New drug could shield transplant patients from deadly fungal infections

NCT ID NCT04368559

Prevention Sponsor: Mundipharma Research Limited Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested a new medicine called rezafungin to see if it can prevent serious fungal infections in adults getting a bone marrow transplant. About 600 participants received either rezafungin or the standard treatment. The goal was to see if rezafungin helps more people stay infection-free and survive.

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Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

602 people

The number who actually took part.

Started

May 2020

Finished

Jan 2026

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Willing and able to provide written informed consent. 2. Males or females ≥18 years of age. 3. 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. 4. Diagnosed with 1 of the following underlying diseases: 1. Acute myeloid leukemia (AML), with or without a history of myelodysplastic syndrome, in first or second complete remission. 2. Acute lymphoblastic leukemia, in first or second complete remission. 3. Acute undifferentiated leukemia in first or second remission. 4. Acute biphenotypic leukemia in first or second complete remission. 5. Chronic myelogenous leukemia in either chronic or accelerated phase. 6. 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. n. Myeloproliferative disorder, unclassified. o. Multiple myeloma (plasma cell myeloma). 5. Receiving myeloablative or reduced-intensity conditioning regimens. 6. Adequate renal and hepatic function prior to initiation of conditioning regimen, therefore between 40 days prior and 10 days prior to BMT, documented as follows: 1. Hepatic: alanine aminotransferase less than or equal to (≤) 2.5 × upper limit of normal (ULN) and total serum bilirubin ≤1.5 × ULN (excluding Gilbert's Syndrome). 2. Renal: serum creatinine ≤2 milligrams (mg)/deciliter (dL) and with creatinine clearance (CrCl) greater than or equal to (≥) 30 milliliters (mL)/minute (min) without a history of renal transplant, or undergoing weekly dialysis within 4 weeks of the BMT. 7. Baseline blood samples drawn for Platelia galactomannan enzyme immunoassay (GM EIA) and β-D glucan levels within 15 days before randomization, with results available prior to randomization. 8. Baseline Toxoplasma serologies available within 6 weeks prior to randomization. Subjects with a positive toxoplasma IgG serology at any time prior to randomization do not need to repeat the toxoplasma serologies (IgG and IgM) and will be considered to have a prior history of toxoplasmosis. 9. Baseline glucose-6-phosphate dehydrogenase (G6PD) deficiency determination by the investigator prior to randomization with no known evidence of G6PD deficiency performed any time prior to randomization. If the Investigator assesses the subject as G6PD sufficient, the G6PD test result does not need to be entered into the EDC system. 10. Female subjects of child-bearing potential \<2 years post-menopausal (unless surgically sterile) must agree to and comply with using 1 barrier method (e.g., female condom with spermicide) plus one other highly effective method of birth control (e.g., oral contraceptive, implant, injectable, indwelling intrauterine device, vasectomized partner), or sexual abstinence (only possible if it corresponds to the subject's usual lifestyle) while participating in this study, and for 30 days after the last dose of study drug. Male subjects must be vasectomized, abstain from sexual intercourse, or agree to use barrier contraception (condom with spermicide), and agree not to donate sperm while participating in the study and for 120 days from the last IV dose of study drug. Exclusion Criteria: 1. Diagnosis of AML not in morphological remission. 2. Diagnosis of chemotherapy-resistant lymphoma: a first relapse can occur provided that a second complete remission has occurred. 3. Suspected or diagnosed invasive fungal disease (IFD) within 4 weeks of randomisation. 4. Diagnosed symptomatic heart failure with left ventricular ejection fraction (LVEF) at rest ≤50%, or shortening fraction ≤26%. 5. 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. 6. 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. 7. Suspected or documented PCP within 2 years of screening. 8. 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. 9. Receipt of previous allogeneic BMT. 10. Planned receipt of cord blood for transplantation. 11. Planned peripheral blood or marrow autograft. 12. Not applicable to protocol Amendment 6. 13. 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. 14. 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). 15. . . 1. 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. 2. 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. 16. Known hypersensitivity to Rezafungin for Injection, any echinocandin, fluconazole, posaconazole, other azole antifungal, or to any of their excipients. 17. 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. 18. 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. 19. Known infection with HIV. Subjects with unknown HIV status should be tested for HIV antibodies per standard of care. 20. Pregnant or lactating females. 21. The Principal Investigator (PI) determines that the subject should not participate in the study. 22. Considered unlikely to follow up for 90 days after receipt of the BMT due to logistic concerns (i.e., location relative to transplant center). 23. Known liver cirrhosis, diagnosed according to country or Medical Society specific guidelines and documented in the medical records prior to initiating conditioning regimen.

