Clinical trial NCT04240886: Open-label Study of APX001 for Treatment of Patients With Invasive Mold Infections Caused by Aspergillus or Rare Molds
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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
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
Lead sponsor: Basilea Pharmaceutica
Sponsor class: INDUSTRY
First posted: 2020-01-27
Enrollment: 21
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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).


Eligibility criteria: 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.

Primary outcomes:
measure: Percentage of Participants Who Died After the First Dose of Study Drug Through Day 42
timeFrame: After first dose on Day 1 through Day 42
Secondary outcomes:
measure: Percentage of Participants With Global Response Based on Data Review Committee (DRC) Assessment at End of Study Drug Treatment (EOST)
timeFrame: Any day from Day 1 until end of study treatment (any day up to Day 42)
measure: 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)
timeFrame: Any day from Day 1 until end of study treatment (any day up to Day 42)
measure: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
timeFrame: 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)
measure: Number of Participants With Clinically Significant Abnormality in Vital Signs
timeFrame: 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)
measure: Number of Participants With Clinically Significant Abnormality in Laboratory Test Evaluations
timeFrame: 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)
measure: Number of Participants With Clinically Significant Abnormal Electrocardiogram (ECG) Findings
timeFrame: 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)
measure: Number of Participants With Clinically Significant Change From Baseline in Physical and Neurological Examinations
timeFrame: 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)
measure: Plasma Concentrations of Fosmanogepix
timeFrame: Days 1, 2, 3, 4, 7: Pre-dose and 3 hours post-dose; Days 6, 13, 14: 3 hours post-dose, Day 15: pre-dose
Other outcomes:
Locations:
city: Duarte
country: United States
facility: City of Hope (City of Hope National Medical Center, City of Hope Medical Center)
state: California
city: Duarte
country: United States
facility: IP Address : City of Hope Investigational Drug Services (IDS)
state: California
city: Durham
country: United States
facility: Duke Department of Pharmacy/Investigational Drug Service (IDS)
state: North Carolina
city: Durham
country: United States
facility: Duke Medicine Pavilion
state: North Carolina
city: Durham
country: United States
facility: Duke University Medical Center (Duke South Clinic)
state: North Carolina
city: Durham
country: United States
facility: Duke University Medical Center(Duke Hospital)
state: North Carolina
city: Brussels
country: Belgium
facility: Cliniques Universitaires de Bruxelles Hôpital Erasme - Department of Infectious Diseases
city: Leuven
country: Belgium
facility: UZ Leuven - Department of Haematology
city: Yvoir
country: Belgium
facility: CHU UCL Namur - Mont- Godinne University Hospital - Intensive Care Unit
city: Munchen
country: Germany
facility: Klinikum Neuperlach, Klinik fur Hamatologie und Onkologie
state: Bavaria
city: Mainz
country: Germany
facility: Universitätsmedizin der Johannes Gutenberg-Universität Mainz III
city: Haifa
country: Israel
facility: Pharmacy
city: Haifa
country: Israel
facility: Rambam Medical Center
city: Ramat-Gan
country: Israel
facility: Pharmacy
city: Ramat-Gan
country: Israel
facility: Sheba Medical Center, Tel Hashomer
Study officials:
affiliation: Basilea Pharmaceutica International Ltd, Allschwil
name: Marc Engelhardt
role: STUDY_DIRECTOR
References: