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DHFR Inhibitor

Pyrimethamine for Head and Neck Cancer

Phase < 1
Recruiting
Led By Paul Zolkind, M.D.
Research Sponsored by Washington University School of Medicine
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights
No Placebo-Only Group

Summary

This trial will evaluate the antitumor activity of pyrimethamine on human tumors, specifically its effects on DHFR and NRF2 pathway activity.

Who is the study for?
Adults with HPV-negative, locally advanced head and neck squamous cell carcinoma (HNSCC) who are fit for surgery can join. They must have good organ function, be able to swallow pills, and agree to use contraception. Excluded are those with recent anticoagulant use, allergies to similar drugs, active alcohol abuse, uncontrolled illnesses like heart disease or infections, certain blood disorders or other cancers treated within the last 2 years.
What is being tested?
The trial is testing Pyrimethamine's effect on tumors by inhibiting a protein called DHFR and reducing NRF2 activity which could help in treating cancer. The goal is to see if it increases DHFR levels in tumor cells by 50% and cuts down NRF2 activity by half in tumors that were already active before treatment.
What are the potential side effects?
Pyrimethamine may cause side effects such as allergic reactions for those sensitive to its components. Since it affects folate metabolism, there might be risks of blood disorders like megaloblastic anemia due to folate deficiency.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Side effects data

From 2016 Phase 1 & 2 trial • 32 Patients • NCT01083667
44%
Nausea
41%
Headache
19%
Diarrhea
16%
Pain
13%
Upper respiratory infection
9%
weight loss
6%
decreased appetite
3%
Pneumonia
100%
80%
60%
40%
20%
0%
Study treatment Arm
Pyrimethamine

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: PyrimethamineExperimental Treatment1 Intervention
Pyrimethamine will be taken by mouth at a dose of 50 mg once daily for 14 days (+/-2 days) with the last dose the day prior to surgery.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Pyrimethamine
2017
Completed Phase 2
~420

Find a Location

Who is running the clinical trial?

Tilde SciencesUNKNOWN
Washington University School of MedicineLead Sponsor
2,000 Previous Clinical Trials
2,344,238 Total Patients Enrolled
Vyera PharmaceuticalsUNKNOWN
Paul Zolkind, M.D.Principal InvestigatorWashington University School of Medicine

Media Library

Pyrimethamine (DHFR Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT05678348 — Phase < 1
Head and Neck Cancers Research Study Groups: Pyrimethamine
Head and Neck Cancers Clinical Trial 2023: Pyrimethamine Highlights & Side Effects. Trial Name: NCT05678348 — Phase < 1
Pyrimethamine (DHFR Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05678348 — Phase < 1
~9 spots leftby Apr 2026