Artesunate at increasing doses for people with idiopathic pulmonary fibrosis

A Dose-Escalation Study Evaluating the Safety and Tolerability of Artesunate in Participants With Idiopathic Pulmonary Fibrosis (SAFE-IPF)

This will test whether taking artesunate by mouth at different doses is safe and tolerable for people with idiopathic pulmonary fibrosis.

Quick facts

• Phase: Phase 1
• Study type: Interventional
• Enrollment: 15 (estimated)
• Ages: 40 Years and up
• Sex: All
• Sponsor: Stanford University (Academic / other)
• Locations: 1 site (Stanford, California)
• Trial ID: NCT05988463 on ClinicalTrials.gov

What this trial studies

This is a single-center, randomized, double-blind, placebo-controlled Phase 1 study conducted at Stanford University. Participants undergo up to 4 weeks of screening, then 12 weeks of oral artesunate treatment given across three escalating dose levels with dose increases every 4 weeks, followed by a 4-week washout. The primary goal is to determine safety and tolerability and to select dose(s) for future testing, while secondary objectives include measuring blood biomarkers before and after treatment. Exploratory outcomes include changes in health-related quality of life and cough scores (K-BILD and Leicester Cough Questionnaire); participants on stable antifibrotic therapy are allowed.

Who should consider this trial

Good fit: Ideal candidates are adults aged 40 or older with a guideline-based diagnosis of IPF, FVC ≥40% predicted and DLco ≥30% predicted, who can attend Stanford for visits; people on stable nintedanib or pirfenidone are permitted.

Not a fit: Patients with very low lung function below entry thresholds, those unable to travel to Stanford, pregnant people, or those with contraindications to artesunate are unlikely to benefit.

Why it matters

Potential benefit: If safe and tolerable, the study could identify a dose of artesunate to advance to larger trials that might slow fibrosis or improve symptoms in people with IPF.

How similar studies have performed: Use of artesunate for IPF is novel in humans, with limited clinical data and mostly preclinical evidence suggesting possible anti-fibrotic effects.

Eligibility criteria

Inclusion Criteria:

1. Participants, aged 40 years or older.
2. Diagnosis of IPF based upon ATS/ERS/JRS/ALAT 2018 guidelines (55).
3. FVC percent of predicted ≥ 40%; historical FVC for entry in the study is permitted if within 3 months of screening.
4. Diffusing capacity of lung for carbon monoxide (DLco) (hemoglobin-adjusted) ≥ 30%; historical DLco for entry in the study is permitted if within 3 months of screening.
5. Participants currently receiving treatment for IPF with nintedanib or pirfenidone are allowed, provided these drugs have been given at a stable dose for at least 6 weeks before the Screening visit (stable dose is defined as the highest dose tolerated by the participant during ≥ 6 weeks).
6. Female participants of childbearing potential (i.e., ovulating, premenopausal, and not surgically sterile) and all male participants with sexual partners of childbearing potential must use highly effective methods of birth control during their participation in the study and for 60 days after the last administration of study drug. Highly effective methods of birth control are defined as those with 99% or greater efficacy.
7. Participants must agree to abstain from egg or sperm donation through 60 days, after administration of the last dose of study drug.
8. Able to read and sign a written informed consent form (ICF).

Exclusion Criteria:

1. Receiving any nonapproved agent intended for treatment of fibrosis in IPF or Participation in other clinical trials.
2. Clinical evidence of active infection, including but not limited to bronchitis, pneumonia, or sinusitis that can affect FVC measurement during screening.
3. Known acute IPF exacerbation or suspicion by the Investigator of such, within 3 months of screening.
4. The extent of emphysema is greater than the fibrotic changes on the most recent HRCT scan as determined by PI.
5. Any medical condition, not limited to cardiac, hepatic, renal disease or malignancy in recent months that will make the patients ineligible for the study, as deemed significant by PI.
6. Any of the following liver function test criteria above specified limits: total bilirubin \>2× the upper limit of normal (ULN); aspartate aminotransferase (AST) or alanine aminotransferase (ALT) \>3× ULN; alkaline phosphatase \> 2.5× ULN, pending PI's discretion.
7. Hemoglobin levels \< 10.0 g/dL.
8. Pregnant or lactating females.
9. Likely to have lung transplantation during the study (being on transplantation list is acceptable).
10. Currently receiving and expected to remain on treatment during the study with: amodiaquine, and efavirenz, nevirapine and ritonavir.

Where this trial is running

Stanford, California

• Stanford University — Stanford, California, United States (Recruiting)

Study contacts

• Principal investigator: Joshua Mooney, MD — Stanford University
• Study coordinator: Joseph Wu, M.D, Ph.D.

Conditions Idiopathic Pulmonary Fibrosis; shortness of breath, fibrosis, cough, hypoxemia