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Trial record 1 of 1 for:    NCT03604705
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An Efficacy and Safety Study of APX001 in Non-Neutropenic Patients With Candidemia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03604705
Recruitment Status : Completed
First Posted : July 27, 2018
Results First Posted : August 5, 2021
Last Update Posted : August 5, 2021
Sponsor:
Information provided by (Responsible Party):
Amplyx Pharmaceuticals

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Candidemia
Intervention Drug: APX001
Enrollment 21
Recruitment Details  
Pre-assignment Details  
Arm/Group Title APX001 Treatment
Hide Arm/Group Description APX001: APX001 IV administration followed by APX001 oral tablet administration
Period Title: Overall Study
Started 21
Completed 20
Not Completed 1
Arm/Group Title Treatment Period - MITT
Hide Arm/Group Description 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.
Overall Number of Baseline Participants 20
Hide Baseline Analysis Population Description
Treatment Period - MITT
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 20 participants
<=18 years
0
   0.0%
Between 18 and 65 years
10
  50.0%
>=65 years
10
  50.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 20 participants
Female
7
  35.0%
Male
13
  65.0%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 20 participants
Hispanic or Latino
0
   0.0%
Not Hispanic or Latino
18
  90.0%
Unknown or Not Reported
2
  10.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 20 participants
American Indian or Alaska Native
0
   0.0%
Asian
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
1
   5.0%
White
19
  95.0%
More than one race
0
   0.0%
Unknown or Not Reported
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 20 participants
Belgium 7
United States 3
Israel 9
Spain 1
1.Primary Outcome
Title Treatment Success at End of Study Treatment (EOST) as Determined by the Data Review Committee (DRC)
Hide Description

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

Time Frame One to forty-two days
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treatment Period - MITT
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 20
Measure Type: Count of Participants
Unit of Measure: Participants
16
  80.0%
2.Secondary Outcome
Title Time to First Negative Blood Culture
Hide Description Time 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.
Time Frame One to forty-nine days
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title MITT Population
Hide Arm/Group Description:
MITT = Modified Intent-to-Treat population
Overall Number of Participants Analyzed 20
Mean (Standard Deviation)
Unit of Measure: Days
2.4  (1.13)
3.Secondary Outcome
Title 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)
Hide Description [Not Specified]
Time Frame End of study treatment (EOST), end of treatment (EOT), and 2 and 4 weeks after end of treatment (EOT)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title EOST (End of Study Drug Treatment) EOT (End of Antifungal Treatment) Follow-up 2 Weeks After EOT Follow-up 4 Weeks After EOT
Hide Arm/Group Description:
Eradication
Eradication
Recurrence
Recurrence
Overall Number of Participants Analyzed 20 20 20 20
Measure Type: Count of Participants
Unit of Measure: Participants
16
  80.0%
15
  75.0%
1
   5.0%
0
   0.0%
4.Secondary Outcome
Title Percentage of Patients With Treatment Success at End of Treatment (EOT), and 2 and 4 Weeks After End of Treatment (EOT)
Hide Description [Not Specified]
Time Frame 2 and 4 weeks after end of treatment (EOT)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treatment Success 2 Weeks After EOT Treatment Success 4 Weeks After EOT
Hide Arm/Group Description:
Treatment Success determined by the DRC by visit for the MITT Population.
Treatment success determined by the DRC by visit for the MITT Population.
Overall Number of Participants Analyzed 20 20
Measure Type: Number
Unit of Measure: percentage of participants
60.0 55.0
5.Secondary Outcome
Title Overall Survival at Study Day 30
Hide Description [Not Specified]
Time Frame Day 30
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title MITT Population
Hide Arm/Group Description:
MITT = Modified Intent-to-Treat population
Overall Number of Participants Analyzed 20
Measure Type: Count of Participants
Unit of Measure: Participants
17
  85.0%
6.Secondary Outcome
Title Number of Patients With Treatment Emergent Adverse Events (TEAEs)
Hide Description [Not Specified]
Time Frame One to forty-nine days
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Safety Population
Hide Arm/Group Description:
Twenty-one patients were dosed with APX001 and composed the ITT/Safety Population.
Overall Number of Participants Analyzed 21
Measure Type: Count of Participants
Unit of Measure: Participants
20
  95.2%
Time Frame One to forty-nine days
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Safety Population
Hide Arm/Group Description Twenty-one patients were dosed with APX001 and composed the ITT/Safety Population.
All-Cause Mortality
Safety Population
Affected / at Risk (%)
Total   5/21 (23.81%)    
Hide Serious Adverse Events
Safety Population
Affected / at Risk (%) # Events
Total   9/21 (42.86%)    
Blood and lymphatic system disorders   
Leukopenia   1/21 (4.76%)  1
Cardiac disorders   
Cardiac failure congestive   1/21 (4.76%)  1
Cardio-respiratory arrest   1/21 (4.76%)  1
Gastrointestinal disorders   
Gastrointestinal fistula   1/21 (4.76%)  1
General disorders   
Euthanasia   1/21 (4.76%)  1
General physical health deterioration   1/21 (4.76%)  1
Infections and infestations   
Septic shock   2/21 (9.52%)  2
Bacteraemia   1/21 (4.76%)  1
Bacterial sepsis   1/21 (4.76%)  1
Enterobacter sepsis   1/21 (4.76%)  1
Necrotising fasciitis   1/21 (4.76%)  1
Sepsis   1/21 (4.76%)  1
Stenotrophomonas sepsis   1/21 (4.76%)  1
Systemic candida   1/21 (4.76%)  1
Urinary tract infection bacterial   1/21 (4.76%)  1
Respiratory, thoracic and mediastinal disorders   
Acute respiratory failure   1/21 (4.76%)  1
Interstitial lung disease   1/21 (4.76%)  1
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Safety Population
Affected / at Risk (%) # Events
Total   20/21 (95.24%)    
Gastrointestinal disorders   
Diarrhoea   3/21 (14.29%)  3
Vomiting   3/21 (14.29%)  3
Nausea   2/21 (9.52%)  2
General disorders   
Oedema peripheral   3/21 (14.29%)  3
Pyrexia   2/21 (9.52%)  2
Infections and infestations   
Bacteraemia   2/21 (9.52%)  2
Septic shock   2/21 (9.52%)  2
Renal and urinary disorders   
Acute kidney injury   2/21 (9.52%)  2
Hydronephrosis   2/21 (9.52%)  2
Renal failure   2/21 (9.52%)  2
Respiratory, thoracic and mediastinal disorders   
Pleural effusion   3/21 (14.29%)  3
Dyspnoea   2/21 (9.52%)  2
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Chief Medical Officer
Organization: Amplyx Pharmaceuticals
Phone: 8588427854
EMail: mhodges@amplyx.com
Layout table for additonal information
Responsible Party: Amplyx Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03604705    
Other Study ID Numbers: APX001-201
First Submitted: July 19, 2018
First Posted: July 27, 2018
Results First Submitted: May 24, 2021
Results First Posted: August 5, 2021
Last Update Posted: August 5, 2021