Invasive Fungal InfectionsPhase 2FORMULA-OLSNCT03583164
Evaluate F901318 (Olorofim) Treatment of Invasive Fungal Infections in Participants Lacking Treatment Options
A Phase 2 study testing Olorofim in people with Invasive Fungal Infections, run by F2G Biotech GmbH at 82 sites in 17 countries.
Trial summary
A study to evaluate olorofim (F901318) for the treatment of invasive fungal infections in participants lacking suitable alternative treatment options. Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Invasive Fungal Infections
- What is being tested?
- Olorofim
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 203 participants
- Where
- 82 sitesin 17 countries
- Study dates
- 6 Jun 2018to 10 Feb 2023
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 9Show fewer
- Participants receiving treatment limited to supportive care due to predicted short survival time.
- Prohibited concomitant medications.
- Any exclusion criteria required by local regulatory authorities.
Trial locations and contacts
82 sites in 17 countries.
82 sites in 17 countries
United States14 sites
Emory University
Atlanta, Georgia, 30322
Valley Fever Institute at Kern Medical Center
Bakersfield, California, 93306
Johns Hopkins Hospital
Baltimore, Maryland, 21287
Brigham and Women's Hospital
Boston, Massachusetts, 02115
University of Chicago
Chicago, Illinois, 60637
Duke University Health System
Durham, North Carolina, 27710
University of Texas MD Anderson Cancer Center
Houston, Texas, 77030
Weill Cornell Medical College
New York, New York, 10065
UPMC
Pittsburgh, Pennsylvania, 15213
UC Davis Medical Center
Sacramento, California, 95817
The University of Texas Health Science Center at San Antonio
San Antonio, Texas, 78229
University of California San Diego Medical Center
San Diego, California, 92103
Washington University School of Medicine
St Louis, Missouri, 63110
Stony Brook University Medical Center
Stony Brook, New York, 11794-0001
Brazil9 sites
Hospital Felício Rocho
Belo Horizonte, Minas Gerais, 30110-934
Santa Casa de Misericórdia de Belo Horizonte
Belo Horizonte, Minas Gerais, 30150-221
HC - UFPR - Hospital de Clínicas da Universidade Federal do Paraná
Curitiba, Paraná, 80060-900
Hospital Erasto Gaertner - Liga Paranaense de Combate ao Câncer
Curitiba, 81520-060
Santa Casa de Misericórdia de Passos
Passos, 37904-020
Santa Casa de Misericórdia de Porto Alegre
Porto Alegre, Rio Grande do Sul, 90020-090
Hospital de Clínicas de Porto Alegre
Porto Alegre, Rio Grande do Sul, 90035-903
Hospital São Lucas da PUCRS
Porto Alegre, Rio Grande do Sul, 90610-000
Hospital da Universidade Federal de Santa Maria CEP/UFSM
Santa Maria, Rio Grande do Sul, 97105-900
Egypt7 sites
Oncology Center, Mansoura University
Al Mansurah, 35516
Alexandria University Hospital
Alexandria, 21131
Cairo University Hospitals
Cairo, 11559
Ain Shams University Hospital
Cairo, 11566
Air Force Specialized Hospital
Cairo, 11566
National Cancer Institute
Cairo, 11796
Nasser Institute
Cairo, 12655
Australia6 sites
Royal Brisbane and Women's Hospital
Herston, Queensland, 4029
Peter MacCallum Centre-East Melbourne
Melbourne, Victoria, 3000
Royal Melbourne Hospital
Melbourne, Victoria, 3000
The Alfred Hospital
Melbourne, Victoria, 3004
Fiona Stanley Hospital
Murdoch, Western Australia, 6150
Westmead Hospital
Westmead, New South Wales, 2145
Israel5 sites
Soroka University Medical Center
Beersheba, 84001
Rambam Health Care Campus
Haifa, 3109601
Hadassah University Hospital - Ein Kerem
