Trial Outcomes & Findings for An Efficacy and Safety Study of APX001 in Non-Neutropenic Patients With Candidemia (NCT NCT03604705)
NCT ID: NCT03604705
Last Updated: 2025-09-16
Results Overview
Treatment Success is defined as meeting all of the following criteria: Two consecutive blood cultures negative for Candida spp. Alive at EOST No concomitant use of any other systemic antifungal therapies through end of study treatment
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
21 participants
Primary outcome timeframe
One to forty-two days
Results posted on
2025-09-16
Participant Flow
Participant milestones
| Measure |
APX001 Treatment
APX001: APX001 IV administration followed by APX001 oral tablet administration
|
|---|---|
|
Overall Study
STARTED
|
21
|
|
Overall Study
COMPLETED
|
20
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
An Efficacy and Safety Study of APX001 in Non-Neutropenic Patients With Candidemia
Baseline characteristics by cohort
| Measure |
Treatment Period - MITT
n=20 Participants
Evaluation of APX001 for the first-line treatment for candidemia, including suspected or confirmed antifungal-resistant candidemia, in non-neutropenic patients ≥ 18 years of age who had at least 1 positive blood culture within the 96 hours prior to starting study drug. Modified Intent-to-Treat (MITT) Population. The MITT Population contained 20 (95.2%) patients.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
10 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
10 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
7 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
13 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
18 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 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
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
19 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
|
|
Region of Enrollment
Belgium
|
7 participants
n=5 Participants
|
|
Region of Enrollment
United States
|
3 participants
n=5 Participants
|
|
Region of Enrollment
Israel
|
9 participants
n=5 Participants
|
|
Region of Enrollment
Spain
|
1 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: One to forty-two daysTreatment Success is defined as meeting all of the following criteria: Two consecutive blood cultures negative for Candida spp. Alive at EOST No concomitant use of any other systemic antifungal therapies through end of study treatment
Outcome measures
| Measure |
Treatment Period - MITT
n=20 Participants
Treatment Success was defined as meeting all of the following criteria: 1) 2 consecutive blood cultures were negative for Candida spp.; 2) Alive at EOST; and 3) No concomitant use of any other systemic antifungal therapies through EOST. Treatment Failure is defined as any case that does not meet the criteria for Treatment Success.
|
EOT (End of Antifungal Treatment)
Eradication
|
Follow-up 2 Weeks After EOT
Recurrence
|
Follow-up 4 Weeks After EOT
Recurrence
|
|---|---|---|---|---|
|
Treatment Success at End of Study Treatment (EOST) as Determined by the Data Review Committee (DRC)
|
16 Participants
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: One to forty-nine daysTime to first negative blood culture was defined as the number of days from first dose date of study drug to the date of first post-Baseline negative blood culture + 1. Patients without a negative blood culture at post-Baseline visits were censored at the last assessment date.
Outcome measures
| Measure |
Treatment Period - MITT
n=20 Participants
Treatment Success was defined as meeting all of the following criteria: 1) 2 consecutive blood cultures were negative for Candida spp.; 2) Alive at EOST; and 3) No concomitant use of any other systemic antifungal therapies through EOST. Treatment Failure is defined as any case that does not meet the criteria for Treatment Success.
|
EOT (End of Antifungal Treatment)
Eradication
|
Follow-up 2 Weeks After EOT
Recurrence
|
Follow-up 4 Weeks After EOT
Recurrence
|
|---|---|---|---|---|
|
Time to First Negative Blood Culture
|
2.4 Days
Standard Deviation 1.13
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: End of study treatment (EOST), end of treatment (EOT), and 2 and 4 weeks after end of treatment (EOT)Outcome measures
| Measure |
Treatment Period - MITT
n=20 Participants
Treatment Success was defined as meeting all of the following criteria: 1) 2 consecutive blood cultures were negative for Candida spp.; 2) Alive at EOST; and 3) No concomitant use of any other systemic antifungal therapies through EOST. Treatment Failure is defined as any case that does not meet the criteria for Treatment Success.
|
EOT (End of Antifungal Treatment)
n=20 Participants
Eradication
|
Follow-up 2 Weeks After EOT
n=20 Participants
Recurrence
|
Follow-up 4 Weeks After EOT
n=20 Participants
Recurrence
|
|---|---|---|---|---|
|
Percentage of Patients With Mycological Outcomes at End of Study Treatment (EOST), End of Treatment (EOT), and 2 and 4 Weeks After End of Treatment (EOT)
|
16 Participants
|
15 Participants
|
1 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: 2 and 4 weeks after end of treatment (EOT)Outcome measures
| Measure |
Treatment Period - MITT
n=20 Participants
Treatment Success was defined as meeting all of the following criteria: 1) 2 consecutive blood cultures were negative for Candida spp.; 2) Alive at EOST; and 3) No concomitant use of any other systemic antifungal therapies through EOST. Treatment Failure is defined as any case that does not meet the criteria for Treatment Success.
|
EOT (End of Antifungal Treatment)
n=20 Participants
Eradication
|
Follow-up 2 Weeks After EOT
Recurrence
|
Follow-up 4 Weeks After EOT
Recurrence
|
|---|---|---|---|---|
|
Percentage of Patients With Treatment Success at End of Treatment (EOT), and 2 and 4 Weeks After End of Treatment (EOT)
|
60.0 percentage of participants
|
55.0 percentage of participants
|
—
|
—
|
SECONDARY outcome
Timeframe: Day 30Outcome measures
| Measure |
Treatment Period - MITT
n=20 Participants
Treatment Success was defined as meeting all of the following criteria: 1) 2 consecutive blood cultures were negative for Candida spp.; 2) Alive at EOST; and 3) No concomitant use of any other systemic antifungal therapies through EOST. Treatment Failure is defined as any case that does not meet the criteria for Treatment Success.
