Refractory IPA
Conditions
Keywords
Refractory IPA, invasive pulmonary aspergillosis
Brief summary
To assess the safety and efficacy of nebulized PC945 in combination with systemic antifungal therapy for the treatment of refractory IPA
Interventions
DRUGPC945
Sterile aqueous liquid for Nebulization
DRUGPlacebo
Sterile aqueous liquid for Nebulization
Sponsors
Pulmocide Ltd
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Masking description
Double Blind
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Participant has proven or probable IPA according to the modified 2019 European Organization for Research and Treatment of Cancer/ Mycoses Study Group Education and Research Consortium (EORTC/MSGERC) consensus definitions or according to the 2010 International Society for Heart and Lung Transplantation (ISHLT) consensus statements for the definitions of infections in cardiothoracic transplant recipients. 2. Participant's IPA has failed to respond to adequate antifungal therapy.
Exclusion criteria
1. Participant with a known or suspected concomitant medical condition or post-surgery complication that, in the opinion of the Investigator, may jeopardize adherence to the protocol requirements or impede the accurate measurement of efficacy or may be an unacceptable additional risk to the Participant should he/she participate in the study. 2. Participant who has previously received PC945. 3. Participant with a known history of allergy, hypersensitivity, or any previous serious reaction to any component of the PC945 or placebo formulations. 4. Participant who has recently received, is receiving or due to receive at any time during the study, an investigational medicinal agent that does not have any regulatory approved indications. Subjects who are participating in any other trials e.g., Observational, diagnostic or using medications with an approved indication may be allowed to participate after consultation with the sponsor on an individual basis
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants Alive With Favorable Overall Response as Assessed by the Data Review Committee (DRC) | At Day 84 | Favorable overall response was defined as being alive and having a complete or partial overall response at Day 84 as determined by the DRC. Complete or partial response was defined by the Classification and Criteria for the Assessment of Overall Response, adapted from Segal, 2008. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Favorable Overall Response | up to 12 weeks (Day 84) | Favorable overall response was defined as having a complete or partial overall response at at any time during the 12-week treatment where the survival component was not included as a part of the response definition. Complete or partial response was defined by the Classification and Criteria for the Assessment of Overall Response, adapted from Segal, 2008. |
| Time to Favorable Overall Response | up to Day 42 or Day 84 | Time to favorable overall response was defined as the time from randomization to the first favorable overall response (complete or partial response). Complete or partial response was defined by the Classification and Criteria for the Assessment of Overall Response, adapted from Segal, 2008. |
| All-cause Mortality | From First Dose of Study Treatment up to Week 16 (Safety Follow-up) | All-cause mortality was defined as death from any cause. |
Countries
Argentina, Australia, Austria, Belgium, Brazil, Canada, Chile, Colombia, France, Germany, Greece, India, Israel, Italy, South Korea, Spain, Taiwan, Thailand, United Kingdom, United States
Baseline characteristics
| Characteristic | — |
|---|---|
| Age, Customized 18 to <64 years | 58 Participants |
| Age, Customized 65 to <84 years | 25 Participants |
| Age, Customized ≥85 years | 0 Participants |
| Ethnicity (NIH/OMB) Hispanic or Latino | 6 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 20 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 4 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 2 Participants |
| Race (NIH/OMB) Asian | 17 Participants |
| Race (NIH/OMB) Black or African American | 2 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 10 Participants |
| Race (NIH/OMB) White | 51 Participants |
| Sex: Female, Male Female | 22 Participants |
| Sex: Female, Male Male | 17 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 21 / 57 | 8 / 28 |
| other Total, other adverse events | 45 / 56 | 25 / 27 |
| serious Total, serious adverse events | 36 / 56 | 18 / 27 |
Outcome results
None listed