Clinical trial NCT03604705: An Efficacy and Safety Study of APX001 in Non-Neutropenic Patients With Candidemia
Official title: An Open-Label Study to Evaluate the Efficacy and Safety of APX001 in Non Neutropenic Patients With Candidemia, With or Without Invasive Candidiasis, Inclusive of Patients With Suspected Resistance to Standard of Care Antifungal Treatment
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Candidemia
Lead sponsor: Basilea Pharmaceutica
Sponsor class: INDUSTRY
First posted: 2018-07-27
Enrollment: 21
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This is a multicenter, open-label, non-comparative, single-arm study to evaluate the efficacy and safety of APX001 for the first-line treatment for candidemia including suspected or confirmed antifungal-resistant candidemia in non-neutropenic patients 18 yeas of age and older.

Suspicion of antifungal-resistant candidemia is sufficient (documented resistance is not required for enrollment). The Study Drug Treatment Period of APX001 will be a maximum of 14 days. After completion of 14 days study drug therapy, if further antifungal treatment is indicated to complete treatment of candidemia in accordance with standard practice guidelines, fluconazole (unless susceptibility results warrant alternative antifungal therapy) may commence for up to a further 7 days. There will be a Follow up Period of 4 weeks (+4 days) after EOT. The total duration of participation in the study is up to approximately 7.5 weeks.

This study will be conducted at approximately 20 sites in the United States and globally.


Eligibility criteria: Key Inclusion Criteria:

* Provision of written consent
* Adults ages 18 and above male or female
* New diagnosis of candidemia
* Able to have pre-existing intravascular catheters removed and replaced (as necessary)

Key Exclusion Criteria:

* neutropenia
* deep-seated Candida-related infections
* hepatosplenic candidiasis
* received more than 2 days of prior systemic antifungal treatment for current candidemia episode
* severe hepatic impairment

Primary outcomes:
measure: Treatment Success at End of Study Treatment (EOST) as Determined by the Data Review Committee (DRC)
timeFrame: One to forty-two days
Secondary outcomes:
measure: Time to First Negative Blood Culture
timeFrame: One to forty-nine days
measure: 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)
timeFrame: End of study treatment (EOST), end of treatment (EOT), and 2 and 4 weeks after end of treatment (EOT)
measure: Percentage of Patients With Treatment Success at End of Treatment (EOT), and 2 and 4 Weeks After End of Treatment (EOT)
timeFrame: 2 and 4 weeks after end of treatment (EOT)
measure: Overall Survival at Study Day 30
timeFrame: Day 30
measure: Number of Patients With Treatment Emergent Adverse Events (TEAEs)
timeFrame: One to forty-nine days
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama at Birmingham School of Medicine
state: Alabama
city: Birmingham
country: United States
facility: University of Alabama at Birmingham (UAB)
state: Alabama
city: Davis
country: United States
facility: University of California, Davis
state: California
city: Sacramento
country: United States
facility: University of California-Davis Medical Center
state: California
city: Augusta
country: United States
facility: Augusta University (Georgia Regents University)
state: Georgia
city: Chicago
country: United States
facility: University of Chicago
state: Illinois
city: Saint Louis
country: United States
facility: Washington University
state: Missouri
city: Durham
country: United States
facility: Duke University Hospital Medical Center
state: North Carolina
city: Houston
country: United States
facility: University of Texas- Health Science Center and Medical School at Houston
state: Texas
city: Brussels
country: Belgium
facility: Institut Jules Bordet,Service De Microbiologie
city: Brussels
country: Belgium
facility: Hopital Erasme
city: Brussels
country: Belgium
facility: Institut Jules Bordet
city: Brussels
country: Belgium
facility: Universite Libre de Bruxelles (ULB) - Hopital Erasme
city: Lodelinsart
country: Belgium
facility: CHU de Charleroi - Hopital Civil Marie Curie
city: Yvoir
country: Belgium
facility: Mont-Godinne University Hospital
city: Yvoir
country: Belgium
facility: University Hospital Mont-Godinne
city: Cologne
country: Germany
facility: Klinik I fur Innere Medizin- Uniklinik Koln
city: Heidelberg
country: Germany
facility: University Hospital Heidelberg
city: Mainz
country: Germany
facility: Universitaetsmedizin der Johannes Gutenberg-Universitaet Mainz - III. Medizinische Klinik Haematologie/Onkologie
city: Haifa
country: Israel
facility: Infectious Diseases Unit, Rambam Medical Center
city: Haifa
country: Israel
facility: Rambam Medical Center
city: Tel Aviv
country: Israel
facility: Infectious Diseases Unit
city: Tel Aviv
country: Israel
facility: Sourasky Medical Center
city: Tel Hashomer
country: Israel
facility: Infectious Diseases Unit,Sheba Medical Center
city: Tel HaShomer
country: Israel
facility: Sheba Medical Center
city: Terrassa
country: Spain
facility: Hospital Universitario Mutua de Terrassa
state: Barcelona
city: Alicante
country: Spain
facility: Hospital General Universitario de Alicante
city: Barcelona
country: Spain
facility: Hospital Universitari Vall d'Hebron
city: Barcelona
country: Spain
facility: Hospital VLL D Hebron
Study officials:
affiliation: Basilea Pharmaceutica International Ltd, Allschwil
name: Marc Engelhardt
role: STUDY_DIRECTOR
References: