Trial Outcomes & Findings for An Open-label Study of APX001 for Treatment of Patients With Candidemia/Invasive Candidiasis Caused by Candida Auris (NCT NCT04148287)

Last Updated: 2025-09-16

Results Overview

Treatment success was defined as meeting all of the following criteria: 1\) Two consecutive blood cultures negative for Candida species, and/or for participants with a deep-seated site of infection, at least 1 negative tissue culture or aspirate/fluid culture. For participants with a deep-seated site of infection involving visceral organs from which a tissue culture was not obtainable, resolution of the attributable clinical signs of infection recorded at Baseline, and as applicable, radiological improvement associated with the site of infection. 2) Alive at EOST and 3) No concomitant use of any other systemic antifungal therapies through EOST.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

9 participants

Primary outcome timeframe

EOST: any day from Day 1 up to maximum of Day 42

Results posted on

2025-09-16

Participant Flow

Participant milestones

Participant milestones
Measure	APX001 (Fosmanogepix) Participants with candidemia and/or invasive candidiasis caused by Candida auris and with limited antifungal treatment options were administered APX001. On Day 1, participants received APX001 1000 milligrams (mg) as a loading dose over 3 hours by intravenous (IV) infusion twice daily (BID). On Days 2 and 3, participants received APX001 600 mg as maintenance dose over 3 hours by IV infusion once daily (QD). On Day 4 and onwards, maintenance dose was administered as either APX001 600 mg IV infusion QD over 3 hours or APX001 800 mg orally QD. Participants who were clinically stable after 3 days of IV administration and could swallow tablets, could switch from IV to oral dosing from Day 4 and onwards. Maximum treatment duration was of 42 days. Participants were followed up for 4 weeks (+ 4 days) post last dose of study drug.
Overall Study STARTED	9
Overall Study COMPLETED	7
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Reasons for withdrawal
Measure	APX001 (Fosmanogepix) Participants with candidemia and/or invasive candidiasis caused by Candida auris and with limited antifungal treatment options were administered APX001. On Day 1, participants received APX001 1000 milligrams (mg) as a loading dose over 3 hours by intravenous (IV) infusion twice daily (BID). On Days 2 and 3, participants received APX001 600 mg as maintenance dose over 3 hours by IV infusion once daily (QD). On Day 4 and onwards, maintenance dose was administered as either APX001 600 mg IV infusion QD over 3 hours or APX001 800 mg orally QD. Participants who were clinically stable after 3 days of IV administration and could swallow tablets, could switch from IV to oral dosing from Day 4 and onwards. Maximum treatment duration was of 42 days. Participants were followed up for 4 weeks (+ 4 days) post last dose of study drug.
Overall Study Death	2

Baseline Characteristics

An Open-label Study of APX001 for Treatment of Patients With Candidemia/Invasive Candidiasis Caused by Candida Auris

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	APX001 (Fosmanogepix) n=9 Participants Participants with candidemia and/or invasive candidiasis caused by Candida auris and with limited antifungal treatment options were administered APX001. On Day 1, participants received APX001 1000 mg as a loading dose over 3 hours by IV infusion BID. On Days 2 and 3, participants received APX001 600 mg as maintenance dose over 3 hours by IV infusion QD. On Day 4 and onwards, maintenance dose was administered as either APX001 600 mg IV infusion QD over 3 hours or APX001 800 mg orally QD. Participants who were clinically stable after 3 days of IV administration and could swallow tablets, could switch from IV to oral dosing from Day 4 and onwards. Maximum treatment duration was of 42 days. Participants were followed up for 4 weeks (+ 4 days) post last dose of study drug.
Age, Continuous	49.8 Years STANDARD_DEVIATION 17.70 • n=5 Participants
Sex: Female, Male Female	3 Participants n=5 Participants
Sex: Female, Male Male	6 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	9 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	5 Participants n=5 Participants
Race (NIH/OMB) White	3 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: EOST: any day from Day 1 up to maximum of Day 42

Population: The modified intent-to-treat (mITT) population included all participants who received at least 1 dose of study drug and had a confirmed diagnosis of candidemia and/or invasive candidiasis caused by Candida auris within 120 hours (for candidemia) or 168 hours (for invasive candidiasis without candidemia) of study qualification (as determined by the DRC).

Outcome measures

Outcome measures
Measure	APX001 (Fosmanogepix) n=9 Participants Participants with candidemia and/or invasive candidiasis caused by Candida auris and with limited antifungal treatment options were administered APX001. On Day 1, participants received APX001 1000 mg as a loading dose over 3 hours by IV infusion BID. On Days 2 and 3, participants received APX001 600 mg as maintenance dose over 3 hours by IV infusion QD. On Day 4 and onwards, maintenance dose was administered as either APX001 600 mg IV infusion QD over 3 hours or APX001 800 mg orally QD. Participants who were clinically stable after 3 days of IV administration and could swallow tablets, could switch from IV to oral dosing from Day 4 and onwards. Maximum treatment duration was of 42 days. Participants were followed up for 4 weeks (+ 4 days) post last dose of study drug.
Percentage of Participants With Treatment Success at End of Study Treatment (EOST) as Determined by Data Review Committee (DRC)	88.9 Percentage of participants Interval 51.8 to 99.7

SECONDARY outcome

Timeframe: Day 1 up to maximum of Day 42

Population: The mITT population included all participants who received at least 1 dose of study drug and had a confirmed diagnosis of candidemia and/or invasive candidiasis caused by Candida auris within 120 hours (for candidemia) or 168 hours (for invasive candidiasis without candidemia) of study qualification (as determined by the DRC). Here, "Overall Number of Participants Analyzed" signifies participants evaluable for this outcome measure.

Time to first negative blood culture was defined as the number of days from date of first dose of study drug to the date of first negative blood culture plus 1. Participants without a negative blood culture at post-baseline visits were censored at the last assessment date. Kaplan-Meier method was used for analysis.

Outcome measures

Outcome measures
Measure	APX001 (Fosmanogepix) n=3 Participants Participants with candidemia and/or invasive candidiasis caused by Candida auris and with limited antifungal treatment options were administered APX001. On Day 1, participants received APX001 1000 mg as a loading dose over 3 hours by IV infusion BID. On Days 2 and 3, participants received APX001 600 mg as maintenance dose over 3 hours by IV infusion QD. On Day 4 and onwards, maintenance dose was administered as either APX001 600 mg IV infusion QD over 3 hours or APX001 800 mg orally QD. Participants who were clinically stable after 3 days of IV administration and could swallow tablets, could switch from IV to oral dosing from Day 4 and onwards. Maximum treatment duration was of 42 days. Participants were followed up for 4 weeks (+ 4 days) post last dose of study drug.
Time to First Negative Blood Culture	6 Days Interval 5.0 to 15.0

SECONDARY outcome

Timeframe: EOST: any day from Day 1 up to maximum of Day 42, 2 and 4 weeks after EOST

Mycological outcomes were determined based on eradication and presumed eradication. Eradication was defined as a negative blood (and/or other infection site) culture(s) for Candida species. Presumed eradication (applicable to invasive candidiasis) was defined as clinical resolution of invasive Candida species infection where tissue samples were unavailable. These would be applicable only if there were no concomitant or additional systemic antifungal usage.

Outcome measures

Outcome measures
Measure	APX001 (Fosmanogepix) n=9 Participants Participants with candidemia and/or invasive candidiasis caused by Candida auris and with limited antifungal treatment options were administered APX001. On Day 1, participants received APX001 1000 mg as a loading dose over 3 hours by IV infusion BID. On Days 2 and 3, participants received APX001 600 mg as maintenance dose over 3 hours by IV infusion QD. On Day 4 and onwards, maintenance dose was administered as either APX001 600 mg IV infusion QD over 3 hours or APX001 800 mg orally QD. Participants who were clinically stable after 3 days of IV administration and could swallow tablets, could switch from IV to oral dosing from Day 4 and onwards. Maximum treatment duration was of 42 days. Participants were followed up for 4 weeks (+ 4 days) post last dose of study drug.
Percentage of Participants With Mycological Outcomes at EOST and 2 and 4 Weeks After EOST EOST	66.7 Percentage of participants Interval 29.9 to 92.5
Percentage of Participants With Mycological Outcomes at EOST and 2 and 4 Weeks After EOST 2 Weeks after EOST	66.7 Percentage of participants Interval 29.9 to 92.5
Percentage of Participants With Mycological Outcomes at EOST and 2 and 4 Weeks After EOST 4 Weeks after EOST	66.7 Percentage of participants Interval 29.9 to 92.5

SECONDARY outcome

Timeframe: EOST: any day from Day 1 up to maximum of Day 42

Outcome measures

Outcome measures
Measure	APX001 (Fosmanogepix) n=9 Participants Participants with candidemia and/or invasive candidiasis caused by Candida auris and with limited antifungal treatment options were administered APX001. On Day 1, participants received APX001 1000 mg as a loading dose over 3 hours by IV infusion BID. On Days 2 and 3, participants received APX001 600 mg as maintenance dose over 3 hours by IV infusion QD. On Day 4 and onwards, maintenance dose was administered as either APX001 600 mg IV infusion QD over 3 hours or APX001 800 mg orally QD. Participants who were clinically stable after 3 days of IV administration and could swallow tablets, could switch from IV to oral dosing from Day 4 and onwards. Maximum treatment duration was of 42 days. Participants were followed up for 4 weeks (+ 4 days) post last dose of study drug.
Percentage of Participants With Treatment Success at EOST Determined by Investigator	88.9 Percentage of participants Interval 51.8 to 99.7

SECONDARY outcome

Timeframe: 2 and 4 weeks after EOST (where EOST is any day from Day 1 up to maximum of Day 42)

Population: The mITT population included all participants who received at least 1 dose of study drug and had a confirmed diagnosis of candidemia and/or invasive candidiasis caused by Candida auris within 120 hours (for candidemia) or 168 hours (for invasive candidiasis without candidemia) of study qualification (as determined by the DRC). Here, "Overall Number of Participants Analyzed"=participants evaluable for this outcome measure and "number analyzed"= number of participants evaluable for specified rows.

Outcome measures

Outcome measures
Measure	APX001 (Fosmanogepix) n=7 Participants Participants with candidemia and/or invasive candidiasis caused by Candida auris and with limited antifungal treatment options were administered APX001. On Day 1, participants received APX001 1000 mg as a loading dose over 3 hours by IV infusion BID. On Days 2 and 3, participants received APX001 600 mg as maintenance dose over 3 hours by IV infusion QD. On Day 4 and onwards, maintenance dose was administered as either APX001 600 mg IV infusion QD over 3 hours or APX001 800 mg orally QD. Participants who were clinically stable after 3 days of IV administration and could swallow tablets, could switch from IV to oral dosing from Day 4 and onwards. Maximum treatment duration was of 42 days. Participants were followed up for 4 weeks (+ 4 days) post last dose of study drug.
Percentage of Participants With Treatment Success at 2 and 4 Weeks After EOST Determined by Investigator and by the DRC 2 Weeks after EOST: DRC	66.7 Percentage of participants Interval 29.9 to 92.5
Percentage of Participants With Treatment Success at 2 and 4 Weeks After EOST Determined by Investigator and by the DRC 4 Weeks after EOST: DRC	66.7 Percentage of participants Interval 29.9 to 92.5
Percentage of Participants With Treatment Success at 2 and 4 Weeks After EOST Determined by Investigator and by the DRC 2 Weeks after EOST: Investigator	77.8 Percentage of participants Interval 40.0 to 97.2
Percentage of Participants With Treatment Success at 2 and 4 Weeks After EOST Determined by Investigator and by the DRC 4 Weeks after EOST: Investigator	77.8 Percentage of participants Interval 40.0 to 97.2

SECONDARY outcome

Timeframe: Day 1 through Day 30

Percentage of participants who died through study Day 30 is reported in this outcome measure.