Jerusalem, 9112001
Chaim Sheba Medical Center
Ramat Gan, 52363
Tel Aviv Sourasky Medical Center
Tel Aviv, 6423906
France4 sites
CHU de Grenoble - Hôpital Albert Michallon
Grenoble, Isere, 38043
Hôpital Necker - Enfants Malades
Paris, Paris, 75015
Hôpital Saint-Louis
Paris, 75475
CHU Strasbourg - Hôpital Hautepierre
Strasbourg, Bas Rhin, 67091
Germany4 sites
Charite Universitaetsmedizin Berlin - Campus Benjamin Franklin
Berlin, 12200
Charite-Campus Benjamin Franklin (CBF)
Berlin, 12200
Universitaetsklinikum Koeln
Cologne, North Rhine-Westphalia, 50937
Klinikum der Universitaet Muenchen Campus Grosshadern
Munich, Bavaria, 81377
Poland4 sites
Uniwersyteckie Centrum Kliniczne
Gdansk, 80-214
SPZOZ Szpital Uniwersytecki w Krakowie
Krakow, 31-501
Wojewodzki Szpital Specjalistyczny im. J. Korczaka
Słupsk, 76-200
Instytut Hematologii i Transfuzjologii
Warsaw, 02-776
Russia4 sites
Leningrad Regional Clinical Hospital
Saint Petersburg, 194291
FBI "Scientific Research Institute of Oncology n. a. N. N. Petrov"
Saint Petersburg
Pavlov First Saint Petersburg State Medical University
Saint Petersburg
SBEIHPE "NWSMU n. a. I.I Mechnikov" of MoH and SD of RH
Saint Petersburg
Spain4 sites
Hospital Universitari Vall d'Hebron
Barcelona, 08035
Hospital Universitario Ramon y Cajal
Madrid, 28034
Hospital Universitario 12 de Octubre
Madrid, 28041
Hospital Universitari i Politecnic La Fe
Valencia, 46021
Vietnam4 sites
Bach Mai Hospital
Hanoi, 100000
National Lung Hospital
Hanoi, 10000
HCMC Hospital for Tropical Diseases
Ho Chi Minh City, 00000
Blood Transfusion Hematology Hospital
Ho Chi Minh City, 0000
Belgium3 sites
Institut Jules Bordet
Brussels, 1000
Hôpital Erasme
Brussels, 1070
UZ Leuven
Leuven, Waals-Brabant, 3000
Netherlands3 sites
Radboudumc
Nijmegen, 6525 GA
Erasmus Medisch Centrum
Rotterdam, 3015 CE
UMC Utrecht
Utrecht, 3584 CX
South Korea3 sites
Asan Medical Center
Seoul, 05505
Samsung Medical Center
Seoul, 06351
The Catholic University of Korea, Seoul St. Mary's Hospital
Seoul, 06591
Turkey (Türkiye)3 sites
Dicle University, Medical Faculty
Diyarbakır, 21280
Acibadem Atakent Hospital
Istanbul, 34303
Marmara University Pendik Research and Training Hospital
Istanbul, 34899
United Kingdom3 sites
King's College Hospital
London, Greater London, SE5 9NU
Manchester Royal Infirmary
Manchester, Greater Manchester, M13 9WL
Wythenshawe Hospital
Manchester, Wythenshawe, M23 9LT
Thailand2 sites
Siriraj Hospital
Bangkoknoi, Bangkok, 10700
King Chulalongkorn Memorial Hospital
Pathum Wan, Bangkok, 10330
About this study
Official title: Phase IIb Study of F901318 as Treatment of Invasive Fungal Infections Due to Lomentospora Prolificans, Scedosporium Spp., Aspergillus Spp., and Other Resistant Fungi in Patients Lacking Suitable Alternative Treatment Options
A study to evaluate olorofim (F901318) for the treatment of invasive fungal infections in participants lacking suitable alternative treatment options.
Read the detailed description
An open label, single arm Phase IIb study of olorofim (F901318) in participants with invasive fungal infections with limited treatment options. Participants received study treatment for up to 12 weeks in the main phase of the study. At the Investigator's request and after discussion with the medical monitor, open-label treatment with F901318 could be continued in patients judged by the Investigator to need therapy beyond 84 days and considered likely to continue to benefit from extended treatment.
- Conditions
- Invasive Fungal Infections
- Being tested
- Olorofim
- Keywords
- Anti fungal, Invasive aspergillosis, Rare moulds, Coccidioidomycosis
- Medical subject headings
- Invasive Fungal Infections, Coccidioidomycosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OlorofimDrug
Also called F901318
30mg oral tablets
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Olorofim |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: Day 42 in the Main Phase of study treatment
Secondary outcomes (22)
DRC Adjudicated Overall Response at Day 42 for All Aspergillus
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 EORTC/MSG criteria. This was also presented by each of the 5 major infections as adjudicated by the DRC at baseline. The Aspergillus- All category is a combination of participants with Aspergillus proven and Aspergillus probable (invasive aspergillosis lower respiratory tract disease) baseline disease category. Overall success is defined as a Complete or Partial Response.
Measured: Day 42 in the Main Phase of study treatment
DRC Adjudicated Overall Response at Day 42 for Lomentospora Prolificans
The primary efficacy variable was the DRC-adjudicated overall response at the Day 42 Study Visit, as determined by an independent Data Review Committee using a combination of clinical, mycological, and radiological results based on EORTC/MSG criteria. This was also presented by each of the 5 major infections as adjudicated by the DRC at baseline, this one is for participants with a proven infection due to Lomentospora prolificans. Success is defined as a Complete or Partial Response.
Measured: 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
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 EORTC/MSG criteria. This was also presented by each of the 5 major infections as adjudicated by the DRC at baseline, this one is for participants with a proven infection due to Scedosporium species. Success is defined as a Complete or Partial Response.
Measured: Day 42 in the Main Phase of study treatment
DRC Adjudicated Overall Response at Day 42 for Coccidioides Species
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 EORTC/MSG criteria. This was also presented by each of the 5 major infections as adjudicated by the DRC at baseline, this one is for participants with proven infection due to Coccidioides species. Success is defined as a Complete or Partial Response.
Measured: Day 42 in the Main Phase of study treatment
DRC Adjudicated Overall Response at Day 42 for Other Olorofim Susceptible Fungi
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 EORTC/MSG criteria. This was also presented by each of the 5 major infections as adjudicated by the DRC at baseline, this one is for participants with proven infection due to other olorofim susceptible fungi. Success is defined as a Complete or Partial Response.
Measured: Day 42 in the Main Phase of study treatment
DRC Adjudicated Overall Response at Day 84
DRC-adjudicated overall response at Day 84, as determined by an independent DRC using a combination of clinical, mycological, and radiological results based on the EORTC/MSG criteria. For the 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 Study Visit could not be collected or participants who were considered not evaluable at the Study Visit).
Measured: Day 84 in the Main Phase of study treatment
Investigator Assessed Overall Response at Day 42
Investigator-assessed overall response (as determined by the Investigator using all available assessment results including clinical, mycological and radiologic results based on the EORTC/MSG criteria) at Day 42, categorised by the same response criteria as in the primary endpoint: Success (Success-Complete, Success-Partial), Failure (Failure-Stable, Failure-Progression, Death, participants for whom data at Day 42 could not be collected or who were considered not evaluable at the specific visit).
Measured: Day 42 in the Main Phase of study treatment
Investigator Assessed Overall Response at Day 84
Investigator-assessed overall response (as determined by the Investigator using all available assessment results including clinical, mycological and radiologic results based on the EORTC/MSG criteria) at Day 84, categorised by the same response criteria as in the primary endpoint: Success (Success-Complete, Success-Partial), Failure (Failure-Stable, Failure-Progression, Death, participants for whom data at Day 84 could not be collected or who were considered not evaluable at the specific visit).
Measured: Day 84 in the Main Phase of study treatment
DRC Adjudicated Clinical Response at Day 42
DRC adjudicated clinical response at the Day 42 Study Visit, as determined by an independent Data Review Committee using clinical response results based on EORTC/MSG criteria. Resolution is defined as a Complete or Partial Response.
