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

Sponsor

Pfizer (Industry)

Overall Status

Completed

CT.gov ID

NCT04240886

Collaborator

(none)

Enrollment

Locations

Arms

28.1

Actual Duration (Months)

1.5

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

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).

Condition or Disease	Intervention/Treatment	Phase
Invasive Fungal Infections	Drug: fosmanogepix	Phase 2

Study Design

Study Type:

Interventional

Actual Enrollment :

21 participants

Allocation:

Non-Randomized

Intervention Model:

Parallel Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

A Phase 2, Open-Label Study to Evaluate the Safety and Efficacy of APX001 in the Treatment of Patients With Invasive Mold Infections Caused by Aspergillus Species or Rare Molds

Actual Study Start Date :

Jan 4, 2020

Actual Primary Completion Date :

Mar 29, 2022

Actual Study Completion Date :

May 9, 2022

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Cohort A: fosmanogepix (APX001)	Drug: fosmanogepix IV and oral fosmanogepix Other Names: APX001 E210
Experimental: Cohort B: fosmanogepix (APX001)	Drug: fosmanogepix IV and oral fosmanogepix Other Names: APX001 E210

Arm

Intervention/Treatment

Experimental: Cohort A: fosmanogepix (APX001)

Drug: fosmanogepix

IV and oral fosmanogepix

Other Names:

APX001

E210

Experimental: Cohort B: fosmanogepix (APX001)

Drug: fosmanogepix

IV and oral fosmanogepix

Other Names:

APX001

E210

Outcome Measures

Primary Outcome Measures

All-Cause Mortality [Day 42]

Secondary Outcome Measures

Global Response [End of Study Treatment or Day 42]

Other Outcome Measures

All-Cause Mortality [Day 84]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Clinical Trial Site	Duarte	California	United States	91010
2	Clinical Trial Site	Sacramento	California	United States	95616
3	Clinical Trial Site	San Diego	California	United States	92093
4	Clinical Trial Site	Boston	Massachusetts	United States	02115
5	Clinical Trial Site	Durham	North Carolina	United States	27710
6	Clinical Trial Site	Brussels		Belgium	1000
7	Clinical Trial Site	Brussels		Belgium	1070
8	Clinical Trial Site	Leuven		Belgium	3000
9	Clinical Trial Site	Yvoir		Belgium	5530
10	Clinical Trial Site	Koeln		Germany
11	Clinical Trial Site	Mainz		Germany
12	Clinical Trial Site	Haifa		Israel
13	Clinical Trial Site	Tel Aviv		Israel
14	Clinical Trial Site	Tel HaShomer		Israel