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Conditions

The condition(s) this trial relates to.

candidemia candidiasis candidiasis, invasive disseminated candidiasis fungal infectious disease Fungemia Infections Inflammation Invasive Fungal Infections Pathologic Processes pneumocystosis Sepsis Systemic Inflammatory Response Syndrome systemic mycosis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Aspergillus Candidemia Fungal infection Fungemia Invasive candidiasis Invasive fungal disease Mold infection Mycoses Pneumocystis Prophylaxis of invasive fungal infections

Contacts and locations

Locations

AZ Sint-Jan

Bruges, West Vlaanderen, 8000, Belgium
Addenbrookes Hospital

Cambridge, cb2 0QQ, United Kingdom
Agostino Gemelli University Policlinic

Rome, 00168, Italy
Augusta University Medical Center

Augusta, Georgia, 30912, United States
Fred Hutchinson Cancer Center

Seattle, Washington, 98108, United States
Grenoble Alpes University Hospital Center

Grenoble, 38043, France
Hackensack University Medical Center

Hackensack, New Jersey, 07601, United States
Hamilton Health Sciences' Juravinski Hospital

Hamilton, L8V1C3, Canada
Henri Mondor Hospital

Créteil, 94000, France
Hospital Clinic of Barcelona

Barcelona, 08036, Spain
Hospital Saint Antoine Ap-Hp

Paris, 75012, France
Humanitas Cancer Center

Rozzano, 20089, Italy
IEO Istituto Europeo di Oncologia

Milan, 20141, Italy
Jean Minjoz Hospital

Besançon, 25030, France
Johannes Gutenberg University Medical Center

Mainz, 55131, Germany
John Hopkins

Baltimore, Maryland, 21218, United States
Kings College Hospital NHS Foundation Trust

London, SE5 9RS, United Kingdom
La Fe University and Polytechnic Hospital

Valencia, 46026, Spain
Lyon-Sud Hospital Center

Pierre-Bénite, 69495, France
Mary Hitchcock Memorial Hospital Dartmouth-Hitchcock

Lebanon, New Hampshire, 03756, United States
Massachusetts General Hospital

Boston, Massachusetts, 02114, United States
Mayo Clinic

Rochester, Minnesota, 55905, United States
McGill University Health Center

Montreal, H4A3J1, Canada
Puerta de Hierro Majadahonda University Hospital

Majadahonda, 28220, Spain
Rush University Medical Center

Chicago, Illinois, 60612, United States
San Martino Polyclinic Hospital

Genova, 16132, Italy
St. George's University Hospitals NHS Foundation Trust

London, SW17 0QT, United Kingdom
Stanford University School of Medicine

Stanford, California, 94304, United States
Stony Brook University Hospital

Stony Brook, New York, 11794, United States
The Royal Marsden Nhs Foundation Trust

London, SW3 6JJ, United Kingdom
The University of Oklahoma College of Medicine

Oklahoma City, Oklahoma, 73104, United States
UCLA Center for Health Sciences

Los Angeles, California, 90095, United States
University Hospital Carl Gustav Carus Dresden

Dresden, 01307, Germany
University Hospital Marques de Valdecilla

Santander, 39008, Spain
University Hospital Münster

Münster, 48149, Germany
University Hospital Ramon y Cajal

Madrid, 28034, Spain
University Hospital Vall d'Hebron

Barcelona, 08035, Spain
University Hospital Wurzburg UKW

Würzburg, 97080, Germany
University Hospital of Bordeaux

Pessac, 33604, France
University Hospital of Cologne

Cologne, 50937, Germany
University Hospital of Limoges

Limoges, 87042, France
University Hospital of Nantes

Nantes, 44093, France
University Hospital of Salamanca

Salamanca, 37007, Spain
University Hospital of Valencia

Valencia, 46010, Spain
University Hospital of Wales

Cardiff, CF144XW, United Kingdom
University Hospitals Geneva

Geneva, 1211, Switzerland
University Hospitals Leuven, Campus Gasthuisberg - UZ Leuven

Leuven, 3000, Belgium
University of Alabama at Birmingham

Birmingham, Alabama, 35233, United States
University of Chicago

Chicago, Illinois, 60637, United States
University of Maryland Medical Center

Baltimore, Maryland, 21201, United States
University of Minnesota Physicians

Minneapolis, Minnesota, 55455, United States
University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, 15213, United States
VCU Medical Center Main Hospital

Richmond, Virginia, 23219, United States

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