Outcome measures

Outcome measures
Measure	APX001 (Fosmanogepix) n=9 Participants Participants with candidemia and/or invasive candidiasis caused by Candida auris and with limited antifungal treatment options were administered APX001. On Day 1, participants received APX001 1000 mg as a loading dose over 3 hours by IV infusion BID. On Days 2 and 3, participants received APX001 600 mg as maintenance dose over 3 hours by IV infusion QD. On Day 4 and onwards, maintenance dose was administered as either APX001 600 mg IV infusion QD over 3 hours or APX001 800 mg orally QD. Participants who were clinically stable after 3 days of IV administration and could swallow tablets, could switch from IV to oral dosing from Day 4 and onwards. Maximum treatment duration was of 42 days. Participants were followed up for 4 weeks (+ 4 days) post last dose of study drug.
All-Cause Mortality Through Study Day 30	11.1 Percentage of participants

SECONDARY outcome

Timeframe: Day 1 up to maximum of 32 days of follow up post last dose of study drug, where maximum treatment duration was 42 days (maximum up to 74 days)

Population: Safety population included all participants who received at least 1 dose of study drug.

An adverse event was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. TEAEs were events between first dose of study drug and up to Week 4 (+4 days) post last dose of study drug that were absent before treatment or that worsened relative to pretreatment state.

Outcome measures

Outcome measures
Measure	APX001 (Fosmanogepix) n=9 Participants Participants with candidemia and/or invasive candidiasis caused by Candida auris and with limited antifungal treatment options were administered APX001. On Day 1, participants received APX001 1000 mg as a loading dose over 3 hours by IV infusion BID. On Days 2 and 3, participants received APX001 600 mg as maintenance dose over 3 hours by IV infusion QD. On Day 4 and onwards, maintenance dose was administered as either APX001 600 mg IV infusion QD over 3 hours or APX001 800 mg orally QD. Participants who were clinically stable after 3 days of IV administration and could swallow tablets, could switch from IV to oral dosing from Day 4 and onwards. Maximum treatment duration was of 42 days. Participants were followed up for 4 weeks (+ 4 days) post last dose of study drug.
Number of Participants With Treatment Emergent Adverse Events (TEAEs)	9 Participants

Adverse Events

APX001 (Fosmanogepix)

Serious events: 2 serious events

Other events: 9 other events

Deaths: 2 deaths

Serious adverse events

Serious adverse events
Measure	APX001 (Fosmanogepix) n=9 participants at risk Participants with candidemia and/or invasive candidiasis caused by Candida auris and with limited antifungal treatment options were administered APX001. On Day 1, participants received APX001 1000 mg as a loading dose over 3 hours by IV infusion BID. On Days 2 and 3, participants received APX001 600 mg as maintenance dose over 3 hours by IV infusion QD. On Day 4 and onwards, maintenance dose was administered as either APX001 600 mg IV infusion QD over 3 hours or APX001 800 mg orally QD. Participants who were clinically stable after 3 days of IV administration and could swallow tablets, could switch from IV to oral dosing from Day 4 and onwards. Maximum treatment duration was of 42 days. Participants were followed up for 4 weeks (+ 4 days) post last dose of study drug.
Cardiac disorders Cardiac arrest	11.1% 1/9 • Day 1 up to maximum of 32 days of follow up post last dose of study drug, where maximum treatment duration was 42 days (maximum up to 74 days) Same event may appear as AE and SAE, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study.
General disorders Multiple organ dysfunction syndrome	22.2% 2/9 • Day 1 up to maximum of 32 days of follow up post last dose of study drug, where maximum treatment duration was 42 days (maximum up to 74 days) Same event may appear as AE and SAE, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study.
Infections and infestations Pneumonia	11.1% 1/9 • Day 1 up to maximum of 32 days of follow up post last dose of study drug, where maximum treatment duration was 42 days (maximum up to 74 days) Same event may appear as AE and SAE, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study.
Vascular disorders Hypotension	11.1% 1/9 • Day 1 up to maximum of 32 days of follow up post last dose of study drug, where maximum treatment duration was 42 days (maximum up to 74 days) Same event may appear as AE and SAE, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study.

