NCT03583164

Brief Summary

A study to evaluate olorofim (F901318) for the treatment of invasive fungal infections in participants lacking suitable alternative treatment options.

Trial Health

98
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
203

participants targeted

Target at P75+ for phase_2

Timeline
Completed

Started Jun 2018

Longer than P75 for phase_2

Geographic Reach
17 countries

82 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

May 17, 2018

Completed
20 days until next milestone

Study Start

First participant enrolled

June 6, 2018

Completed
1 month until next milestone

First Posted

Study publicly available on registry

July 11, 2018

Completed
4.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 10, 2023

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

February 10, 2023

Completed
1.4 years until next milestone

Results Posted

Study results publicly available

July 10, 2024

Completed
Last Updated

July 10, 2024

Status Verified

July 1, 2024

Enrollment Period

4.7 years

First QC Date

May 17, 2018

Results QC Date

June 13, 2024

Last Update Submit

July 9, 2024

Conditions

Keywords

Anti fungalInvasive aspergillosisRare mouldsCoccidioidomycosis

Outcome Measures

Primary Outcomes (1)

  • Data Review Committee (DRC) Adjudicated Overall Response at Day 42

    The primary efficacy variable was the DRC-adjudicated overall response at the Day 42 Study Visit, as determined by an independent DRC using a combination of clinical, mycological, and radiological results based on 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. For the primary statistical analysis of overall response rate, values were assigned to the DRC adjudicated overall response as follows: Success (Success-Complete, Success-Partial); Failure (Failure-Stable, Failure-Progression, Death, and participants for whom data at the Day 42 Study Visit could not be collected or participants who were considered not evaluable at the Day 42 Study Visit).

    Day 42 in the Main Phase of study treatment

Secondary Outcomes (22)

  • DRC Adjudicated Overall Response at Day 42 for All Aspergillus

    Day 42 in the Main Phase of study treatment

  • DRC Adjudicated Overall Response at Day 42 for Lomentospora Prolificans

    Day 42 in the Main Phase of study treatment Day 42 in the Main Phase of study treatment Day 42 in the Main Phase of study treatment Day 42 in the Main Phase of study treatment Day 42 in the Main Phase of study treatment

  • DRC Adjudicated Overall Response at Day 42 for for Scedosporium Species

    Day 42 in the Main Phase of study treatment

  • DRC Adjudicated Overall Response at Day 42 for Coccidioides Species

    Day 42 in the Main Phase of study treatment

  • DRC Adjudicated Overall Response at Day 42 for Other Olorofim Susceptible Fungi

    Day 42 in the Main Phase of study treatment

  • +17 more secondary outcomes

Study Arms (1)

Olorofim (F901318)

EXPERIMENTAL

Olorofim (F901318) was given orally for up to 90 days in the Main Study Phase and could be continued for those participants entering the Extended Treatment Phase. Patients received fixed doses comprising of a 1-day loading dose of 150 mg of olorofim twice a day followed by a maintenance dose of 90 mg of olorofim twice a day. Up until Protocol Amendment 06, 58 patients received a weight-based olorofim dosing consisting of a 1-day loading dose of 4 mg/kg/day on Day 1, then a maintenance dose of 2.5 mg/kg/day (divided into 2 or 3 doses). The dose was then adjusted based on plasma levels of olorofim with the maximum total daily dose of 300 mg.

Drug: Olorofim

Interventions

30mg oral tablets

Also known as: F901318
Olorofim (F901318)

Eligibility Criteria

Age16 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Male and female aged at least 18 years, or male and female aged 16 years or 17 years and who weigh at least 40 kg whom have given informed consent
  • Ability and willingness to comply with the protocol.
  • Able to take oral medication
  • Females must be non-lactating and at no risk of pregnancy
  • Male with female partners of childbearing potential must either abstain from sexual intercourse or use a highly effective means of contraception
  • Participants with invasive fungal disease
  • Participants who have limited alternative treatment options

You may not qualify if:

  • Women who are pregnant or breastfeeding.
  • Known history of allergy, hypersensitivity, or any serious reaction to any component of the study drug.
  • Participants with chronic aspergillosis, aspergilloma or allergic bronchopulmonary aspergillosis.
  • Human Immunodeficiency Virus (HIV) infection but not currently receiving antiretroviral therapy.
  • Participants with a medical condition that may jeopardize adherence to the protocol or may cause unacceptable additional risk to the participant
  • Previously enrolled participants or participants enrolled in any clinical trial within the last 30 days
  • Participants receiving treatment limited to supportive care due to predicted short survival time.
  • Prohibited concomitant medications.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (82)