Measured: Day 42 in the Main phase of study treatment
DRC Adjudicated Clinical Response at Day 84
DRC adjudicated clinical response at the Day 84 Study Visit, as determined by an independent DRC using clinical response results based on EORTC/MSG criteria. Resolution is defined as a Complete or Partial Response.
Measured: Day 84 in the Main phase of study treatment
Investigator Assessed Clinical Response at Day 42
Investigator assessed clinical response at the Day 42 Study Visit using clinical response results based on the EORTC/MSG criteria. Resolution is defined as a Complete or Partial Response.
Measured: Day 42 in the Main phase of study treatment
Investigator Assessed Clinical Response at Day 84
Investigator assessed clinical response at the Day 84 Study Visit using clinical response results based on the EORTC/MSG criteria. Resolution is defined as a Complete or Partial Response.
Measured: Day 84 in the Main Phase of study treatment
DRC Adjudicated Mycological Response at Day 42
DRC adjudicated mycological response was assessed at the Day 42 Study Visit, as determined by an independent Data Review Committee using mycological response results based on EORTC/MSG criteria. Success is defined as eradication or presumed eradication.
Measured: Day 42 in the Main Phase of study treatment
DRC Adjudicated Mycological Response at Day 84
DRC adjudicated mycological response was assessed at the Day 84 Study Visit, as determined by an independent DRC using mycological response results based on EORTC/MSG criteria. Success is defined as eradication or presumed eradication.
Measured: Day 84 in the Main phase of study treatment
Investigator Assessed Mycological Response at Day 42
Mycological response was assessed by the Investigator at the Day 42 Study Visit using mycological response results based on EORTC/MSG criteria. Success is defined as eradication or presumed eradication.
Measured: Day 42 in the Main Phase of study treatment
Investigator Assessed Mycological Response at Day 84
Mycological response was assessed by the Investigator at the Day 84 Study Visit using mycological response results based on EORTC/MSG criteria. Success is defined as eradication or presumed eradication.
Measured: Day 84 in the Main Phase of study treatment
DRC Adjudicated Radiological Response at Day 42
In participants for whom radiology formed a part of their diagnosis, radiology evaluations were required on Day 42 and were adjudicated by an independent DRC. Radiological responses were assigned as per EORTC/MSG criteria.
Measured: Day 42 in the Main phase of study treatment
DRC Adjudicated Radiological Response at Day 84
In participants for whom radiology formed a part of their diagnosis, radiology evaluations were required on Day 84 and were adjudicated by an independent DRC. Radiological responses were assigned as per EORTC/MSG criteria.
Measured: Day 84 in the Main Phase of study treatment
Investigator Assessed Radiological Response at Day 42
In participants for whom radiology formed a part of their diagnosis, radiology evaluations were required on Day 42. Radiological responses were assessed by the Investigator using EORTC/MSG criteria.
Measured: Day 42 in the Main Phase of study treatment
Investigator Assessed Radiological Response at Day 84
In participants for whom radiology formed a part of their diagnosis, radiology evaluations were required on Day 84. Radiological responses were assessed by the Investigator using EORTC/MSG criteria.
Measured: Day 84 in the Main Phase of study treatment
All Cause Mortality Rate at Day 42
The all cause mortality rate at Day 42 uses the survival status that was entered at the study visit (which employs a window around each nominal study day).
Measured: Day 42 in the Main Phase of study treatment
All Cause Mortality Rate at Day 84
The all cause mortality rate at Day 84 uses the survival status that was entered at the study visit (which employs a window around each nominal study day).
