Uveal MelanomaPhase 2OCULE-01NCT06717126
A Randomised Phase II Study of Roginolisib in Patients With Advanced/Metastatic Uveal Melanoma
A Phase 2 study testing roginolisib and Investigator choice of standard therapy in people with Uveal Melanoma and Ocular Melanoma, run by iOnctura at 16 sites in 3 countries.
Trial summary
The goal of this clinical trial is to learn how roginolisib works in comparison to standard treatment in adult patients with uveal/ocular melanoma. The main questions it aims to answer are: Does roginolisib extend overall survival compared to standard treatment? How does dosing of roginolisib impact quality of life… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Uveal Melanoma, Ocular Melanoma
- What is being tested?
- roginolisib and Investigator choice of standard therapy
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 85 participants
- Where
- 16 sitesin 3 countries
- Study dates
- 27 Feb 2025to Dec 2028
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female aged 18 years or older;
- Histologically or cytologically proven diagnosis of advanced or metastatic UM or ocular melanoma (arising from ocular melanocytes regardless of intraocular location)
- Patients who have progressed following at least 1 prior immunotherapy treatment for advanced or metastatic UM. For patients who are HLA-A*02:01 positive prior treatment should have included tebentafusp, if available or patients clinically suitable. Patients who have also received prior melphalan hepatic infusion may be included;
- Presence of at least one lesion suitable for biopsy. Biopsies will be mandatory at Screening and C5D1 (see Sections 8.1.3 and 8.6 for more information);
- Presence of at least one measurable lesion as per RECIST v1.1. Any lesion that is biopsied cannot be used as a measurable lesion for the purposes of RECIST v1.1 assessments;
- ECOG performance status of 0 to 1;
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- Male or female patients of child-bearing potential must be willing to use highly effective forms of contraception (refer to APPENDIX 7 for details on highly effective methods of contraception and definitions of women of childbearing potential and of fertile men)
- All other relevant medical conditions must be well managed and stable, in the Investigator's opinion, for at least 28 days prior to first dose of roginolisib;
- Provision of signed and dated, written informed consent prior to any study specific procedures, sampling and analyses.
Who cannot join
- Inability to swallow oral medication;
- a). History of a prior Grade 3 or 4 irAE or any grade ocular irAE from prior immunotherapy which did not respond to corticosteroid therapy or resolved with treatment interruptions and returned to at least Grade 1; b). Have not recovered from toxic effect(s) of prior therapy to ≤ Grade 1, other than alopecia or fatigue or neuropathy which must be ≤ Grade 1;
- Presence of symptomatic or untreated CNS metastases or CNS metastases that require doses of corticosteroids within the prior 3 weeks to first dose of roginolisib. Patients with brain metastases are eligible if lesions have been treated with localised therapy and there is no evidence of progressive disease for at least 4 weeks prior to the first dose of IMP;
- Abnormal liver enzymes defined as:
- ALT or AST ≥ 3× upper limit of normal (ULN) (≥ 5× ULN in patients with liver metastases);
- Total bilirubin ≥ 1.5 × ULN are excluded unless direct bilirubin is ≤ ULN. If there is no institutional ULN, then direct bilirubin must be \< 40% of total bilirubin to be eligible (except patients with Gilbert syndrome);
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- Any other clinically significant out of range laboratory values;
- Clinically significant cardiac disease or impaired cardiac function which may limit the patient´s participation in the clinical study. These may include unstable angina (i.e., not responsive to medical intervention), myocardial infarct in last 6 months, QTcF prolongation of more than 500 ms;
- Evidence of interstitial lung disease or active, non-infectious pneumonitis, pulmonary fibrosis;
- Active infection requiring systemic antibiotic therapy. Patients requiring systemic antibiotics for infection must have completed therapy at least 1 week prior to the first dose of IMP;
- Active hepatitis B virus (HBV) or hepatitis C virus (HCV) infection per institutional protocol;
- Malignant disease, other than that being treated in this study (e.g., skin/cutaneous and/or mucosal melanoma). Exceptions to this exclusion include the following: malignancies that were treated curatively and have not recurred within 2 years prior to first dose of IMP; completely resected basal cell and squamous cell skin cancers; any malignancy considered to be indolent and that has never required therapy; and completely resected carcinoma in situ of any type;
- Any medical condition that would, in the Investigator\'s or Sponsor\'s judgment, prevent the patient\'s participation in the clinical study due to safety concerns, compliance with clinical study procedures or interpretation of study results;
- Treatment with anti-tumour medications or investigational drugs within 14 days or 5 half-lives (whichever is longer) of administration of first dose of IMP;
- Major surgery within 2 weeks of the first dose of IMP (minimally invasive procedures such as bronchoscopy, tumour biopsy, insertion of a central venous access device, and insertion of a feeding tube are not considered major surgery and are not exclusionary);
- Radiotherapy within 4 weeks of the first dose of IMP, with the exception of palliative radiotherapy to a limited field, such as for the treatment of bone pain or a focally painful tumour mass;
- Pregnant, likely to become pregnant, or lactating women.
