Phase 2 Rare Disease Trial, Terminated NCT04240886 Sponsor: Basilea Pharmaceutica Condition: Rare Disease
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Trial results

AI generated results summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI

According to the results reported on ClinicalTrials.gov, this trial (NCT04240886) enrolled 21 adults who received the study drug fosmanogepix (also called APX001) as part of Cohort A. The trial was investigating a treatment for serious fungal infections, and it tracked what happened to participants over roughly six weeks (up to Day 42). Of the 21 people who started the treatment phase, 11 completed it and 10 did not. The trial measured things like survival, how the fungal infection responded, and what medical events occurred during the study. The reported data shows that 25% of participants (that is, roughly 1 in 4) died at some point between their first dose and Day 42. When an independent review committee assessed how each participant's fungal infection had responded by the end of their treatment, the reported data shows that 20% had a complete response (all signs of infection resolved), 20% had a partial response (improvement but not full resolution), 10% had a stable response (little change), 30% had disease progression (the infection got worse), and 20% had died. Combining these categories, 40% were counted as "treatment successes" (complete or partial response) and 60% were counted as "treatment failures" (stable, worsening, or death). The reported data also shows that all 21 participants experienced at least one adverse event (an unexpected medical occurrence during the study), and 13 of the 21 experienced a serious adverse event — meaning a medical event considered significant, such as one requiring hospitalisation. Five participants had a clinically significant abnormality in vital signs (such as blood pressure, heart rate, or temperature), and 12 participants had a clinically significant abnormality in laboratory test results. These figures describe what was observed and recorded; they do not on their own tell us what caused these events. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

These are the results as reported to ClinicalTrials.gov, not medical advice. Verify independently with the trial site and discuss what they mean for you with your doctor.

Phase 2 Rare Disease Trial, Terminated

NCT04240886
Terminated Phase 2

Who may be able to join

AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI

Who might be able to join this trial:

  • You are a man or woman aged 18 or older.
  • You have been diagnosed with, or are strongly suspected to have, a serious invasive fungal infection caused by mold, such as Aspergillus or similar fungi (for example, Fusarium, Mucor, or Rhizopus).
  • Your current standard antifungal treatments are not working well, cannot be used safely, or are not expected to work for you — for example, because the fungus is resistant to them, you cannot tolerate them, or guidelines suggest they are not suitable for your situation.
  • Your doctor believes the study drug (APX001) may offer an advantage over standard treatments for your specific situation — for example, because of concerns about safety, drug interactions, or because the infection is in a location that standard treatments cannot reach well (confirm with trial site).

Who may not be able to join:

  • You have a blood cancer that is not responding to treatment.
  • Your fungal infection is a long-term (chronic) form of aspergillosis, a fungal ball (aspergilloma), or an allergic lung condition caused by Aspergillus.
  • You have received certain antifungal medicines (taken by mouth, by injection, or inhaled) in the 120 hours (5 days) before starting the study drug — unless the fungus causing your infection is known to be resistant to those medicines (confirm with trial site).
  • You have significant liver problems or poor liver function.

Important: Always verify eligibility with the trial site directly before applying.

Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.

This is a simplified plain English summary of the eligibility criteria. Full criteria are set by the trial investigators and may include additional requirements not shown here. Never self-exclude from a trial based on this summary. Contact the trial site directly to confirm your eligibility.
Last synced 28 July 2026
Phase 2: approximately ~30% of drugs entering this phase reach regulatory approval, based on published industry-wide historical data. This is not specific to this trial.
Phase success rates shown are historical industry-wide averages based on published data. They are not a prediction for this specific trial or your individual situation.

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Trial details

Status
Terminated
Phase
Phase 2
Sponsor
Basilea Pharmaceutica
Registry
ClinicalTrials.gov
Start date
4 January 2020
Est. completion
29 March 2022

Where this trial is recruiting

🇧🇪 Belgium 🇩🇪 Germany 🇮🇱 Israel 🇺🇸 United States

Primary endpoints

Percentage of Participants Who Died After the First Dose of Study Drug Through Day 42

Can't join this trial?

Expanded access pathways

If this trial is not available to you, other access pathways may exist. In Australia, the TGA Special Access Scheme allows access to unapproved therapeutic goods for individual patients.

TGA Special Access Scheme information

Find other recruiting trials on ClinicalTrials.gov

Data last synced from ClinicalTrials.gov: 28 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.

Trial recruitment status can change without notice between our nightly data updates. Always contact the trial site directly to confirm current recruitment status before making any decisions or travel arrangements.

View original record on ClinicalTrials.gov