Other adverse events

Other adverse events
Measure	APX001 (Fosmanogepix) n=9 participants at risk Participants with candidemia and/or invasive candidiasis caused by Candida auris and with limited antifungal treatment options were administered APX001. On Day 1, participants received APX001 1000 mg as a loading dose over 3 hours by IV infusion BID. On Days 2 and 3, participants received APX001 600 mg as maintenance dose over 3 hours by IV infusion QD. On Day 4 and onwards, maintenance dose was administered as either APX001 600 mg IV infusion QD over 3 hours or APX001 800 mg orally QD. Participants who were clinically stable after 3 days of IV administration and could swallow tablets, could switch from IV to oral dosing from Day 4 and onwards. Maximum treatment duration was of 42 days. Participants were followed up for 4 weeks (+ 4 days) post last dose of study drug.
Cardiac disorders Angina pectoris	11.1% 1/9 • Day 1 up to maximum of 32 days of follow up post last dose of study drug, where maximum treatment duration was 42 days (maximum up to 74 days) Same event may appear as AE and SAE, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study.
Cardiac disorders Atrial fibrillation	11.1% 1/9 • Day 1 up to maximum of 32 days of follow up post last dose of study drug, where maximum treatment duration was 42 days (maximum up to 74 days) Same event may appear as AE and SAE, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study.
Gastrointestinal disorders Constipation	22.2% 2/9 • Day 1 up to maximum of 32 days of follow up post last dose of study drug, where maximum treatment duration was 42 days (maximum up to 74 days) Same event may appear as AE and SAE, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study.
Gastrointestinal disorders Diarrhoea	11.1% 1/9 • Day 1 up to maximum of 32 days of follow up post last dose of study drug, where maximum treatment duration was 42 days (maximum up to 74 days) Same event may appear as AE and SAE, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study.
Gastrointestinal disorders Nausea	11.1% 1/9 • Day 1 up to maximum of 32 days of follow up post last dose of study drug, where maximum treatment duration was 42 days (maximum up to 74 days) Same event may appear as AE and SAE, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study.
General disorders Pyrexia	33.3% 3/9 • Day 1 up to maximum of 32 days of follow up post last dose of study drug, where maximum treatment duration was 42 days (maximum up to 74 days) Same event may appear as AE and SAE, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study.
General disorders Generalised oedema	11.1% 1/9 • Day 1 up to maximum of 32 days of follow up post last dose of study drug, where maximum treatment duration was 42 days (maximum up to 74 days) Same event may appear as AE and SAE, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study.
General disorders Hypothermia	11.1% 1/9 • Day 1 up to maximum of 32 days of follow up post last dose of study drug, where maximum treatment duration was 42 days (maximum up to 74 days) Same event may appear as AE and SAE, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study.
Infections and infestations Pneumonia	11.1% 1/9 • Day 1 up to maximum of 32 days of follow up post last dose of study drug, where maximum treatment duration was 42 days (maximum up to 74 days) Same event may appear as AE and SAE, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study.
Infections and infestations Pneumonia bacterial	22.2% 2/9 • Day 1 up to maximum of 32 days of follow up post last dose of study drug, where maximum treatment duration was 42 days (maximum up to 74 days) Same event may appear as AE and SAE, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study.
Infections and infestations Bacteraemia	11.1% 1/9 • Day 1 up to maximum of 32 days of follow up post last dose of study drug, where maximum treatment duration was 42 days (maximum up to 74 days) Same event may appear as AE and SAE, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study.
Infections and infestations Bacterial sepsis	11.1% 1/9 • Day 1 up to maximum of 32 days of follow up post last dose of study drug, where maximum treatment duration was 42 days (maximum up to 74 days) Same event may appear as AE and SAE, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study.
Infections and infestations Conjunctivitis	11.1% 1/9 • Day 1 up to maximum of 32 days of follow up post last dose of study drug, where maximum treatment duration was 42 days (maximum up to 74 days) Same event may appear as AE and SAE, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study.
Infections and infestations Urinary tract infection	11.1% 1/9 • Day 1 up to maximum of 32 days of follow up post last dose of study drug, where maximum treatment duration was 42 days (maximum up to 74 days) Same event may appear as AE and SAE, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study.
