Study to evaluate the safety and efficacy of APX001 in the treatment of patients with invasive mold infections
- Conditions
- Treatment of patients with invasive mold infections (IMIs) caused by Aspergillus species (spp.) or rare moldsMedDRA version: 20.1Level: LLTClassification code 10062642Term: Invasive mycosisSystem Organ Class: 100000004862Therapeutic area: Diseases [C] - Bacterial Infections and Mycoses [C01]
- Registration Number
- EUCTR2019-001386-33-BE
- Lead Sponsor
- Amplyx Pharmaceuticals, Inc., a wholly owned subsidiary of Pfizer, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 50
1.Males or females, 18 years or older.
2.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.
Cohort A: Diagnosis of a proven or probable IMI will be defined in accordance with the Revision and Update of the Consensus Definitions of Invasive Fungal Disease from the EORTC/MSGERC a modified version of the 2008 Revised Definitions of Invasive Fungal Disease from the European Organization for Research and Treatment of Cancer/Mycosis Study Group (EORTC/MSG) Consensus Group criteria.
Cohort B: Diagnosis of proven or putative aspergillosis will be defined in accordance the diagnostic criteria specified in Appendix F of the protocol.
3.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.
4.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).
9.Cohort B only: Patients must have a lower respiratory tract infection due to SARS-CoV-2 or influenza A/B.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 40
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 10
1.Refractory hematologic malignancy.
2.Chronic aspergillosis, aspergilloma, or allergic bronchopulmonary aspergillosis.
3.Treatment with systemic (PO, IV, or inhaled) antifungal therapy with a mold-active azole or polyene 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, as well as patients with polyene-induced renal toxicity or those who have received empirical treatment with echinocandins, may have received >120 hours prior treatment and remain eligible for the study.
4.Evidence of significant hepatic dysfunction
13.Patients on mechanical ventilation
14.Cohort A only: Suspected or confirmed COVID-19 or Influenza A/B infection.
15. Patients with severe renal impairment as determined by estimated glomerular filtration rate (eGFR) < 30 mL/min/1.173 m2 calculated by CKD-EPI, including patients on dialysis.
16. Patients with a Karnofsky Performance Status = 30 at screening
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: The primary objective of this proof of concept study is to evaluate the safety and efficacy of APX001 for the treatment of adult patients aged 18 years and above with IMIs caused by Aspergillus spp. or rare molds (eg, Scedosporium spp., Fusarium spp., and Mucorales fungi), who have limited antifungal treatment options. ;Secondary Objective: •Evaluate global response at End of Study Treatment (EOST) <br>•Evaluate safety parameters of APX001<br>•Evaluate pharmacokinetic (PK) parameters of APX001<br>;Primary end point(s): The primary efficacy endpoint is all-cause mortality through Day 42.;Timepoint(s) of evaluation of this end point: At Day 42
- Secondary Outcome Measures
Name Time Method Secondary end point(s): •Global response at EOST.<br>•All-cause mortality through Day 84.<br>•Change from Baseline in galactomannan.<br>•Change from Baseline in beta-d-glucan;Timepoint(s) of evaluation of this end point: •At EOST<br>•At Day 84<br>•Change from Baseline