Measured: Day 84 in the Main Phase of study treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Jul 2018
- Study start6 Jun 2018
- Primary completion10 Feb 2023
- Study completion10 Feb 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Invasive Fungal InfectionsStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 203 participants (actual)
Sponsor and investigators
- Lead sponsor
- F2G Biotech GmbH
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- IQVIA Pty Ltd
- Responsible party
- Sponsor
- Investigators
- Sharon Chen, Principal investigator, Westmead Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03583164
- Sponsor's protocol ID
- F901318/0032
- Acronym
- FORMULA-OLS
- Status last verified
- Jul 2024
- First submitted
- 17 May 2018
- Last updated
- 10 Jul 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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)
Publications
Donovan FM, Johnson RH, Patterson TF, Hoenigl M, Spec A, Sikka MK, Holland SM, Shoham S, Schaenman JM, Mehta SR, Kuran RA, Galgiani JN, Bresnik M, Rex JH, Thompson GR 3rd. Olorofim in Treatment of Patients With Poorly Controlled Disseminated Coccidioidomycosis : A Single-Group, Open-Label, Phase 2b, Multicenter Study. Ann Intern Med. 2026 Jul 21. doi: 10.7326/ANNALS-26-00103. Online ahead of print.
PubMed 42475691Cornelissen K, Rex JH, Zinzi D, Law D, Upcott Gill R, Jones HM, Maertens J, Chen SC-A, Bruggemann R. Evaluation of the cytochrome P450-mediated drug interaction profile of olorofim. Antimicrob Agents Chemother. 2026 Jun 3;70(6):e0174525. doi: 10.1128/aac.01745-25. Epub 2026 Apr 20.
PubMed 42008658Maertens J, Chen SCA, Thompson GR 3rd, Donovan F, Dane A, Birch M, Pinder C, Bennett J, Ravenna V, Marin Fernandez O, Ross G, Zinzi D, Rex JH; Phase 2b Olorofim Salvage Therapy Study Group. Olorofim in combination with azole antifungals: results from the Phase 2 salvage therapy study. J Antimicrob Chemother. 2026 Mar 4;81(4):dkag118. doi: 10.1093/jac/dkag118.
PubMed 41914555Maertens 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.
PubMed 40541222
Study at a glance
From the registry record, last updated by the sponsor on 10 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase IIb Study of F901318 as Treatment of Invasive Fungal Infections Due to Lomentospora Prolificans, Scedosporium Spp., Aspergillus Spp., and Other Resistant Fungi in Patients Lacking Suitable Alternative Treatment Options
- ClinicalTrials.gov ID
- NCT03583164
- Acronym / sponsor ID
- FORMULA-OLS / F901318/0032
- Conditions
- Invasive Fungal Infections
- Keywords
- Anti fungal, Invasive aspergillosis, Rare moulds, Coccidioidomycosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 203 participants (actual)
- Lead sponsor
- F2G Biotech GmbHIndustry (pharma, biotech, devices)
- Collaborators
- IQVIA Pty Ltd
- Study start
- 6 Jun 2018 (actual)
- Primary completion
- 10 Feb 2023 (actual)
- Study completion
- 10 Feb 2023 (actual)
- First posted
- 11 Jul 2018 (actual)
- Results
- Posted 10 Jul 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A study to evaluate olorofim (F901318) for the treatment of invasive fungal infections in participants lacking suitable alternative treatment options.
Detailed description
An open label, single arm Phase IIb study of olorofim (F901318) in participants with invasive fungal infections with limited treatment options. Participants received study treatment for up to 12 weeks in the main phase of the study. At the Investigator's request and after discussion with the medical monitor, open-label treatment with F901318 could be continued in patients judged by the Investigator to need therapy beyond 84 days and considered likely to continue to benefit from extended treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Olorofim (F901318) | Experimental | Drug: OlorofimOlorofim (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. |
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (9 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| OlorofimAlso: F90131830mg oral tablets | Drug | Olorofim (F901318) |
Primary outcome
Data Review Committee (DRC) Adjudicated Overall Response at Day 42
Time frame: Day 42 in the Main Phase of study treatment
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).
