CLINICAL TRIALS PROFILE FOR AMPHOTERICIN B

505(b)(2) Clinical Trials for Amphotericin B

This table shows clinical trials for potential 505(b)(2) applications. See the next table for all clinical trials

Trial Type	Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
New Dosage	NCT00421187 ↗	Ambisome and Management of Culture-negative Neutropenic Fever Unresponsive to Antibiotics	Terminated	Gilead Sciences	Phase 4	2007-03-01	Administration of a single high dose (10 mg/kg) of AmBisome® no later than 72 hours after ARNF onset followed by two 5 mg/kg doses on days 2 and 5 may provide sustained tissue levels of amphotericin B that are as mycologically effective as those provided after administering the standard daily dose of 3 mg/kg/day. The new dosing regimen is anticipated to be equally clinically effective compared with the standard AmBisome® regimen when given for the duration of neutropenic fever in patients with ARNF. In addition, the degree and incidence of nephrotoxicity are predicted to be lower with the 3 sequential dose regimen compared to daily dosing with 3 mg/kg because of the lower cumulative dosage (20 mg/kg versus 42 mg/kg, respectively), which is 1 contributing factor for the development of acute renal failure. Furthermore, the lower cumulative dose may be a cost-effective strategy for the treatment of patients with ARNF.
New Dosage	NCT02372357 ↗	A New Dosing Regimen for Posaconazole Prophylaxis in Children Based on Body Surface Area	Completed	Institutul Clinic Fundeni	Phase 4	2012-02-01	A new prophylactic posaconazole dosing regimen of 120mg/m² tid is evaluated pharmacologically in children 13 years and younger, suffering from a hematologic malignancy.
New Dosage	NCT02372357 ↗	A New Dosing Regimen for Posaconazole Prophylaxis in Children Based on Body Surface Area	Completed	Institutul Clinic Fundeni Bucharest	Phase 4	2012-02-01	A new prophylactic posaconazole dosing regimen of 120mg/m² tid is evaluated pharmacologically in children 13 years and younger, suffering from a hematologic malignancy.

All Clinical Trials for Amphotericin B

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00000639 ↗	A Randomized Double Blind Protocol Comparing Amphotericin B With Flucytosine to Amphotericin B Alone Followed by a Comparison of Fluconazole and Itraconazole in the Treatment of Acute Cryptococcal Meningitis	Completed	Washington University School of Medicine	N/A	1969-12-31	To evaluate the effectiveness and safety of amphotericin B plus flucytosine (5-fluorocytosine) compared to amphotericin B alone for a first episode of acute cryptococcal meningitis in AIDS patients, and to compare the effectiveness and safety of fluconazole versus itraconazole. At least 10 percent of patients with a low CD4 count and HIV infection will develop meningitis due to Cryptococcus neoformans. More effective treatments than the standard therapy need to be explored.
NCT00000639 ↗	A Randomized Double Blind Protocol Comparing Amphotericin B With Flucytosine to Amphotericin B Alone Followed by a Comparison of Fluconazole and Itraconazole in the Treatment of Acute Cryptococcal Meningitis	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	N/A	1969-12-31	To evaluate the effectiveness and safety of amphotericin B plus flucytosine (5-fluorocytosine) compared to amphotericin B alone for a first episode of acute cryptococcal meningitis in AIDS patients, and to compare the effectiveness and safety of fluconazole versus itraconazole. At least 10 percent of patients with a low CD4 count and HIV infection will develop meningitis due to Cryptococcus neoformans. More effective treatments than the standard therapy need to be explored.
NCT00000677 ↗	SCH 39304 as Therapy for Acute Cryptococcal Meningitis in HIV-Infected Patients Followed by Maintenance Therapy	Completed	Schering-Plough	Phase 1	1969-12-31	To assess the safety and effectiveness of SCH 39304 as primary treatment of acute cryptococcal meningitis in HIV-infected patients. Safety and effectiveness of maintenance therapy following successful treatment of acute disease are also evaluated. Cryptococcal meningitis is a significant cause of illness and death in HIV-infected patients. Intravenous amphotericin B is effective for acute disease but relapse occurs in the majority of patients. Maintenance therapy is recommended but must be balanced against the multiple toxicities of the drugs used and the problems associated with the weekly administration of intravenous therapy. Treatments that are equally or more effective and less toxic than traditional methods are needed, especially oral therapy. SCH 39304 is an orally active antifungal drug that in animal studies is active against a wide range of systemic fungal infections including infections due to Cryptococcus. Features of SCH 39304 suggest that it might be of value in the treatment of cryptococcal meningitis.

Clinical Trial Conditions for Amphotericin B

Condition Name

Condition Name for Amphotericin B
Intervention	Trials
HIV Infections	25
Cryptococcal Meningitis	18
Visceral Leishmaniasis	15

Condition MeSH

Condition MeSH for Amphotericin B
Intervention	Trials
Meningitis, Cryptococcal	32
Meningitis	30
Mycoses	28

Clinical Trial Locations for Amphotericin B

Trials by Country

Trials by Country for Amphotericin B
Location	Trials
United States	356
India	22
China	20
Brazil	15
Italy	15

Trials by US State

Trials by US State for Amphotericin B
Location	Trials
California	25
Texas	23
New York	23
Pennsylvania	21
Maryland	18

Clinical Trial Progress for Amphotericin B

Clinical Trial Phase

Clinical Trial Phase for Amphotericin B
Clinical Trial Phase	Trials
PHASE4	3
PHASE3	5
PHASE2	5

Clinical Trial Status

Clinical Trial Status for Amphotericin B
Clinical Trial Phase	Trials
Completed	113
Recruiting	16
Terminated	15

Clinical Trial Sponsors for Amphotericin B

Sponsor Name

Sponsor Name for Amphotericin B
Sponsor	Trials
National Institute of Allergy and Infectious Diseases (NIAID)	18
Pfizer	13
Gilead Sciences	11

Sponsor Type

Sponsor Type for Amphotericin B
Sponsor	Trials
Other	200
Industry	92
NIH	24

Last updated: April 28, 2026

Amphotericin B: Clinical Trials Update and Market Analysis With Projections

What does the current clinical-trials landscape show for Amphotericin B?

Amphotericin B remains clinically active across multiple program formats, but development is dominated by reformulations (liposomal, lipid complexes, and alternative delivery systems) rather than large new, first-in-class registrations. Public trial activity tracks those reformulations, including hospital-based and investigator-led studies focused on safety, comparative efficacy, and outcomes in invasive fungal disease, leishmaniasis, and other endemic mycoses.

Clinical-trials update (signal-level view)

• Active/ongoing studies: Contemporary activity is skewed to Amphotericin B lipid formulations (liposomal amphotericin B and amphotericin B lipid complex) and new delivery platforms aimed at reducing nephrotoxicity and improving dosing practicality.
• Trial endpoints: Common endpoints include incidence of nephrotoxicity, mycological response, mortality at end of therapy or follow-up, and infusion-related reactions.
• Geographic pattern: Higher visibility in regions with endemic fungal disease and high-leishmaniasis burden, where amphotericin B remains an anchor therapy.

Because “Amphotericin B” is a class name used across multiple branded products and reformulations, any market-facing update that treats “Amphotericin B” as one monolithic product can misstate both trial relevance and commercial exposure. Market projections should be anchored to formulation and label, not only the active ingredient.

What is the commercial market reality for Amphotericin B today?

Amphotericin B is a mature, established antifungal used primarily in serious invasive fungal infections and leishmaniasis. Commercial dynamics are shaped by:

1. Formulation substitutions (liposomal versions vs conventional amphotericin B),
2. Hospital formularies and guideline-aligned use in severe disease,
3. Competitor penetration from azoles, echinocandins, and newer antifungals in eligible indications,
4. Safety and administration requirements that favor reformulated amphotericin B in many settings.

Market segmentation that matters for projection

• By formulation
  • Liposomal amphotericin B: Higher adoption where nephrotoxicity risk is a central driver.
  • Amphotericin B lipid complex: Similar end-use positioning; utilization depends on procurement and local guideline patterns.
  • Conventional amphotericin B deoxycholate: Still used where cost and availability dominate, often in settings with stronger monitoring capacity for toxicity.
• By indication
  • Invasive aspergillosis and other invasive mycoses: Typically hospital-directed use in severe cases or when other agents are not suitable.
  • Mucormycosis: Amphotericin B formulations are frequently guideline-relevant in severe disease.
  • Leishmaniasis: Amphotericin B retains a defined role, especially where resistance, contraindications, or systemic options are limited.

Competitive context

• Competitive pressure is real where newer agents can displace amphotericin B in less severe disease or where safety allows outpatient or shorter-course alternatives.
• Displacement is less complete in severe invasive fungal disease and certain endemic indications where amphotericin B formulations remain standard-of-care, particularly when rapid fungicidal action and broad-spectrum coverage matter.

How should you project near- to mid-term demand for Amphotericin B formulations?

A projection should be anchored to (1) the disease incidence base and (2) formulation mix shifts driven by safety and guideline language. The dominant commercial lever is liposomal and lipid-complex share gain within hospitals and tenders, rather than growth in total treated cases alone.

Projection framework (formulation-mix driven)

Core demand drivers

• Higher-risk patient populations: oncology, transplant, and advanced immunosuppression.
• Ongoing incidence of invasive fungal disease and mucormycosis in clinical settings.
• Continued guideline support for amphotericin B formulations as a key option in severe infections and when alternatives fail or are unsuitable.
• Safety-driven procurement: nephrotoxicity reduction favors liposomal formats.

Downside factors

• Substitution by newer antifungals in some scenarios, especially for species and severity profiles where azoles/echinocandins show strong positioning.
• Budget pressure that can pull procurement toward conventional amphotericin B when monitoring is feasible.
• Supply chain and pricing dynamics typical of older injectables and their reformulated successors.

Quantitative projection (what can be stated from available information)

No defensible numeric market model or quantified clinical-to-commercial conversion can be produced from the information available in this prompt alone. A credible projection requires a dated baseline for:

• current global sales by amphotericin B formulation,
• treated incidence by indication,
• and country procurement mix.

Given the constraints, a numeric forecast would be speculative and cannot be produced.

What clinical trial evidence would most influence market share?

For investors and R&D leadership, the trial signals that matter most are those that affect formulary decisions:

1. Nephrotoxicity reduction vs comparator
   • Lower incidence, lower severity, and less need for dose interruption are direct drivers of procurement for liposomal and lipid-complex formats.
2. Efficacy in invasive fungal subtypes
   • Evidence in mucormycosis and other difficult-to-treat species profiles can sustain use despite competitive pressure.
3. Feasibility and dosing practicality
   • Shorter infusion times, reduced monitoring burden, and dosing flexibility can accelerate adoption in high-throughput hospital systems.
4. Special populations
   • Pediatric safety, renal impairment handling, and immunocompromised cohorts influence adoption across formularies.

Key commercial and clinical implications for R&D and investment screening

Product strategy implications

• Programs that reduce nephrotoxicity and maintain fungicidal efficacy are the most likely to win formulary traction.
• Trials should be designed to generate adoption-grade outcomes: toxicity, need for discontinuation, and mortality endpoints at defined time points.

Portfolio implications

• “Amphotericin B” should be treated as a formulation portfolio. Each format competes on safety, dosing, and procurement economics.
• Pipeline value is more sensitive to clinical differentiation than to incremental improvements that do not change hospital decision-making.

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Key Takeaways

• Amphotericin B development remains anchored in reformulations that reduce toxicity and improve administration.
• Market demand is driven more by hospital formulary and safety mix (liposomal/lipid-complex share) than by broad new indications.
• Trial signals that most affect market share are nephrotoxicity outcomes, efficacy in severe invasive fungal subtypes, and evidence in immunocompromised and pediatric cohorts.
• Any numeric market projection must be formulation- and indication-specific; a monolithic “Amphotericin B” forecast is not decision-grade.

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FAQs

1) Is Amphotericin B still used for invasive fungal infections in 2026?

Yes. Amphotericin B formulations remain a core option in severe invasive fungal disease contexts, especially where safety, broad coverage, and clinical severity align with guideline use.

2) Which factor most drives use of liposomal amphotericin B vs conventional amphotericin B?

Nephrotoxicity profile and the resulting ability to treat patients who are at higher renal risk with fewer treatment interruptions.

3) Do new antifungals reduce Amphotericin B demand?

They reduce share in selected settings, but amphotericin B formulations retain relevance in severe disease and certain endemic or difficult-to-treat infections where amphotericin B remains a key option.

4) What trial endpoints have the highest decision value for procurement?

Nephrotoxicity incidence and severity, mortality at defined follow-up points, mycological response, and infusion-related tolerability.

5) Should you model the market for “Amphotericin B” as one product?

No. Projection should be built by formulation (liposomal, lipid complex, conventional) and by indication because share and adoption differ materially.

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References

[1] FDA. Amphotericin B products and labeling (various formulations). https://www.fda.gov/ (accessed 2026-04-28)
[2] EMA. Amphotericin B product information and EPARs (various formulations). https://www.ema.europa.eu/ (accessed 2026-04-28)
[3] ClinicalTrials.gov. Amphotericin B studies (various formulations and indications). https://clinicaltrials.gov/ (accessed 2026-04-28)