Study of Rezafungin Compared to Standard Antimicrobial Regimen for Prevention of Invasive Fungal Diseases in Adults Undergoing Allogeneic Blood and Marrow Transplantation
NCT ID: NCT04368559
Last Updated: 2026-02-11
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
ACTIVE_NOT_RECRUITING
PHASE3
602 participants
INTERVENTIONAL
2020-05-11
2026-02-28
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
Study of Rezafungin Compared to Caspofungin in Subjects With Candidemia and/or Invasive Candidiasis
NCT03667690
Rezafungin Paediatric PK Study in Paediatric Subjects From Birth to <18 Years of Age
NCT05534529
Comparing the Effectiveness of Fluconazole and a New Medicine (FK463) in Preventing Fungal Infections in Bone Marrow Transplant Patients
NCT00001937
Safety and Pharmacokinetics of Rezafungin
NCT04117607
A Study to Compare Efficacy and Safety of Mycamine® and Itraconazole for Preventing Fungal Infections
NCT00794703
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
RANDOMIZED
PARALLEL
PREVENTION
QUADRUPLE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Group 1: Rezafungin for Injection
Subjects in Rezafungin treatment group will receive a 400 mg loading dose in Week 1, followed by 200 mg once weekly, for a total of 13 weeks. Subjects will receive oral placebo for standard antimicrobial regimen (SAR) azole prophylaxis and oral placebo for SAR anti-Pneumocystis pneumonia (PCP) prophylaxis in accordance with the respective SAR dosing regimens for each. For subjects who are switched to a SAR IV regimen, oral placebo for SAR azole prophylaxis will be changed to IV placebo. There is no IV option for SAR anti-PCP prophylaxis.
Rezafungin for Injection
Intravenous antifungal therapy
Intravenous Placebo
Normal saline
Oral Placebo
Microcrystalline cellulose
Group 2: Oral Antifungal
Subjects randomized to the SAR will receive either fluconazole or posaconazole as the first-line SAR as per site's standard practice. Fluconazole will be administered orally at once daily doses of 400 mg for 13 weeks. Posaconazole will be administered orally as 300 mg twice daily on the first day and 300 mg once daily thereafter for 13 weeks. Azole-based antifungal therapy (fluconazole or posaconazole) can be switched from oral therapy to IV therapy if there is oral intolerance, at the discretion of the Investigator.
Subjects who started on fluconazole SAR may be switched to posaconazole at the discretion of the Investigator if they develop acute clinically significant GVHD; In addition, subjects in the SAR group will receive anti PCP prophylaxis with oral TMP/SMX (80 mg TMP/ 400 mg SMX) once daily. There is no IV option for SAR anti-PCP prophylaxis.
Posaconazole
Oral antifungal therapy
Fluconazole
Oral antifungal therapy
Trimethoprim-sulfamethoxazole (TMP/SMX)
Oral antibacterial therapy
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
Rezafungin for Injection
Intravenous antifungal therapy
Posaconazole
Oral antifungal therapy
Fluconazole
Oral antifungal therapy
Trimethoprim-sulfamethoxazole (TMP/SMX)
Oral antibacterial therapy
Intravenous Placebo
Normal saline
Oral Placebo
Microcrystalline cellulose
Other Intervention Names
Discover alternative or legacy names that may be used to describe the listed interventions across different sources.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
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
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.
18 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Mundipharma Research Limited
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Laura Cox, PhD
Role: STUDY_DIRECTOR
Mundipharma Research Limited
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
University of Alabama at Birmingham
Birmingham, Alabama, United States
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 Hospital of Limoges
Limoges, , France
University Hospital of Nantes
Nantes, , France
Hospital Saint Antoine Ap-Hp
Paris, , France
University Hospital of Bordeaux
Pessac, , France
Lyon-Sud Hospital Center
Pierre-Bénite, , France
University Hospital of Cologne
Cologne, , Germany
University Hospital Carl Gustav Carus Dresden
Dresden, , Germany
Johannes Gutenberg University Medical Center
Mainz, , Germany
University Hospital Münster
Münster, , Germany
University Hospital Wurzburg UKW
Würzburg, , Germany
San Martino Polyclinic Hospital
Genova, , Italy
IEO Istituto Europeo di Oncologia
Milan, , Italy
Agostino Gemelli University Policlinic
Rome, , Italy
Humanitas Cancer Center
Rozzano, , Italy
University Hospital Vall d'Hebron
Barcelona, , Spain
Hospital Clinic of Barcelona
Barcelona, , Spain
University Hospital Ramon y Cajal
Madrid, , Spain
Puerta de Hierro Majadahonda University Hospital
Majadahonda, , Spain
University Hospital of Salamanca
Salamanca, , Spain
University Hospital Marques de Valdecilla
Santander, , Spain
University Hospital of Valencia
Valencia, , Spain
La Fe University and Polytechnic Hospital
Valencia, , Spain
University Hospitals Geneva
Geneva, , Switzerland
Addenbrookes Hospital
Cambridge, , United Kingdom
University Hospital of Wales
Cardiff, , United Kingdom
Kings College Hospital NHS Foundation Trust
London, , United Kingdom
St. George's University Hospitals NHS Foundation Trust
London, , United Kingdom
The Royal Marsden Nhs Foundation Trust
London, , United Kingdom
Countries
Review the countries where the study has at least one active or historical site.
References
Explore related publications, articles, or registry entries linked to this study.
Cross SJ, Wolf J, Patel PA. Prevention, Diagnosis and Management of Pneumocystis jirovecii Infection in Children With Cancer or Receiving Hematopoietic Cell Therapy. Pediatr Infect Dis J. 2023 Dec 1;42(12):e479-e482. doi: 10.1097/INF.0000000000004102. Epub 2023 Sep 21. No abstract available.
Ham YY, Lewis JS 2nd, Thompson GR 3rd. Rezafungin: a novel antifungal for the treatment of invasive candidiasis. Future Microbiol. 2021 Jan;16(1):27-36. doi: 10.2217/fmb-2020-0217.
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
2017-004981-85
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
CD101.IV.3.08
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.