A Phase 3 Efficacy and Safety Study of Fosmanogepix for the Treatment of Adult Patients With Invasive Mold Infections.

Phase 3

Not yet recruiting

• Conditions: Invasive Mold Infections
• Interventions: Drug: Fosmanogepix IV infusion; Drug: Standard of care antifungal therapy; Drug: Fosmanogepix oral tablet

• Registration Number: NCT06925321
• Lead Sponsor: Basilea Pharmaceutica

Brief Summary

The purpose of this study is to evaluate the efficacy and safety of fosmanogepix (administered IV or oral) for the treatment of adult patients with invasive mold infections. The study is looking for patients who have been diagnosed with invasive mold infections. The maximum study duration will be approximately 8 months, including a target study treatment duration of 84 days which can be extended up to 180 days and follow-up period.

The patient will be assigned to one of two treatment cohorts:

Cohort A (primary therapy): Patients will receive either the study drug or institutional standard of care antifungal treatment.

Cohort B (salvage treatment; i.e. treatment given after patients did not respond to previous treatments or did not tolerate them): Patients will receive the study drug

The primary aim is to compare the all cause mortality with a fixed threshold at Day 42.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2025-03-27
• Study First Submitted QC: 2025-04-11
• Study First Posted: 2025-04-13
• Status Verified: 2025-07
• Study Start: 2025-07-01
• Last Update Submitted: 2025-07-03
• Last Update Posted: 2025-07-04
• Primary Completion: 2028-02
• Study Completion: 2028-02-01

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 219

Inclusion Criteria

1. Diagnosis of proven or probable Invasive mold infection (IMI) defined in accordance with the Revision and Update of the Consensus Definitions of Invasive Fungal Disease from the EORTC/MSGERC as adapted for this study and caused by Aspergillus spp. (in patients with limited treatment options), Fusarium spp., Lomentospora prolificans, Mucorales fungi, or other multi-drug resistant molds.
2. Patient's condition allows for appropriate infection source control measures.

Main Exclusion Critera:

1. Refractory hematologic malignancy.
2. Chronic aspergillosis, aspergilloma, or allergic bronchopulmonary aspergillosis.
3. COVID-19 associated mucormycosis.
4. Invasive fungal disease caused by more than one fungal pathogen is not permitted in Cohort A but is permitted in Cohort B.
5. Patients with a Karnofsky Performance Status < 20 at Screening.
6. Requirement, or anticipated requirement, for hemodialysis, peritoneal dialysis, or hemofiltration.
7. Patients with known human immunodeficiency virus infection.
8. Ongoing neurological disorders.
9. Patients receiving hospice/comfort care only.
10. Other medical or psychiatric condition.
11. Current use of any prohibited concomitant medication(s).
12. Current/ previous administration of an investigational drug within 30 days.
13. Prior enrollment in this or any previous study of fosmanogepix.
14. Moderate or severe hepatic impairment.
15. Patient who is pregnant or lactating.
16. Known hypersensitivity to fosmanogepix, manogepix, or any of their excipients.

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Cohort A: Experimental Treatment	Fosmanogepix IV infusion	Patients will receive the study drug. Fosmanogepix will be administered as an Intravenous (IV) infusion or in oral form.
Cohort A: Experimental Treatment	Fosmanogepix oral tablet	Patients will receive the study drug. Fosmanogepix will be administered as an Intravenous (IV) infusion or in oral form.
Cohort A: Comparator Antifungal Treatment	Standard of care antifungal therapy	Best available therapy (BAT) administered as IV or orally per standard guidelines.
Cohort B	Fosmanogepix IV infusion	Patients will receive the study drug. Fosmanogepix will be administered as an Intravenous (IV) infusion or in oral form.
Cohort B	Fosmanogepix oral tablet	Patients will receive the study drug. Fosmanogepix will be administered as an Intravenous (IV) infusion or in oral form.

Primary Outcome Measures

Name	Time	Method
Day 42 all-cause mortality rate	Day 42

Secondary Outcome Measures

Name	Time	Method
All-cause mortality rate at Day 84	Day 84
Number of patients with abnormal neurological examination findings	Up to follow-up 6 weeks after EOST (target duration approximately up to 8 months)
Assessment of 12-lead electrocardiogram corrected QT (Fridericia method) Interval (ECG QTcF Interval)	Up to follow-up 6 weeks after EOST (target duration approximately up to 8 months)
Plasma concentrations versus time of fosmanogepix (prodrug) and manogepix (active moiety) following IV administration	Pre-dose, 3,6, and 9 hours post-start of the 3-hour IV infusion on Day 3, and at 24 hours (prior to Day 4 dosing)
Plasma concentrations versus time of fosmanogepix (prodrug) and manogepix (active moiety) following oral administration	On days 7, 14, 28, and 42. Post-dose plasma samples will also be collected: 72 hrs and 192 hrs after last dose.
Proportion of patients with overall response of treatment success	Day 42, Day 84 and up to 180 days
Proportion of patients with clinical response of treatment success	Day 42, Day 84 and up to 180 days
Proportion of patients with mycological response of eradication or presumed eradication	Day 42, Day 84 and up to 180 days
Proportion of patients with radiological response of complete response or partial response	Day 42, Day 84 and up to 180 days
Incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), treatment-related AEs, adverse events of special interest (AESI), and AEs leading to discontinuation	Screening up to follow-up 6 weeks after EOST (target duration approximately up to 8 months)
Number of patients with clinically significant laboratory abnormalities	Up to follow-up 6 weeks after EOST (target duration approximately up to 8 months)