22 secondary outcomes
DRC Adjudicated Overall Response at Day 42 for All Aspergillus
Time frame: Day 42 in the Main Phase of study treatment
DRC Adjudicated Overall Response at Day 42 for Lomentospora Prolificans
Time frame: 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
Time frame: Day 42 in the Main Phase of study treatment
DRC Adjudicated Overall Response at Day 42 for Coccidioides Species
Time frame: Day 42 in the Main Phase of study treatment
DRC Adjudicated Overall Response at Day 42 for Other Olorofim Susceptible Fungi
Time frame: Day 42 in the Main Phase of study treatment
DRC Adjudicated Overall Response at Day 84
Time frame: Day 84 in the Main Phase of study treatment
Investigator Assessed Overall Response at Day 42
Time frame: Day 42 in the Main Phase of study treatment
Investigator Assessed Overall Response at Day 84
Time frame: Day 84 in the Main Phase of study treatment
DRC Adjudicated Clinical Response at Day 42
Time frame: Day 42 in the Main phase of study treatment
DRC Adjudicated Clinical Response at Day 84
Time frame: Day 84 in the Main phase of study treatment
Investigator Assessed Clinical Response at Day 42
Time frame: Day 42 in the Main phase of study treatment
Investigator Assessed Clinical Response at Day 84
Time frame: Day 84 in the Main Phase of study treatment
DRC Adjudicated Mycological Response at Day 42
Time frame: Day 42 in the Main Phase of study treatment
DRC Adjudicated Mycological Response at Day 84
Time frame: Day 84 in the Main phase of study treatment
Investigator Assessed Mycological Response at Day 42
Time frame: Day 42 in the Main Phase of study treatment
Investigator Assessed Mycological Response at Day 84
Time frame: Day 84 in the Main Phase of study treatment
DRC Adjudicated Radiological Response at Day 42
Time frame: Day 42 in the Main phase of study treatment
DRC Adjudicated Radiological Response at Day 84
Time frame: Day 84 in the Main Phase of study treatment
Investigator Assessed Radiological Response at Day 42
Time frame: Day 42 in the Main Phase of study treatment
Investigator Assessed Radiological Response at Day 84
Time frame: Day 84 in the Main Phase of study treatment
All Cause Mortality Rate at Day 42
Time frame: Day 42 in the Main Phase of study treatment
All Cause Mortality Rate at Day 84
Time frame: Day 84 in the Main Phase of study treatment
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Jul 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 42475691Donovan FM, Johnson RH, Patterson TF, Hoenigl M, Spec A, Sikka MK, Holland SM, Shoham S, Schaenman JM, Mehta SR, Kuran RA, Galgiani JN, Bresnik M, Rex JH, Thompson GR 3rd. Olorofim in Treatment of Patients With Poorly Controlled Disseminated Coccidioidomycosis : A Single-Group, Open-Label, Phase 2b, Multicenter Study. Ann Intern Med. 2026 Jul 21. doi: 10.7326/ANNALS-26-00103. Online ahead of print.
- Derived from this study: PubMed 42008658Cornelissen K, Rex JH, Zinzi D, Law D, Upcott Gill R, Jones HM, Maertens J, Chen SC-A, Bruggemann R. Evaluation of the cytochrome P450-mediated drug interaction profile of olorofim. Antimicrob Agents Chemother. 2026 Jun 3;70(6):e0174525. doi: 10.1128/aac.01745-25. Epub 2026 Apr 20.
- Derived from this study: PubMed 41914555Maertens J, Chen SCA, Thompson GR 3rd, Donovan F, Dane A, Birch M, Pinder C, Bennett J, Ravenna V, Marin Fernandez O, Ross G, Zinzi D, Rex JH; Phase 2b Olorofim Salvage Therapy Study Group. Olorofim in combination with azole antifungals: results from the Phase 2 salvage therapy study. J Antimicrob Chemother. 2026 Mar 4;81(4):dkag118. doi: 10.1093/jac/dkag118.
- Derived from this study: PubMed 40541222Maertens 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.
- Individual participant dataSharing planned: Yes. 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)
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Sharon Chen principal investigator
Westmead Hospital
82 sites in 17 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Olorofim in people with Invasive Fungal Infections, run by F2G Biotech GmbH at 82 sites in 17 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 10 Jul 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 82 sites in United States, Brazil, Egypt, Australia, Israel and 12 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 10 Jul 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Data Review Committee (DRC) Adjudicated Overall Response at Day 42 (time frame: Day 42 in the Main Phase of study treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03583164, which also lists 4 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Jul 2024.