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

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ClinicalTrials.gov Identifier: NCT04148287
Recruitment Status : Terminated (The trial was closed early as the result of the impact of COVID-19 on trial-related activities. The study was not terminated due to any safety issues or concerns. The objectives of the study were successfully met.)
First Posted : November 1, 2019
Last Update Posted : April 23, 2021
Sponsor:
Information provided by (Responsible Party):
Amplyx Pharmaceuticals

Tracking Information
First Submitted Date  ICMJE October 30, 2019
First Posted Date  ICMJE November 1, 2019
Last Update Posted Date April 23, 2021
Actual Study Start Date  ICMJE September 15, 2019
Actual Primary Completion Date November 30, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: October 30, 2019)
Treatment Success [ Time Frame: Up to 42 days ]
The primary efficacy parameter is percentage of patients with Treatment Success at end of study treatment as determined by the Data Review Committee (DRC).
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE An Open-label Study of APX001 for Treatment of Patients With Candidemia/Invasive Candidiasis Caused by Candida Auris
Official Title  ICMJE An Open-Label Study to Evaluate the Efficacy and Safety of APX001 in Patients With Candidemia and/or Invasive Candidiasis Caused by Candida Auris
Brief Summary This is a multicenter, open-label, single arm study to evaluate the efficacy and safety of APX001 for the treatment of candidemia and/or invasive candidiasis caused by C. auris in patients aged 18 years and over with limited antifungal treatment options.
Detailed Description This is a multicenter, open-label, non-comparative, single arm study to evaluate the efficacy and safety of APX001 for the treatment of candidemia and/or invasive candidiasis caused by C. auris in patients aged 18 years and over with limited antifungal treatment options. The Study Drug Treatment Period will be up to a maximum of 42 days (inclusive of the loading dose [Study Day 1]). There will be a Follow up Period of 4 weeks (+4 days) after EOST. The total duration of participation in the study is up to approximately 10.5 weeks (inclusive of the Screening Period [168 hours prior to Baseline]).
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description:
Open-label, single arm intervention. All patients will receive APX001. On Days 1-3, APX001 will be administered intravenously. Thereafter, if the patient is able to take oral medication, APX001 tablets will be taken orally.
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Candidemia
  • Invasive Candidiases
  • Candida Infection
Intervention  ICMJE Drug: APX001
Day 1: APX001 1000 mg IV BID over a 3-hour infusion Days 2-3: APX001 600 mg IV QD over a 3-hour infusion Days 4 - 42: APX001 600 mg IV QD over a 3-hour infusion or APX001 800 mg QD oral.
Other Name: fosmanogepix
Study Arms  ICMJE Experimental: APX001
APX001 IV or oral for up to 42 days
Intervention: Drug: APX001
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Terminated
Actual Enrollment  ICMJE
 (submitted: March 5, 2021)
9
Original Estimated Enrollment  ICMJE
 (submitted: October 30, 2019)
15
Actual Study Completion Date  ICMJE December 31, 2020
Actual Primary Completion Date November 30, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Limited or no treatment options due to resistance, contraindication, intolerance or lack of clinical response to standard of care antifungal therapy, as advocated by the relevant regional/country treatment guidelines
  • Established mycological and clinical diagnosis of candidemia and/or invasive candidiasis caused by Candida auris
  • Able to have pre-existing intravascular catheters removed and replaced (if necessary)
  • Females of childbearing potential with male partners, and males with female partner(s) of childbearing potential, must agree to use 2 forms of highly effective contraception throughout the duration of the study and for 90 days following the last study drug administration. Females of childbearing potential must have a negative urine pregnancy test within 96 hours prior study entry.
  • Wiling to participate in the study, willing to give written informed consent, and willing to comply with the study restrictions; where permitted by local regulations, written informed consent from a legal authorized representative (LAR) will be obtained for patients who are unable to give consent

Exclusion Criteria:

  • Life expectancy of less than 7 days in the opinion of the Investigator
  • Human immunodeficiency virus-infected patients who are receiving antiretroviral therapy that are moderate to strong inducers of CYP3A4, or who have detectable viremia, or who have had an active opportunistic infection within 6 months prior
  • Alanine aminotransferase or aspartate aminotransferase greater than or equal to 5 times the upper limit of normal
  • Total bilirubin greater than 3 time the upper limit of normal, unless isolated hyperbilirubinemia or due to documented Gilbert's disease
  • Pregnant or lactating female patient
  • Inappropriate fungal infection source control
  • Investigational drug administered within 30 days prior to dosing or five half-lives whichever is longer
  • Diagnosis of deep-seated Candida-related infections causing hardware associated septic arthritis, osteomyelitis, endocarditis, myocarditis, hepatosplenic candidiasis, or a central nervous system infection or site of infection that would require antifungal therapy to exceed the maximal duration of study drug treatment
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Panama,   South Africa
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04148287
Other Study ID Numbers  ICMJE APX001-203
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Yes
Plan Description: IPD will be shared in a manuscript. All IPD will be anonymized. IPD will include summarized efficacy, safety and pharmacokinetics data
Supporting Materials: Study Protocol
Supporting Materials: Clinical Study Report (CSR)
Time Frame: Approximately 6 months following the last patient's last visit in the study.
Responsible Party Amplyx Pharmaceuticals
Study Sponsor  ICMJE Amplyx Pharmaceuticals
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Amplyx Pharmaceuticals
Verification Date July 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP