Treating congenital toxoplasmosis with specific medications

Phase IV Randomized Study of Pyrimethamine, Sulfadiazine, and Leucovorin Calcium for Congenital Toxoplasmosis

Phase 4 Interventional Office of Rare Diseases (ORD) · NCT00004317

This study is testing the best way to use a combination of medications to treat congenital toxoplasmosis in infants and also looks at treating infected fetuses in pregnant women.

Quick facts

PhasePhase 4
Study typeInterventional
Enrollment600 (estimated)
SexAll
SponsorOffice of Rare Diseases (ORD) NIH
Drugs / interventionsprednisone
Locations1 site (Chicago, Illinois)
Trial IDNCT00004317 on ClinicalTrials.gov

What this trial studies

This phase IV trial aims to determine the most effective regimen of pyrimethamine when combined with sulfadiazine and leucovorin for treating congenital toxoplasmosis in infants. Participants are randomly assigned to one of two treatment groups, with variations in the dosing schedule of pyrimethamine over a 12-month period. The study also includes nonrandomized treatment for infected fetuses of pregnant women after the first trimester. Historical controls will be referred for comparison, focusing on untreated cases or those with minimal prior therapy.

Who should consider this trial

Good fit: Ideal candidates include infants diagnosed with congenital toxoplasmosis before 2.5 months of age and pregnant women with evidence of toxoplasma infection.

Not a fit: Patients who have already received extensive treatment for congenital toxoplasmosis or those without a confirmed diagnosis may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could lead to improved treatment protocols for congenital toxoplasmosis, potentially reducing the risk of severe complications in affected infants.

How similar studies have performed: Other studies have explored treatments for congenital toxoplasmosis, but this specific regimen and its effectiveness are being evaluated for the first time in this context.

Eligibility criteria

Show full inclusion / exclusion criteria
PROTOCOL ENTRY CRITERIA:

* Infants with congenital toxoplasmosis Toxoplasma gondii confirmed prior to age 2.5 months
* Pregnant women with evidence of toxoplasma infection by clinical observation and amniotic fluid sampling
* Acute infection acquired during gestation with evidence of fetal infection
* Untreated older children entered as controls
* Asymptomatic congenital toxoplasmosis
* Age more than 1 year
* No treatment within the first year of life
* No more than 1 month of prior therapy

Where this trial is running

Chicago, Illinois

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions Toxoplasmosisimmunologic disorders and infectious disordersrare diseasetoxoplasmosis
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.