Valley Fever Institute at Kern Medical Center

Bakersfield, California, 93306, United States

Location

UC Davis Medical Center

Sacramento, California, 95817, United States

Location

University of California San Diego Medical Center

San Diego, California, 92103, United States

Location

Emory University

Atlanta, Georgia, 30322, United States

Location

University of Chicago

Chicago, Illinois, 60637, United States

Location

Johns Hopkins Hospital

Baltimore, Maryland, 21287, United States

Location

Brigham and Women's Hospital

Boston, Massachusetts, 02115, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

Weill Cornell Medical College

New York, New York, 10065, United States

Location

Stony Brook University Medical Center

Stony Brook, New York, 11794-0001, United States

Location

Duke University Health System

Durham, North Carolina, 27710, United States

Location

UPMC

Pittsburgh, Pennsylvania, 15213, United States

Location

University of Texas MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

The University of Texas Health Science Center at San Antonio

San Antonio, Texas, 78229, United States

Location

Westmead Hospital

Westmead, New South Wales, 2145, Australia

Location

Royal Brisbane and Women's Hospital

Herston, Queensland, 4029, Australia

Location

Peter MacCallum Centre-East Melbourne

Melbourne, Victoria, 3000, Australia

Location

Royal Melbourne Hospital

Melbourne, Victoria, 3000, Australia

Location

The Alfred Hospital

Melbourne, Victoria, 3004, Australia

Location

Fiona Stanley Hospital

Murdoch, Western Australia, 6150, Australia

Location

UZ Leuven

Leuven, Waals-Brabant, 3000, Belgium

Location

Institut Jules Bordet

Brussels, 1000, Belgium

Location

Hôpital Erasme

Brussels, 1070, Belgium

Location

Hospital Felício Rocho

Belo Horizonte, Minas Gerais, 30110-934, Brazil

Location

Santa Casa de Misericórdia de Belo Horizonte

Belo Horizonte, Minas Gerais, 30150-221, Brazil

Location

HC - UFPR - Hospital de Clínicas da Universidade Federal do Paraná

Curitiba, Paraná, 80060-900, Brazil

Location

Santa Casa de Misericórdia de Porto Alegre

Porto Alegre, Rio Grande do Sul, 90020-090, Brazil

Location

Hospital de Clínicas de Porto Alegre

Porto Alegre, Rio Grande do Sul, 90035-903, Brazil

Location

Hospital São Lucas da PUCRS

Porto Alegre, Rio Grande do Sul, 90610-000, Brazil

Location

Hospital da Universidade Federal de Santa Maria CEP/UFSM

Santa Maria, Rio Grande do Sul, 97105-900, Brazil

Location

Hospital Erasto Gaertner - Liga Paranaense de Combate ao Câncer

Curitiba, 81520-060, Brazil

Location

Santa Casa de Misericórdia de Passos

Passos, 37904-020, Brazil

Location

Oncology Center, Mansoura University

Al Mansurah, 35516, Egypt

Location

Alexandria University Hospital

Alexandria, 21131, Egypt

Location

Cairo University Hospitals

Cairo, 11559, Egypt

Location

Ain Shams University Hospital

Cairo, 11566, Egypt

Location

Air Force Specialized Hospital

Cairo, 11566, Egypt

Location

National Cancer Institute

Cairo, 11796, Egypt

Location

Nasser Institute

Cairo, 12655, Egypt

Location

CHU Strasbourg - Hôpital Hautepierre

Strasbourg, Bas Rhin, 67091, France

Location

CHU de Grenoble - Hôpital Albert Michallon

Grenoble, Isere, 38043, France

Location

Hôpital Necker - Enfants Malades

Paris, Paris, 75015, France

Location

Hôpital Saint-Louis

Paris, 75475, France

Location

Klinikum der Universitaet Muenchen Campus Grosshadern

Munich, Bavaria, 81377, Germany

Location

Universitaetsklinikum Koeln

Cologne, North Rhine-Westphalia, 50937, Germany

Location

Charite Universitaetsmedizin Berlin - Campus Benjamin Franklin

Berlin, 12200, Germany

Location

Charite-Campus Benjamin Franklin (CBF)

Berlin, 12200, Germany

Location

Soroka University Medical Center

Beersheba, 84001, Israel

Location

Rambam Health Care Campus

Haifa, 3109601, Israel

Location

Hadassah University Hospital - Ein Kerem

Jerusalem, 9112001, Israel

Location

Chaim Sheba Medical Center

Ramat Gan, 52363, Israel

Location

Tel Aviv Sourasky Medical Center

Tel Aviv, 6423906, Israel

Location

Radboudumc

Nijmegen, 6525 GA, Netherlands

Location

Erasmus Medisch Centrum

Rotterdam, 3015 CE, Netherlands

Location

UMC Utrecht

Utrecht, 3584 CX, Netherlands

Location

Uniwersyteckie Centrum Kliniczne

Gdansk, 80-214, Poland

Location

SPZOZ Szpital Uniwersytecki w Krakowie

Krakow, 31-501, Poland

Location

Wojewodzki Szpital Specjalistyczny im. J. Korczaka

Słupsk, 76-200, Poland

Location

Instytut Hematologii i Transfuzjologii

Warsaw, 02-776, Poland

Location

Leningrad Regional Clinical Hospital

Saint Petersburg, 194291, Russia

Location

FBI "Scientific Research Institute of Oncology n. a. N. N. Petrov"

