N38-(1,3-DIHYDROXYPROPAN-2-YL)-2'-EPI-AMPHOTERICIN B-38-AMIDE for Invasive mould infection

Inclusion criteria

• {"criterion_text":"- 1_Males or females 18 years and older"}
• {"criterion_text":"- 2_Présenter un risque d’infections fongiques invasives (IFI), au motif d’au moins 1 des éléments suivants : -Avoir reçu une greffe de moelle osseuse d’un donneur allogénique, avec du sang, de la moelle osseuse ou du sang de cordon comme source de cellules souches. -Recevoir actuellement ou avoir reçu récemment (dans un délai de 1 mois) des traitement(s) cytotoxique(s), biologique(s) ou immunomodulateur(s) pour traiter une tumeur maligne hématologique. -Avoir reçu des corticoïdes à des doses minimales moyennes de 0,3 mg/kg/jour d’équivalent prednisone pendant plus de 3 semaines. -Avoir reçu d’autres immunosuppresseurs reconnus agissant sur les lymphocytes T, tels que la cyclosporine, des inhibiteurs du facteur de nécrose tumorale alpha (TNF-α) ou des anticorps monoclonaux spécifiques au cours des 3 derniers mois."}
• {"criterion_text":"- 3_Has suspected invasive mould infection (IMI) as defined in the protocol"}
• {"criterion_text":"- 4_Must be willing to adhere to dosing, study visit schedule, and mandatory procedures as described in the protocol"}

Exclusion criteria

• {"criterion_text":"- 1_Diagnosis of proven or probable IMI within 1 month prior to randomization (including meeting the criteria for proven or probable IMI during the screening period), or relapsed/recurrent IMI which has not responded to other antifungal therapies"}
• {"criterion_text":"- 2_Prior antifungal treatment (azole prophylaxis permitted) for >96 hours prior to randomization or would require use of non-study antifungals during the period of the study"}
• {"criterion_text":"- 3_Systemic bacterial infection diagnosed within the 14 days prior to randomization"}
• {"criterion_text":"- 4_Presence of 1 or more of the following laboratory abnormalities: -Alanine aminotransferase (ALT) ≥5 × upper limit of normal (ULN). -Total serum bilirubin ≥5 × ULN (excluding Gilbert’s Syndrome).-Serum creatinine ≥2 mg/dL or creatinine clearance (CrCL) ≤30 mL/minute"}
• {"criterion_text":"- 5_Presence of 1 or more of the following concomitant diseases: -Known cirrhosis of the liver - Diagnosed symptomatic heart failure -Diagnosed reduced lung function"}
• {"criterion_text":"- 6_Receiving either hemodialysis or peritoneal dialysis"}
• {"criterion_text":"- 7_Personal or family history of long QT interval on ECG (QT) syndrome or a prolonged QT interval corrected for heart rate by Fridericia’s formula (QTcF; >470 msec in males and >480 msec in females)"}
• {"criterion_text":"- 8_Prior recipient of orthotopic lung transplant"}

Primary endpoints

• {"endpoint_text":"- 1_All-cause mortality at Day 42 in the Intent-to-Treat (ITT) analysis set","definition_or_measurement_approach":"All-cause mortality assessed at Day 42 in the ITT analysis set."}
• {"endpoint_text":"- 2_Serious adverse events (SAEs) and treatment emergent adverse events (TEAEs) categorized in a tiered approach in the Safety analysis set. Tier1 TEAEs include renal, electrolyte, hepatic, infusion-related reactions, photophobia, and photosensitivity. Tier 2 includes all other TEAEs","definition_or_measurement_approach":"SAEs and TEAEs recorded and categorized by tier in the Safety analysis set; Tier 1 includes renal, electrolyte, hepatic, infusion-related reactions, photophobia and photosensitivity; Tier 2 includes all other TEAEs."}

