In short
Clinical trials investigating Amphotericin B are studying how it is used in different patient groups, including people with fungal infections, cancer, kidney transplant recipients, and patients with inflammatory bowel disease. These studies look at safety, effectiveness, drug delivery, and outcomes such as infection control or lesion size.
Key points
- Clinical trials of Amphotericin B are being done in several different patient groups, not only in fungal infections but also in surgery, transplant care, and immune-related research. The studies include Phase 1, Phase 2, Phase 3, and low-intervention designs. Some trials test inhaled or intrabronchial use for lung fungal disease, while others study oral use to prevent infection after esophagectomy. Main outcomes include lung drug deposition, pneumonia, lesion size, mortality, immune cell energy metabolism, transplant rejection, and MRD negativity in leukemia.
Overview of the trials
The trial data show that Amphotericin B is being studied in several different clinical settings, mostly around fungal disease and infection prevention. The studies also include research in inflammatory bowel disease, kidney transplantation, and leukemia, where Amphotericin B appears as part of a broader treatment plan or study list.
Conditions and patient groups
The trials include healthy adults with no history of gastrointestinal disease and patients with inflammatory bowel disease (IBD) in remission. IBD in remission means the disease is not active at the time of the study.
Other studies focus on people with esophageal cancer undergoing surgery, patients with chronic pulmonary aspergillosis, and patients with simple aspergilloma. One large study also includes adults with invasive mold infections caused by several fungi, including Aspergillus spp., Fusarium spp., Lomentospora prolificans, Mucorales fungi, and other multidrug resistant molds.
Two additional trials involve kidney transplant recipients and adults with very high-risk T-lineage acute lymphoblastic leukemia (ALL). In these studies, Amphotericin B is listed among several other treatments being used in the overall care plan.
Study designs and phases
The trials cover a wide range of research stages, from Phase 1 to Phase 3, plus a low-intervention study. Phase 1 studies usually focus on early safety and how a treatment behaves in the body, while Phase 2 and Phase 3 studies look more closely at possible benefit in larger groups.
The NIMBUS study is a Phase 1 trial with 18 participants and uses inhaled liposomal Amphotericin B to study lung distribution. The simple aspergilloma study is a Phase 2, single-center, prospective, nonrandomized, open-label study with 20 participants. The invasive mold infection study is Phase 3 with 210 participants, and the esophagectomy prevention study is a low-intervention trial with 853 participants.
Other studies include a Phase 2 trial in IBD and healthy volunteers with 45 participants, a Phase 1 kidney transplant study with 27 participants, and a Phase 2 leukemia study with 31 participants.
Main outcomes and endpoints
The main outcome in the NIMBUS study is the amount of AmBisome deposited in the lungs, measured by SPECT/CT, which is an imaging test that shows where the treatment goes in the body. The study also compares lung levels with blood concentrations and looks for the best nebulised dose based on pulmonary deposition and pharmacokinetics.
In the simple aspergilloma study, the primary outcome is the relative change in lesion size from before treatment to after treatment. This helps show whether the treatment may reduce the fungal mass in the lung.
The Phase 3 invasive mold infection study measures all-cause mortality at Day 42, meaning death from any cause within that time point. The esophagectomy study measures the cumulative incidence of postoperative pneumonia within 30 days after surgery.
The IBD study measures energy metabolism of major immune cell types in blood, using single-cell energetic metabolism profiling after translation inhibition. The kidney transplant study measures safety outcomes such as acute toxicity, over-suppression of the immune system, chronic toxicity, and biopsy-confirmed acute rejection within 60 weeks. The leukemia study measures MRD negativity after induction, which means no minimal residual disease is detected after the first treatment cycle.
Key trial details
NIMBUS study: This Phase 1 trial studies inhaled liposomal Amphotericin B in adults with chronic pulmonary aspergillosis and uses Technetium-99m labelled AmBisome to help measure where the drug goes in the lungs. The goal is to understand lung deposition and support treatment design.
Simple aspergilloma trial: This Phase 2 study is open-label and nonrandomized, and it tests intrabronchial instillation of liposomal Amphotericin B in patients with simple aspergilloma. “Open-label” means both the researchers and the participants know what treatment is given.
Esophagectomy prevention trial: This low-intervention randomized multicenter study looks at selective decontamination of the digestive tract to prevent severe infectious complications after surgery for primary resectable esophageal carcinoma. Amphotericin B is one of the oral study drugs used in this prevention approach.
Invasive mold infection trial: This Phase 3 study compares fosmanogepix with several other treatment options, including Amphotericin B, in adults with invasive mold infections caused by difficult-to-treat fungi. The study is focused on survival at Day 42.
IBD and healthy volunteer study: This Phase 2 study examines how the gut microbiota, and changes in it, affect energy metabolism in blood immune cells. Amphotericin B is listed among the oral interventions in this study.
Kidney transplant study: This Phase 1 trial tests regulatory T cells in kidney transplant recipients and includes Ampho-Moronal among the listed oral medicines. The study focuses on safety, immune effects, and whether the transplant is rejected.
Leukemia study: This Phase 2 study adds daratumumab to the national treatment program for very high-risk T-cell ALL and includes Amphotericin B among multiple supportive medicines in the treatment list. The main endpoint is MRD negativity after induction therapy.
What these studies mean for patients
For patients, these trials show that Amphotericin B is being studied in different ways: as an inhaled treatment, an intrabronchial treatment, an oral prevention drug, and as part of broader treatment plans.
Some studies are trying to improve how much of the drug reaches the lungs, while others are trying to prevent infection after surgery or measure whether a treatment plan leads to better clinical results.
Because the trials involve different diseases and different study methods, the expected benefit and the type of patient who may join are not the same from one trial to another.