Phase 2 EL219 versus Liposomal Amphotericin B for Early Antifungal Therapy

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Elion Therapeutics Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Bacterial Infections and Mycoses [C01]

Trial duration

8 May 2026 → ongoing

Decision date (initial)

2026-02-26

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Elion Therapeutics, Inc

External identifiers

EU CT number

2025-522835-32-00

WHO UTN

U1111-1324-9950

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy, Safety, Pharmacokinetic, Efficacy

Evaluate the efficacy and safety of EL219 compared to liposomal amphotericin B (LAmB) followed by voriconazole for early antifungal therapy (EAT) of suspected invasive mould infections (IMIs).

Secondary objectives 3

1. 1_Evaluate the efficacy of EL219 compared to LAmB followed by voriconazole for treatment of invasive aspergillosis (IA).
2. 2_Evaluate the incidence of invasive fungal infections (IFIs) in EL219 compared to LAmB followed by voriconazole after 14 days of EAT.
3. 3_Evaluate the duration of hospitalization and rehospitalization rates in EL219 compared to LAmB.

Conditions and MedDRA coding

Suspected Invasive Mould Infection

Regulatory references

Scientific advice from competent authorities

Food And Drug Administration

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. 1_Males or females 18 years and older
2. 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.
3. 3_Has suspected invasive mould infection (IMI) as defined in the protocol
4. 4_Must be willing to adhere to dosing, study visit schedule, and mandatory procedures as described in the protocol

Exclusion criteria 8

1. 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
2. 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
3. 3_Systemic bacterial infection diagnosed within the 14 days prior to randomization
4. 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
5. 5_Presence of 1 or more of the following concomitant diseases: -Known cirrhosis of the liver - Diagnosed symptomatic heart failure -Diagnosed reduced lung function
6. 6_Receiving either hemodialysis or peritoneal dialysis
7. 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)
8. 8_Prior recipient of orthotopic lung transplant

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. 1_All-cause mortality at Day 42 in the Intent-to-Treat (ITT) analysis set
2. 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

Secondary endpoints 5

1. 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)
2. 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)
3. 3_Breakthrough possible, probable, or proven IFI established after 14 days of study drug in the ITT analysis set
4. 4_Duration of the initial hospitalization after randomization, in the ITT analysis set
5. 5_Motif et durée de réhospitalisation après la sortie d’hôpital à l’issue de l’hospitalisation initiale, dans l’ensemble d’analyse ITT

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Comparator 3

Placebo 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Elion Therapeutics Inc.

Sponsor organisation

Elion Therapeutics Inc.

Address

1 Lincoln Street Suite 2900-121

City

Boston

Postcode

02111-2904

Country

United States

Scientific contact point

Organisation

Elion Therapeutics Inc.

Contact name

Taylor Sandison

Public contact point

Organisation

Elion Therapeutics Inc.

Contact name

Laura Navalta

Third parties 7

Locations

4 EU/EEA countries · 13 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 41 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

2 submissions — initial application plus substantial / non-substantial modifications