|
EOT (End of Antifungal Treatment)
Eradication
|
Follow-up 2 Weeks After EOT
Recurrence
|
Follow-up 4 Weeks After EOT
Recurrence
|
|---|---|---|---|---|
|
Overall Survival at Study Day 30
|
17 Participants
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: One to forty-nine daysOutcome measures
| Measure |
Treatment Period - MITT
n=21 Participants
Treatment Success was defined as meeting all of the following criteria: 1) 2 consecutive blood cultures were negative for Candida spp.; 2) Alive at EOST; and 3) No concomitant use of any other systemic antifungal therapies through EOST. Treatment Failure is defined as any case that does not meet the criteria for Treatment Success.
|
EOT (End of Antifungal Treatment)
Eradication
|
Follow-up 2 Weeks After EOT
Recurrence
|
Follow-up 4 Weeks After EOT
Recurrence
|
|---|---|---|---|---|
|
Number of Patients With Treatment Emergent Adverse Events (TEAEs)
|
20 Participants
|
—
|
—
|
—
|
Adverse Events
Safety Population
Serious events: 9 serious events
Other events: 20 other events
Deaths: 5 deaths
Serious adverse events
| Measure |
Safety Population
n=21 participants at risk
Twenty-one patients were dosed with APX001 and composed the ITT/Safety Population.
|
|---|---|
|
Infections and infestations
Septic shock
|
9.5%
2/21 • Number of events 2 • One to forty-nine days
|
|
Infections and infestations
Bacteraemia
|
4.8%
1/21 • Number of events 1 • One to forty-nine days
|
|
Infections and infestations
Bacterial sepsis
|
4.8%
1/21 • Number of events 1 • One to forty-nine days
|
|
Infections and infestations
Enterobacter sepsis
|
4.8%
1/21 • Number of events 1 • One to forty-nine days
|
|
Infections and infestations
Necrotising fasciitis
|
4.8%
1/21 • Number of events 1 • One to forty-nine days
|
|
Infections and infestations
Sepsis
|
4.8%
1/21 • Number of events 1 • One to forty-nine days
|
|
Infections and infestations
Stenotrophomonas sepsis
|
4.8%
1/21 • Number of events 1 • One to forty-nine days
|
|
Infections and infestations
Systemic candida
|
4.8%
1/21 • Number of events 1 • One to forty-nine days
|
|
Infections and infestations
Urinary tract infection bacterial
|
4.8%
1/21 • Number of events 1 • One to forty-nine days
|
|
Cardiac disorders
Cardiac failure congestive
|
4.8%
1/21 • Number of events 1 • One to forty-nine days
|
|
Cardiac disorders
Cardio-respiratory arrest
|
4.8%
1/21 • Number of events 1 • One to forty-nine days
|
|
General disorders
Euthanasia
|
4.8%
1/21 • Number of events 1 • One to forty-nine days
|
|
General disorders
General physical health deterioration
|
4.8%
1/21 • Number of events 1 • One to forty-nine days
|
|
Respiratory, thoracic and mediastinal disorders
Acute respiratory failure
|
4.8%
1/21 • Number of events 1 • One to forty-nine days
|
|
Respiratory, thoracic and mediastinal disorders
Interstitial lung disease
|
4.8%
1/21 • Number of events 1 • One to forty-nine days
|
|
Blood and lymphatic system disorders
Leukopenia
|
4.8%
1/21 • Number of events 1 • One to forty-nine days
|
|
Gastrointestinal disorders
Gastrointestinal fistula
|
4.8%
1/21 • Number of events 1 • One to forty-nine days
|
Other adverse events
| Measure |
Safety Population
n=21 participants at risk
Twenty-one patients were dosed with APX001 and composed the ITT/Safety Population.
|
|---|---|
|
Infections and infestations
Bacteraemia
|
9.5%
2/21 • Number of events 2 • One to forty-nine days
|
|
Infections and infestations
Septic shock
|
9.5%
2/21 • Number of events 2 • One to forty-nine days
|
|
Gastrointestinal disorders
Diarrhoea
|
14.3%
3/21 • Number of events 3 • One to forty-nine days
|
|
Gastrointestinal disorders
Vomiting
|
14.3%
3/21 • Number of events 3 • One to forty-nine days
|
|
Gastrointestinal disorders
Nausea
|
9.5%
2/21 • Number of events 2 • One to forty-nine days
|
|
General disorders
Oedema peripheral
|
14.3%
3/21 • Number of events 3 • One to forty-nine days
|
|
General disorders
Pyrexia
|
9.5%
2/21 • Number of events 2 • One to forty-nine days
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
14.3%
3/21 • Number of events 3 • One to forty-nine days
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
9.5%
2/21 • Number of events 2 • One to forty-nine days
|
|
Renal and urinary disorders
Acute kidney injury
|
9.5%
2/21 • Number of events 2 • One to forty-nine days
|
|
Renal and urinary disorders
Hydronephrosis
|
9.5%
2/21 • Number of events 2 • One to forty-nine days
|
|
Renal and urinary disorders
Renal failure
|
9.5%
2/21 • Number of events 2 • One to forty-nine days
|
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
- Publication restrictions are in place