Saint Petersburg, Russia

Location

Pavlov First Saint Petersburg State Medical University

Saint Petersburg, Russia

Location

SBEIHPE "NWSMU n. a. I.I Mechnikov" of MoH and SD of RH

Saint Petersburg, Russia

Location

Asan Medical Center

Seoul, 05505, South Korea

Location

Samsung Medical Center

Seoul, 06351, South Korea

Location

The Catholic University of Korea, Seoul St. Mary's Hospital

Seoul, 06591, South Korea

Location

Hospital Universitari Vall d'Hebron

Barcelona, 08035, Spain

Location

Hospital Universitario Ramon y Cajal

Madrid, 28034, Spain

Location

Hospital Universitario 12 de Octubre

Madrid, 28041, Spain

Location

Hospital Universitari i Politecnic La Fe

Valencia, 46021, Spain

Location

Siriraj Hospital

Bangkoknoi, Bangkok, 10700, Thailand

Location

King Chulalongkorn Memorial Hospital

Pathum Wan, Bangkok, 10330, Thailand

Location

Dicle University, Medical Faculty

Diyarbakır, 21280, Turkey (Türkiye)

Location

Acibadem Atakent Hospital

Istanbul, 34303, Turkey (Türkiye)

Location

Marmara University Pendik Research and Training Hospital

Istanbul, 34899, Turkey (Türkiye)

Location

King's College Hospital

London, Greater London, SE5 9NU, United Kingdom

Location

Manchester Royal Infirmary

Manchester, Greater Manchester, M13 9WL, United Kingdom

Location

Wythenshawe Hospital

Manchester, Wythenshawe, M23 9LT, United Kingdom

Location

Bach Mai Hospital

Hanoi, 100000, Vietnam

Location

National Lung Hospital

Hanoi, 10000, Vietnam

Location

HCMC Hospital for Tropical Diseases

Ho Chi Minh City, 00000, Vietnam

Location

Blood Transfusion Hematology Hospital

Ho Chi Minh City, 0000, Vietnam

Location

Related Publications (1)

  • Maertens JA, Thompson GR 3rd, Spec A, Donovan FM, Hammond SP, Bruns AHW, Rahav G, Shoham S, Johnson R, Rijnders B, Schaenman J, Hoenigl M, Morrissey CO, Mehta SR, Heath CH, Koehler P, Paterson DL, Slavin MA, Fortun J, Nguyen MH, Patterson TF, Uspenskaya O, Van de Veerdonk FL, Verweij PE, Aoun M, Georgala A, Alexander BD, Chayakulkeeree M, Mehra V, Miceli MH, Sikka MK, Sole A, Walsh TJ, Aguado JM, Holland SM, Moussa M, Rautemaa-Richardson R, Bazaz R, Schwartz S, Walsh SR, Plate M, Yehudai-Ofir D, Bruggemann RJ, Cornely OA, Ostrosky-Zeichner L, Vazquez JA, White PL, Cornelissen K, Ross GG, Fitton L, Dane A, Zinzi D, Rex JH, Chen SC. Olorofim for the treatment of invasive fungal diseases in patients with few or no therapeutic options: a single-arm, open-label, phase 2b study. Lancet Infect Dis. 2025 Nov;25(11):1177-1188. doi: 10.1016/S1473-3099(25)00224-5. Epub 2025 Jun 18.

MeSH Terms

Conditions

Invasive Fungal InfectionsCoccidioidomycosis

Interventions

olorofim

Condition Hierarchy (Ancestors)

MycosesBacterial Infections and MycosesInfections

Results Point of Contact

Title
Daniela Zinzi
Organization
F2G Ltd

Study Officials

  • Sharon Chen

    Westmead Hospital

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 17, 2018

First Posted

July 11, 2018

Study Start

June 6, 2018

Primary Completion

February 10, 2023

Study Completion

February 10, 2023

Last Updated

July 10, 2024

Results First Posted

July 10, 2024

Record last verified: 2024-07

Data Sharing

IPD Sharing
Will share

Individual participant data that underlie the results can be available to researchers after the primary publication of results for this study, after deidentification (text, tables, figures, appendices)

Shared Documents
STUDY PROTOCOL, SAP
Time Frame
From 3 months after publication of the primary manuscript (no end date)
Access Criteria
Requests should be directed to F2G for review.

Locations