Metabolism and nutrition disorders Hypokalaemia	11.1% 1/9 • Day 1 up to maximum of 32 days of follow up post last dose of study drug, where maximum treatment duration was 42 days (maximum up to 74 days) Same event may appear as AE and SAE, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study.
Musculoskeletal and connective tissue disorders Muscle atrophy	11.1% 1/9 • Day 1 up to maximum of 32 days of follow up post last dose of study drug, where maximum treatment duration was 42 days (maximum up to 74 days) Same event may appear as AE and SAE, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study.
Nervous system disorders Seizure	11.1% 1/9 • Day 1 up to maximum of 32 days of follow up post last dose of study drug, where maximum treatment duration was 42 days (maximum up to 74 days) Same event may appear as AE and SAE, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study.
Psychiatric disorders Adjustment disorder with depressed mood	11.1% 1/9 • Day 1 up to maximum of 32 days of follow up post last dose of study drug, where maximum treatment duration was 42 days (maximum up to 74 days) Same event may appear as AE and SAE, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study.
Psychiatric disorders Anxiety	11.1% 1/9 • Day 1 up to maximum of 32 days of follow up post last dose of study drug, where maximum treatment duration was 42 days (maximum up to 74 days) Same event may appear as AE and SAE, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study.
Psychiatric disorders Confusional state	11.1% 1/9 • Day 1 up to maximum of 32 days of follow up post last dose of study drug, where maximum treatment duration was 42 days (maximum up to 74 days) Same event may appear as AE and SAE, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study.
Psychiatric disorders Insomnia	11.1% 1/9 • Day 1 up to maximum of 32 days of follow up post last dose of study drug, where maximum treatment duration was 42 days (maximum up to 74 days) Same event may appear as AE and SAE, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study.
Respiratory, thoracic and mediastinal disorders Bronchospasm	11.1% 1/9 • Day 1 up to maximum of 32 days of follow up post last dose of study drug, where maximum treatment duration was 42 days (maximum up to 74 days) Same event may appear as AE and SAE, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study.
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	11.1% 1/9 • Day 1 up to maximum of 32 days of follow up post last dose of study drug, where maximum treatment duration was 42 days (maximum up to 74 days) Same event may appear as AE and SAE, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study.
Respiratory, thoracic and mediastinal disorders Pneumonia aspiration	11.1% 1/9 • Day 1 up to maximum of 32 days of follow up post last dose of study drug, where maximum treatment duration was 42 days (maximum up to 74 days) Same event may appear as AE and SAE, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study.
Respiratory, thoracic and mediastinal disorders Pulmonary oedema	11.1% 1/9 • Day 1 up to maximum of 32 days of follow up post last dose of study drug, where maximum treatment duration was 42 days (maximum up to 74 days) Same event may appear as AE and SAE, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study.
Respiratory, thoracic and mediastinal disorders Respiratory distress	11.1% 1/9 • Day 1 up to maximum of 32 days of follow up post last dose of study drug, where maximum treatment duration was 42 days (maximum up to 74 days) Same event may appear as AE and SAE, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study.
Skin and subcutaneous tissue disorders Pruritus	22.2% 2/9 • Day 1 up to maximum of 32 days of follow up post last dose of study drug, where maximum treatment duration was 42 days (maximum up to 74 days) Same event may appear as AE and SAE, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study.
Vascular disorders Hypertension	22.2% 2/9 • Day 1 up to maximum of 32 days of follow up post last dose of study drug, where maximum treatment duration was 42 days (maximum up to 74 days) Same event may appear as AE and SAE, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study.
Vascular disorders Hypotension	11.1% 1/9 • Day 1 up to maximum of 32 days of follow up post last dose of study drug, where maximum treatment duration was 42 days (maximum up to 74 days) Same event may appear as AE and SAE, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study.

Additional Information

Study Director

Basilea Pharmaceutica International Ltd, Allschwil

Phone: +41 79 701 0551

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee The Sponsor has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
Publication restrictions are in place

Restriction type: OTHER