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

NCT ID: NCT04240886

Last Updated: 2025-09-16

Study Results

Results available

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE2

Total Enrollment

21 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-01-04

Study Completion Date

2022-05-09

Brief Summary

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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).

Detailed Description

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Conditions

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Invasive Fungal Infections

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Cohort A: fosmanogepix (APX001)

Group Type EXPERIMENTAL

fosmanogepix

Intervention Type DRUG

IV and oral fosmanogepix

Cohort B: fosmanogepix (APX001)

Group Type EXPERIMENTAL

fosmanogepix

Intervention Type DRUG

IV and oral fosmanogepix

Interventions

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fosmanogepix

IV and oral fosmanogepix

Intervention Type DRUG

Other Intervention Names

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APX001 E210

Eligibility Criteria

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Inclusion Criteria

* 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).

Exclusion Criteria

* 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.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Basilea Pharmaceutica

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Marc Engelhardt

Role: STUDY_DIRECTOR

Basilea Pharmaceutica International Ltd, Allschwil

Locations

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

Duarte, California, United States

Site Status

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

Duarte, California, United States

Site Status

Duke Department of Pharmacy/Investigational Drug Service (IDS)

Durham, North Carolina, United States

Site Status

Duke Medicine Pavilion

Durham, North Carolina, United States

Site Status

Duke University Medical Center (Duke South Clinic)

Durham, North Carolina, United States

Site Status

Duke University Medical Center(Duke Hospital)

Durham, North Carolina, United States

Site Status

Cliniques Universitaires de Bruxelles Hôpital Erasme - Department of Infectious Diseases

Brussels, , Belgium

Site Status

UZ Leuven - Department of Haematology

Leuven, , Belgium

Site Status

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

Yvoir, , Belgium

Site Status

Klinikum Neuperlach, Klinik fur Hamatologie und Onkologie

München, Bavaria, Germany

Site Status

Universitätsmedizin der Johannes Gutenberg-Universität Mainz III

Mainz, , Germany

Site Status

Pharmacy

Haifa, , Israel

Site Status

Rambam Medical Center

Haifa, , Israel

Site Status

Pharmacy

Ramat Gan, , Israel

Site Status

Sheba Medical Center, Tel Hashomer

Ramat Gan, , Israel

Site Status

Countries

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United States Belgium Germany Israel

References

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Hodges MR, Tawadrous M, Cornely OA, Thompson GR 3rd, 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 Apr 9:ciaf185. doi: 10.1093/cid/ciaf185. Online ahead of print.

Reference Type RESULT
PMID: 40203286 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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C4791010

Identifier Type: OTHER

Identifier Source: secondary_id

2019-001386-33

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

APX001-202

Identifier Type: -

Identifier Source: org_study_id

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