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Liposomal Amphotericin B and Flucytosine Antifungal Strategy for Talaromycosis (LAmB-FAST)

Phase 3
Not yet recruiting
Conditions
Talaromycosis
Interventions
Registration Number
NCT06525389
Lead Sponsor
Duke University
Brief Summary

LAmB-FAST is a factorial randomized controlled trial simultaneously testing two interventions in one trial. LAmB-FAST seeks to inform treatment guidelines on the induction and maintenance therapy of HIV-associated talaromycosis (formerly called penicilliosis) and will answer the following three questions:

1. Is induction therapy using a single 10 mg\\/kg dose of liposomal amphotericin B (LAmB) is more effective than 14 days of the conventional deoxycholate amphotericin B (DAmB)?

2. Is adding flucytosine (5FC) to amphotericin B more effective than amphotericin B alone?

3. Is HIV viral load guided stopping of itraconazole maintenance therapy as effective as the current CD4 guided strategy in the prevention of talaromycosis relapse?

Detailed Description

Talaromycosis (formerly known as penicilliosis) is caused by the dimorphic fungus Talaromyces marneffei (Tm) endemic in Southeast Asia where it is a leading the cause of death among people with advanced HIV disease (AHD, CD4 count \<200 cells/mm3 and/or WHO disease stage III or IV). Despite the mortality on treatment as high as 30%, current treatment options are limited to just two drugs: amphotericin B deoxycholate (DAmB) - which has substantial toxicity, and itraconazole - which has poor bioavailability.

As a roadmap to identify safer and more effective antifungal strategies, LAmB-FAST applies major advances made in HIV-associated mycoses to accelerate treatment for HIV-associated talaromycosis. First, clinical trials in cryptococcosis showed that shorter courses (5 to 7 days) of DAmB was as effective and less toxic than the standard 14-day course of DAmB. The recent AMBITION cryptococcal meningitis showed that a single 10 mg/kg dose of liposomal amphotericin B (LAmB) was as effective as 7 to 14 days of DAmB but had 30% less toxicity, leading to rapid endorsement by the WHO as the first-line therapy for cryptococcal meningitis in 2022. A single LAmB induction therapy strategy has also been demonstrated in a phase II HIV-associated histoplasmosis trial which showed that a single 10 mg/kg dose of LAmB had similar mortality compared to 2 doses or 14 daily doses of LAmB. Second, the addition of flucytosine (5FC) to DAmB has been shown to improves fungal clearance in the cerebrospinnal fluid and survival of patients with cryptococcal meningitis. These advances in other HIV-associated mycoses lead us to hypothesize that 1) a single 10mg/kg dose of LAmB will be superior to 14 days of DAmB and 2) the addition of 5FC will be superior to DAmB or LAmB alone in the induction therapy of talaromyosis.

LAmB-FAST will test three related but independent specific aims: AIM 1: To determine if a single 10mg/kg dose of LAmB is superior to 14 days of DAmB in Tm complication-free survival. AIM 2:

To determine if combination therapy with 5FC is superior to DAmB or LAmB alone in Tm complication-free survival.

The primary outcome (for both AIM 1 and AIM 2) is hazard of a composite of death, Tm complications, and adverse events (AEs) grade 3 or higher.

The secondary outcomes include:

1. All-cause mortality;

2. Fungal clearance rate over first 14 days;

3. A novel 4-scale hierarchical outcome of i. Mortality, ii. Tm complications, iii. AEs grade 3, iv. Quality of life scores;

4. Rates of Tm DNA and Tm antigen decline over first 12 weeks.

AIM 3 will leverage access to a well-characterized and treated talaromycosis cohort in AIM 1 and AIM 2 to conduct a follow-on nested randomized controlled sub-study testing whether a HIV viral load guided strategy of stopping itraconazole chemoprophylaxis (STOP SHORT) is non-inferior to the current CD4 guided strategy in the prevention of talaromycosis relapse and death.

Recruitment & Eligibility

Status
NOT_YET_RECRUITING
Sex
All
Target Recruitment
428
Inclusion Criteria
  • HIV infected adults (age greater or equal to 18), on ART or no ART
  • Definitive talaromycosis confirmed by microscopy, histology, or culture
Exclusion Criteria
  • Known severe allergy to AmB or 5FC
  • Absolute neutrophil count <500 cells
  • Concurrent cryptococcal or TB meningitis
  • Received > 2 doses of DAmB
  • Pregnancy

Study & Design

Study Type
INTERVENTIONAL
Study Design
FACTORIAL
Arm && Interventions
GroupInterventionDescription
Deoxycholate Amphotericin B (DAmB) plus Flucytosine (5FC) placeboDeoxycholate Amphotericin B (DAmB)DAmB (0.7mg/kg/d IV x 14 days) + 5FC placebo (25 mg/kg oral 3x daily x 14 days)
Deoxycholate Amphotericin B (DAmB) plus Flucytosine (5FC)Flucytosine (5FC)DAmB (0.7 mg/kg/d IV x 14 days) + 5FC (25 mg/kg oral 3x daily x 14 days)
Deoxycholate Amphotericin B (DAmB) plus Flucytosine (5FC)Deoxycholate Amphotericin B (DAmB)DAmB (0.7 mg/kg/d IV x 14 days) + 5FC (25 mg/kg oral 3x daily x 14 days)
Deoxycholate Amphotericin B (DAmB) plus Flucytosine (5FC) placeboFlucytosine (5FC) placebo pillDAmB (0.7mg/kg/d IV x 14 days) + 5FC placebo (25 mg/kg oral 3x daily x 14 days)
Liposomal Amphotericin B (LAmB) plus Flucytosine (5FC) placeboLiposomal Amphotericin B (LAmB)LAmB (10 mg/kg IV x 1 dose) + 5FC placebo (25 mg/kg oral 3x daily x 14 days)
Liposomal Amphotericin B (LAmB) plus Flucytosine (5FC)Liposomal Amphotericin B (LAmB)LAmB (10 mg/kg IV x 1 dose) + 5FC (25 mg/kg oral 3x daily x 14 days)
Liposomal Amphotericin B (LAmB) plus Flucytosine (5FC)Flucytosine (5FC)LAmB (10 mg/kg IV x 1 dose) + 5FC (25 mg/kg oral 3x daily x 14 days)
Liposomal Amphotericin B (LAmB) plus Flucytosine (5FC) placeboFlucytosine (5FC) placebo pillLAmB (10 mg/kg IV x 1 dose) + 5FC placebo (25 mg/kg oral 3x daily x 14 days)
Primary Outcome Measures
NameTimeMethod
Time from enrollment to a composite of poor outcomesup to 24 weeks

Poor outcomes are defined as of death, talaromycosis complications (defined as relapse, immune inflammatory reconstitution inflammatory syndrome \[IRIS\], wasting syndrome \[\>10% weight loss from enrollment\], re-hospitalization, and grade 3 or higher adverse events

Secondary Outcome Measures
NameTimeMethod
Fungal clearance rate as measured by early fungicidal activity (EFA) in log10 CFUs/mL/day14 days
All cause mortalityup to 24 weeks
Composite ordinal desirability of outcome ranking (DOOR) scaleover 24 weeks

All-cause mortality, Talaromycosis complications and adverse events grade 4, Adverse events grade 3, and Quality of Life (QOL) utility scores by EQ5D scale.

Change in Talaromyces marneffei antigen in µg/mL/weekbaseline to 24 weeks
Change in Talaromyces marneffei DNA in copies/mL/weekbaseline to 24 weeks

Trial Locations

Locations (5)

Duke University Medical Center

🇺🇸

Durham, North Carolina, United States

Bach Mai Hospital

🇻🇳

Hanoi, Vietnam

National Hospital for Tropical Diseases

🇻🇳

Hanoi, Vietnam

Hospital for Tropical Diseases

🇻🇳

Ho Chi Minh City, Vietnam

Pham Ngoc Thach University of Medicine

🇻🇳

Ho Chi Minh City, Vietnam

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