Study of Rezafungin Compared to Standard Antimicrobial Regimen for Prevention of Invasive Fungal Diseases in Adults Undergoing Allogeneic Blood and Marrow Transplantation

Completed · Phase 3 · Has a placebo group

Conditions studied: Candidemia, Mycoses, Fungal Infection, Fungemia, Invasive Candidiasis, Pneumocystis, Mold Infection, Invasive Fungal Disease, Prophylaxis of Invasive Fungal Infections, Aspergillus

In brief

The purpose of this pivotal study is to determine if intravenous Rezafungin is efficacious and safe in the prevention of invasive fungal diseases when compared to the standard antimicrobial regimen.

Key facts

Study ID

NCT04368559

Run by

Mundipharma Research Limited

People needed

602

Starts

2020-05-11

Expected to finish

2026-01-29

Last updated by the study team

2026-04-13

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Willing and able to provide written informed consent.
• Males or females ≥18 years of age.
• Receiving a human leukocyte antigen (HLA) matched allogeneic peripheral BMT from a family or unrelated donor, HLA-mismatched related or unrelated donor, or haploidentical donor.
• Diagnosed with 1 of the following underlying diseases:
• Acute myeloid leukemia (AML), with or without a history of myelodysplastic syndrome, in first or second complete remission.
• Acute lymphoblastic leukemia, in first or second complete remission.
• Acute undifferentiated leukemia in first or second remission.
• Acute biphenotypic leukemia in first or second complete remission.
• Chronic myelogenous leukemia in either chronic or accelerated phase.
• One of the following myelodysplastic syndrome(s) defined by the following:
• i. Refractory anemia.
• ii. Refractory anemia with ringed sideroblasts.
• iii. Refractory cytopenia with multilineage dysplasia.
• iv. Refractory cytopenia with multilineage dysplasia and ringed sideroblasts.
• v. Refractory anemia with excess blasts - 1 (5-10% blasts).
• vi. Refractory anemia with excess blasts - 2 (10-20% blasts).
• vii. Myelodysplastic syndrome, unclassified.
• viii. Myelodysplastic syndrome associated with isolated del (5q).
• g. Lymphoma (including Hodgkin's) with chemosensitive disease (i.e., response to chemotherapy) and receiving a related or unrelated donor transplant.
• h. Aplastic anemia.
• i. Primary or secondary myelofibrosis.
• j. Chronic myelomonocytic leukemia.
• k. Chronic lymphocytic leukemia.
• l. Drepanocytosis (sickle cell anemia).
• m. Red blood cell aplasia.

You may not qualify if…

• Diagnosis of AML not in morphological remission.
• Diagnosis of chemotherapy-resistant lymphoma: a first relapse can occur provided that a second complete remission has occurred.
• Suspected or diagnosed invasive fungal disease (IFD) within 4 weeks of randomisation.
• Diagnosed symptomatic heart failure with left ventricular ejection fraction (LVEF) at rest ≤50%, or shortening fraction ≤26%.
• Personal or family history of Long QT interval on electrocardiogram (ECG) (QT) syndrome or a prolonged QT interval corrected for heart rate by Fridericia's formula (QTcF) (>470 milliseconds [msec] in males and >480 msec in females); or concurrent administration of terfenadine, cisapride, astemizole, erythromycin, pimozide, quinidine, or halofantrine.
• Diagnosed reduced lung function with either diffusion capacity (corrected for hemoglobin) or forced expiratory volume in 1 second (FEV1) ≤65% of predicted value, or O2 saturation ≤82% on room air.
• Suspected or documented PCP within 2 years of screening.
• Positive baseline serum Platelia GM EIA (≥ 0.5) and/or β-D glucan assay (Fungitell ≥80 picograms [pg]/mL or Fujifilm Wako >11 pg/mL) within 15 days prior to the transplant.
• Receipt of previous allogeneic BMT.
• Planned receipt of cord blood for transplantation.
• Planned peripheral blood or marrow autograft.
• Not applicable to protocol Amendment 6.
• Grade 2 or higher ataxia, tremor, motor neuropathy, or sensory neuropathy, per National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
• History of severe (Grade ≥3) ataxia, neuropathy or tremors; or a diagnosis of multiple sclerosis or a movement disorder (including Parkinson's disease or Huntington's disease).
• . .
• Planned or ongoing intake at screening of a known severe neurotoxic medication or with a known moderate neurotoxic medication in a patient with ataxia, tremor, motor neuropathy, or sensory neuropathy of CTCAE version 5.0 Grade 1 or higher.
• Any contraindication or a medication or supplement known to severely interact with the standard antimicrobial regimen (SAR) as detailed in the US Prescribing Information (USPI) or Summary of Product Characteristics (SmPC) of fluconazole, posaconazole, or TMP/SMX.
• Known hypersensitivity to Rezafungin for Injection, any echinocandin, fluconazole, posaconazole, other azole antifungal, or to any of their excipients.
• Known hypersensitivity or inability to receive TMP/SMX or any of its excipients, including but not limited to anaphylaxis, exfoliative skin disorders, or acute porphyria.
• Recent use of an investigational medicinal product within 28 days or 5 half-lives of the investigational medicinal product, whichever is greater, to prevent overlapping toxicities when this study's investigational product is dosed, or presence of an investigational device at the time of screening. In some cases, use of investigational products may be acceptable in consultation with the Sponsor's Medical Monitor.
• Known infection with HIV. Subjects with unknown HIV status should be tested for HIV antibodies per standard of care.
• Pregnant or lactating females.
• The Principal Investigator (PI) determines that the subject should not participate in the study.
• Considered unlikely to follow up for 90 days after receipt of the BMT due to logistic concerns (i.e., location relative to transplant center).
• Known liver cirrhosis, diagnosed according to country or Medical Society specific guidelines and documented in the medical records prior to initiating conditioning regimen.

Where it is running

• UCLA Center for Health Sciences — Los Angeles, California, United States
• Stanford University School of Medicine — Stanford, California, United States
• Augusta University Medical Center — Augusta, Georgia, United States
• Rush University Medical Center — Chicago, Illinois, United States
• University of Chicago — Chicago, Illinois, United States
• University of Maryland Medical Center — Baltimore, Maryland, United States
• John Hopkins — Baltimore, Maryland, United States
• Massachusetts General Hospital — Boston, Massachusetts, United States
• University of Minnesota Physicians — Minneapolis, Minnesota, United States
• Mayo Clinic — Rochester, Minnesota, United States
• Mary Hitchcock Memorial Hospital Dartmouth-Hitchcock — Lebanon, New Hampshire, United States
• Hackensack University Medical Center — Hackensack, New Jersey, United States
• Stony Brook University Hospital — Stony Brook, New York, United States
• The University of Oklahoma College of Medicine — Oklahoma City, Oklahoma, United States
• University of Pittsburgh Medical Center — Pittsburgh, Pennsylvania, United States
• VCU Medical Center Main Hospital — Richmond, Virginia, United States
• Fred Hutchinson Cancer Center — Seattle, Washington, United States
• AZ Sint-Jan — Bruges, West Vlaanderen, Belgium
• University Hospitals Leuven, Campus Gasthuisberg - UZ Leuven — Leuven, Belgium
• Hamilton Health Sciences' Juravinski Hospital — Hamilton, Canada
• McGill University Health Center — Montreal, Canada
• Jean Minjoz Hospital — Besançon, France
• Henri Mondor Hospital — Créteil, France
• Grenoble Alpes University Hospital Center — Grenoble, France
• University of Alabama at Birmingham — Birmingham, Alabama, United States

Full record on ClinicalTrials.gov

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