Open-label Study of APX001 for Treatment of Patients With Invasive Mold Infections Caused by Aspergillus or Rare Molds

NCT04240886 | Terminated Phase 2 Results DMC FDA Drug

• 3 other identifiers: APX001-202C47910102019-001386-33
• Study Type: interventional
• Target: 21
• Locations: 4 countries
• Sites: 15

Brief Summary

This is a Phase 2, multicenter study to evaluate APX001 for the treatment of invasive fungal infections caused by Aspergillus spp. or rare molds (eg, Scedosporium spp., Fusarium spp., and Mucorales fungi).

Conditions

Invasive Fungal Infections

Keywords

APX001; APX001A; fosmanogepix; manogepix; aspergillus; aspergillosis; covid; covid19; mold; mould; pulmonary aspergillosis; pulmonary aspergillus; galactomannan; Karius; SARS-CoV-2; fusarium; mucorales; mucor; rhizopus; scedosporium; fungal disease; invasive fungal disease; EORTC

Outcome Measures

Primary Outcomes (1)

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

  Percentage of participants who died after first dose in the study through Day 42 were reported in this outcome measure. This outcome measure included all deaths from Day 1 through Day 42.

  After first dose on Day 1 through Day 42

Secondary Outcomes (8)

• Percentage of Participants With Global Response Based on Data Review Committee (DRC) Assessment at End of Study Drug Treatment (EOST)

  Any day from Day 1 until end of study treatment (any day up to Day 42)

• Percentage of Participants With Treatment Success or Treatment Failure for Global Response Based on Data Review Committee (DRC) Assessment at End of Study Drug Treatment (EOST)

  Any day from Day 1 until end of study treatment (any day up to Day 42)

• Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

  Day 1 up to maximum of 31 days of follow up post last dose of study treatment, where maximum treatment duration was 42 days (maximum up to 73 days)

• Number of Participants With Clinically Significant Abnormality in Vital Signs

  Day 1 up to maximum of 31 days of follow up post last dose of study treatment, where maximum treatment duration was 42 days (maximum up to 73 days)

• Number of Participants With Clinically Significant Abnormality in Laboratory Test Evaluations

  Day 1 up to maximum of 31 days of follow up post last dose of study treatment, where maximum treatment duration was 42 days (maximum up to 73 days)

Study Arms (2)

Cohort A: fosmanogepix (APX001)

EXPERIMENTAL

Drug: fosmanogepix

Cohort B: fosmanogepix (APX001)

EXPERIMENTAL

Drug: fosmanogepix

Interventions

fosmanogepix DRUG

IV and oral fosmanogepix

Also known as: APX001, E210

Cohort A: fosmanogepix (APX001); Cohort B: fosmanogepix (APX001)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Males or females, 18 years or older.
• Patients with proven or probable IMI caused by Aspergillus spp. Patients who present with IMI due to other filamentous fungi (eg, Scedosporium spp., Fusarium spp., and Mucorales fungi such as Mucor spp. or Rhizopus spp.) may also be enrolled.
• Have limited or no treatment options due to documented or anticipated resistance, contraindication, intolerance, or lack of clinical response to SOC antifungal therapy, as advocated by the relevant regional/country treatment guidelines.
• Patients where the Investigator considers that there is a potential advantage of using APX001 over current SOC (eg, broad spectrum of activity, emergence of IMI during antifungal prophylaxis, activity against resistant mold pathogens, IV and PO formulations, favorable DDI profile, favorable hepatic and renal safety profile, wide tissue distribution including brain), and/or where the SOC antifungal therapy carries significant risk of toxicity or treatment failure (eg, DDI risk, safety/toxicity risk, site of infection not accessible by SOC).

You may not qualify if:

• Refractory hematologic malignancy.
• Chronic aspergillosis, aspergilloma, or allergic bronchopulmonary aspergillosis.
• Treatment with systemic (PO, IV, or inhaled) mold active antifungal therapy for 120 hours immediately before initial dosing. Note: patients with invasive fungal infection caused by a mold with documented resistance to or lack of coverage by the prior SOC in question, may have received \>120 hours prior treatment and remain eligible for the study.
• Evidence of significant hepatic dysfunction.

Sponsors & Collaborators

• Basilea Pharmaceutica: lead

Study Sites (15)

City of Hope (City of Hope National Medical Center, City of Hope Medical Center)

Duarte, California, 91010, United States

Location

IP Address : City of Hope Investigational Drug Services (IDS)

Duarte, California, 91010, United States

Location

Duke Department of Pharmacy/Investigational Drug Service (IDS)

Durham, North Carolina, 27710, United States

Location

Duke Medicine Pavilion

Durham, North Carolina, 27710, United States

Location

Duke University Medical Center (Duke South Clinic)

Durham, North Carolina, 27710, United States

Location

Duke University Medical Center(Duke Hospital)

Durham, North Carolina, 27710, United States

Location

Cliniques Universitaires de Bruxelles Hôpital Erasme - Department of Infectious Diseases

Brussels, 1070, Belgium

Location

UZ Leuven - Department of Haematology

Leuven, 3000, Belgium

Location

CHU UCL Namur - Mont- Godinne University Hospital - Intensive Care Unit

Yvoir, 5530, Belgium

Location

Klinikum Neuperlach, Klinik fur Hamatologie und Onkologie

München, Bavaria, 81737, Germany

Location

Universitätsmedizin der Johannes Gutenberg-Universität Mainz III

Mainz, 55131, Germany

Location

Pharmacy

Haifa, 3109601, Israel

Location

Rambam Medical Center

Haifa, 3109601, Israel

Location

Pharmacy

Ramat Gan, 5262160, Israel

Location

Sheba Medical Center, Tel Hashomer

Ramat Gan, 5262160, Israel

Location

Related Publications (1)

• Hodges MR, Tawadrous M, Cornely OA, Thompson GR, Slavin MA, Maertens JA, Dadwal SS, Rahav G, Hazel S, Almas M, Jakate A, Pypstra R. Fosmanogepix for the Treatment of Invasive Mold Diseases Caused by Aspergillus Species and Rare Molds: A Phase 2, Open-Label Study (AEGIS). Clin Infect Dis. 2025 Dec 24;81(5):e302-e309. doi: 10.1093/cid/ciaf185.

  PMID: 40203286 RESULT

MeSH Terms

Conditions

Invasive Fungal Infections; Aspergillosis; COVID-19; Pulmonary Aspergillosis; Mycoses

Condition Hierarchy (Ancestors)

Bacterial Infections and Mycoses; Infections; Pneumonia, Viral; Pneumonia; Respiratory Tract Infections; Virus Diseases; Coronavirus Infections; Coronaviridae Infections; Nidovirales Infections; RNA Virus Infections; Lung Diseases; Respiratory Tract Diseases; Lung Diseases, Fungal

Results Point of Contact

• Title: Study Director
• Organization: Basilea Pharmaceutica International Ltd, Allschwil

marc.engelhardt@basilea.com

+41 79 701 0551

Study Officials

• Marc Engelhardt

  Basilea Pharmaceutica International Ltd, Allschwil

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 22, 2020
• First Posted: January 27, 2020
• Study Start: January 4, 2020
• Primary Completion: March 29, 2022
• Study Completion: May 9, 2022
• Last Updated: September 16, 2025
• Results First Posted: May 19, 2023

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share

Locations

BE (3); IL (4); DE (2); US (6)