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Recruiting Phase 3 NCT04851015

NCT04851015 Low Dose Trimethoprim-Sulfamethoxazole for the Treatment of Pneumocystis Jirovecii Pneumonia

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Clinical Trial Summary
NCT ID NCT04851015
Status Recruiting
Phase Phase 3
Sponsor Todd C. Lee MD MPH FIDSA
Condition Pneumocystis
Study Type INTERVENTIONAL
Enrollment 416 participants
Start Date 2025-11-28
Primary Completion 2030-09-30

Trial Parameters

Condition Pneumocystis
Sponsor Todd C. Lee MD MPH FIDSA
Study Type INTERVENTIONAL
Phase Phase 3
Enrollment 416
Sex ALL
Min Age 18 Years
Max Age 100 Years
Start Date 2025-11-28
Completion 2030-09-30
Interventions
trimethoprim-sulfamethoxazoletrimethoprim-sulfamethoxazole

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Brief Summary

Pneumocystis jirovecii pneumonia (PCP) is an opportunistic fungal infection of immunocompromised hosts which causes in significant morbidity and mortality. The current standard of care, trimethoprim-sulfamethoxazole (TMP-SMX) at a dose of 15-20 mg/kg/day of TMP, is associated with serious adverse events, including hypersensitivity reactions, drug-induced liver injury, cytopenia, and renal failure occurring among 20-60% of patients. The frequency of adverse events increases in a dose dependent manner and commonly limits the use of TMP-SMX. Reduced treatment doses of TMP-SMX for PCP reduced ADEs without mortality differences in a recent meta-analysis of observational studies. We therefore propose a Phase III randomized, placebo-controlled trial to directly compare the efficacy and safety of low dose (10 mg/kg/day of TMP) compared to the standard-of-care (15 mg/kg/day) among patients with PCP for the primary outcome of Win Ratio hierarchical composite of death, ECMO, invasive ventilation, grade 4 toxicity, non-invasive ventilation, change of therapy and length of stay.

Eligibility Criteria

Inclusion Criteria: * 18 years or older * Immunocompromised (including but not limited to HIV, solid organ transplant, solid tumors, hematological stem cell transplant and malignancies, systemic diseases, chemotherapy, long term corticosteroid use, and immunosuppressive therapies, as well as primary immunodeficiencies * Presentation to a day hospital, emergency department, or admitted to hospital * Proven or probable diagnosis of PCP using an adapted version of the 2021 EORTC/MSGERC criteria. Exclusion Criteria: * Previous severe adverse reaction to TMP-SMX, any sulfa drug, or any component of formulation * Compliant with PCP prophylaxis for ≥4 weeks with TMP-SMX at enrollment * More than 96 hours of any therapy for PCP * Hepatic impairment marked by alanine aminotransferase levels ≥5 times the upper limit of normal * Known G6PD deficiency * Known diagnosis of porphyria * Known pregnancy or breastfeeding (as per Health Canada) * Unable to provide informed consent and no available healt

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