: Despite slight differences in pyrimethamine volumes of distribution and elimination half-life, these data show similar exposure to SP over the critical initial seven days of treatment and support the current use of SP in combination with either AQ or AS for uncomplicated falciparum malaria treatment in young Malian children.
Superiority of 3 over 2 doses of intermittent preventive treatment with sulfadoxine-pyrimethamine for the prevention of malaria during pregnancy in mali: a randomized controlled trial. [2011.08.01]
BACKGROUND: In 2003, Mali introduced intermittent preventive therapy in pregnancy (ITPp) with sulfadoxine-pyrimethamine (SP) for the control of malaria in pregnancy, consisting of 2 doses of SP given in the 2nd and 3rd trimester. This widely used regimen, although very effective, leaves many women unprotected from malaria during the last 4-to-8 weeks of gestation, which is a pivotal period for fetal weight gain. The aim of the study was to compare the efficacy and safety of 3-dose versus 2-dose IPTp-SP for the prevention of placental malaria and associated low birth weight (LBW)... CONCLUSIONS: Adding a third dose of ITPp-SP halved the risk of placental malaria, LBW, and preterm births in all gravidae, compared with the standard 2-dose regimen, in this area of highly seasonal transmission with low levels of SP resistance. Clinical Trials Registration: ISRCTN 74189211.
Pharmacokinetic properties of conventional and double-dose sulfadoxine-pyrimethamine given as intermittent preventive treatment in infancy. [2011.04]
Intermittent preventive treatment in infancy (IPTi) entails routine administration of antimalarial treatment doses at specified times in at-risk infants...
Population pharmacokinetics of sulfadoxine and pyrimethamine in Malawian children with malaria. [2011.02]
In addition to parasite resistance, inadequate levels of exposure to antimalarial drugs may contribute to treatment failure. We developed population pharmacokinetic (PK) models to describe the distribution of sulfadoxine (SDX) and pyrimethamine (PYM) in children with uncomplicated malaria in Malawi...
Randomized trial of intravitreal clindamycin and dexamethasone versus pyrimethamine, sulfadiazine, and prednisolone in treatment of ocular toxoplasmosis. [2011.01]
PURPOSE: To compare the efficacy of intravitreal injection of clindamycin and dexamethasone with classic treatment for ocular toxoplasmosis... CONCLUSIONS: Intravitreal injection of clindamycin and dexamethasone may be an acceptable alternative to the classic treatment in ocular toxoplasmosis. It may offer the patient more convenience, a safer systemic side effect profile, greater availability, and fewer follow-up visits and hematologic evaluations. Copyright (c) 2011 American Academy of Ophthalmology. Published by Elsevier Inc. All rights reserved.
Clinical Trials Related to Daraprim (Pyrimethamine)
Prevention of Congenital Toxoplasmosis With Pyrimethamine + Sulfadiazine Versus Spiramycine During Pregnancy [Recruiting]
Background : When a mother contracts toxoplasmosis during pregnancy, the parasite may be
transmitted from to her unborn child. This results in congenital toxoplasmosis, which may
cause damage to the eyes and nervous system of the child. To date, no method has been proved
effective to prevent this transmission. In France, spiramycin is usually prescribed to women
who have toxoplasma seroconversion in pregnancy, however its efficacy has not been
determined. The standard treatment for toxoplasmosis is the combination of the antiparasitic
drugs pyrimethamine and sulfadiazine, but this strategy has not been evaluated for the
prevention of mother-to-child transmission.
Purpose : Randomized phase 3 trial to determine whether pyrimethamine + sulfadiazine is
more effective than spiramycin to prevent congenital toxoplasmosis.
Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease) [Recruiting]
The objectives of this clinical trial are to assess the safety and tolerability, as well as
efficacy, of a stepwise dosing regimen of pyrimethamine, starting at 25 mg/day, given as a
single dose daily for 4 weeks in patients affected with chronic Tay-Sachs or Sandhoff
Pyrimethamine for the Treatment of Relapsed Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma [Recruiting]
In this research study we will start by looking for the highest dose of pyrimethamine that
can be given safely to CLL patients without severe or unmanageable side effects. This dose
will then be used for a larger Phase II study to assess the efficacy of pyrimethamine for
the treatment of CLL/SLL. Pyrimethamine is an antibiotic that is used for the treatment of
certain infections. Previous research studies have shown that pyrimethamine may target a
protein in tumor cells, called STAT3, which may be important for the growth of chronic
lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL) cells. Pyrimethamine can kill
CLL/SLL cells in the laboratory, and we are therefore undertaking this study to assess
whether pyrimethamine will result in clinical benefit or tumor responses in CLL in patients.
Impact IPT With Sulfadoxine-pyrimethamine or Sulfadoxine-pyrimethamine Plus Piperaquine in Schoolchildren [Recruiting]
Considering the facts that: (i) IPT of malaria provides substantial protection against
anaemia and malaria in school children (ii); SP resistance has no significant impact on the
prophylactic efficacy (iii) SP-PQ is safe and as efficacious as SP: the investigators
hypothesize that antimalarial IPT with SP and SP-PQ will improve haemoglobin concentration,
reduce anaemia prevalence, malaria incidence and parasitaemia, and improve malnutrition and
school performance in school-aged children of Congo.
Pyrimethamine to Treat Autoimmune Lymphoproliferative Syndrome [Completed]
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