Secondary endpoints

• {"endpoint_text":"- 1_Overall success at Day 42, confirmed by the Data Review Committee (DRC), in the population with proven or probable IA (modified Intent-to-Treat [mITT]) as measured by: - Participant is alive, - favorable composite clinical, mycologic, and radiographic response (European Organization for Research and Treatment of Cancer/Invasive Fungal Infections Cooperative Group and the National Institute of Allergy and Infectious Diseases Mycoses Study Group [EORTC/MSG] criteria)","definition_or_measurement_approach":"Overall success at Day 42 in mITT for proven/probable IA: survival plus favourable composite clinical, mycologic and radiographic response per EORTC/MSG criteria; confirmation by Data Review Committee (DRC)."}
• {"endpoint_text":"- 2_Early antifungal therapy (EAT) success at Day 42 in the ITT analysis set defined by non-occurrence of the following: death, receipt of non-study drug systemic antifungal therapy for a cumulative exposure >10 days for progression of disease and/or toxicity, missing data (classified as indeterminate but analyzed as a failure)","definition_or_measurement_approach":"EAT success at Day 42 in ITT defined as absence of death, no receipt of non-study systemic antifungal therapy for >10 cumulative days for progression/toxicity, and no missing data (missing classified as failure)."}
• {"endpoint_text":"- 3_Breakthrough possible, probable, or proven IFI established after 14 days of study drug in the ITT analysis set","definition_or_measurement_approach":"Occurrence of breakthrough IFI (possible/probable/proven) after 14 days of study drug in the ITT analysis set."}
• {"endpoint_text":"- 4_Duration of the initial hospitalization after randomization, in the ITT analysis set","definition_or_measurement_approach":"Duration (days) of initial hospitalization after randomization measured in the ITT analysis set."}
• {"endpoint_text":"- 5_Motif et durée de réhospitalisation après la sortie d’hôpital à l’issue de l’hospitalisation initiale, dans l’ensemble d’analyse ITT","definition_or_measurement_approach":"Reason and duration of rehospitalization after discharge from initial hospitalization measured in the ITT analysis set."}

Spain

Earliest CTIS Part Ii Submission Date

29-01-2026

Latest Decision Or Authorization Date

22-04-2026

Processing Time Days

83

Number Of Sites

5

Number Of Participants

16

Italy

Earliest CTIS Part Ii Submission Date

02-02-2026

Latest Decision Or Authorization Date

22-04-2026

Processing Time Days

79

Number Of Sites

2

Number Of Participants

8

Belgium

Earliest CTIS Part Ii Submission Date

28-01-2026

Latest Decision Or Authorization Date

22-04-2026

Processing Time Days

84

Number Of Sites

4

Number Of Participants

20

France

Earliest CTIS Part Ii Submission Date

06-02-2026

Latest Decision Or Authorization Date

22-04-2026

Processing Time Days

75

Number Of Sites

2

Number Of Participants

10

Primary sponsor

Full Name

Elion Therapeutics Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

Medpace Finland Oy

Responsibilities

1,11,12,13,14,2,4,5,9

Name

Suvoda LLC

Responsibilities

3

Name

Propharma Group LLC

Responsibilities

8

Name

Jones Microbiology Institute Inc.

Responsibilities

4

Name

Sannova Analytical LLC

Responsibilities

4

Name

Mms Holdings Inc.

Responsibilities

10,6

Third parties

• {"country":"Finland","full_name":"Medpace Finland Oy","duties_or_roles":"1,11,12,13,14,2,4,5,9","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Suvoda LLC","duties_or_roles":"3","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Jones Microbiology Institute Inc.","duties_or_roles":"4","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Sannova Analytical LLC","duties_or_roles":"4","organisation_type":"Pharmaceutical company"}
• {"country":"Netherlands","full_name":"Radboud universitair medisch centrum Stichting","duties_or_roles":"4","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"United States","full_name":"Propharma Group LLC","duties_or_roles":"8","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Mms Holdings Inc.","duties_or_roles":"10,6","organisation_type":"Pharmaceutical company"}