Trial locations and contacts
16 sites in 3 countries.
16 sites in 3 countries
United Kingdom6 sites
The Clatterbridge Cancer Centre NHS Foundation Trust
Bebington, Wirral, CH63 4JH
Beatson West of Scotland Cancer Centre, Glasgow (NHS Greater Glasgow & Clyde)
Glasgow, G12 0YN
University College London Hospital NHS
London, NW1 2PG
Royal Marsden Hospital
London, SW3 6JJ
East and North Hertfordshire NHS Trust (Mount Vernon Cancer Centre)
Northwood, Middlesex, HA6 2RN
University Hospital Southampton NHS Foundation
Southampton, SO16 6YD
Italy5 sites
SSD Tumori Rari e Melanoma Viale Orazio Flacco
Bari, 70124
IRCSS National Cancer Institute, "G.Pascale" Foundation Dip. CORP-S di Ricerca ed Assistenziale Cute, Melanoma lmmunologia Oncologica Sperimentale e Terapie Innovative
Naples, 80131
IRCSS Istituto Oncologico Veneto UOS Oncologia 2 del Melanoma Ospedale Busonera
Padova, 35128
IRCCS Istituto Clinico Humanitas
Rozzano, 20089
A.O.U.S. Santa Maria delle Scotte
Siena, 53100
Spain5 sites
Institut Catala d'Oncologia - ICO L'Hospitalet
Barcelona, 08908
Hospital Universitario La Paz
Madrid, 28046
Complejo Hospitalario Universitario de Santiago - CHUS
Santiago de Compostela, 15706
Hospital Universitario Virgen Macarena, University of Seville
Seville
Consorcio Hospital General Universitario de València - CHGUV
Valencia, 46014
About this study
Official title: A Phase II, Multi-centre, Open Label, Randomised Study to Evaluate the Anti-tumour Activity of Roginolisib in Patients With Advanced/Metastatic Ocular/Uveal Melanoma
The goal of this clinical trial is to learn how roginolisib works in comparison to standard treatment in adult patients with uveal/ocular melanoma. The main questions it aims to answer are:
Does roginolisib extend overall survival compared to standard treatment? How does dosing of roginolisib impact quality of life compared to standard treatment?
Read the detailed description
A Phase II open-label, randomised, parallel-arm study, which will assess the clinical efficacy of oral roginolisib (IOA 244 [roginolisib hemi-fumarate]) as monotherapy against a control of Investigator´s treatment choice in patients with advanced or metastatic uveal melanoma (UM).
This study will enrol approximately 85 male and female patients aged over 18 years with advanced or metastatic UM, who have progressed following at least 1 prior immunotherapy treatment. The disease must be measurable (i.e., at least 1 measurable lesion) as per RECIST v1.1 by Computerised Tomography (CT) scan or Magnetic Resonance Imaging (MRI).
- Conditions
- Uveal Melanoma, Ocular Melanoma
- Being tested
- roginolisib, Investigator choice of standard therapy
- Keywords
- Uveal, Ocular, Melanoma
- Medical subject headings
- Uveal Melanoma, Melanoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
roginolisibDrug
Also called IOA-244
rognolisib
Investigator choice of standard therapyDrug
Investigator will choose the most appropriate treatment standardly given to patients
| Study group | Role | What they receive |
|---|---|---|
| Arm 1 - Roginolisib 80mg | Experimental | IOA-244: 80 mg (corresponding to 72 mg roginolisib) daily Receives: Drug: roginolisib |
| Arm 2 - Investigator choice of standard of care | Active comparator | Investigator´s choice of therapy Receives: Drug: Investigator choice of standard therapy |
| Arm 3 - Roginolisib 40mg | Experimental | IOA-244: 40 mg (corresponding to 36 mg roginolisib) daily Receives: Drug: roginolisib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall survival
To evaluate clinical efficacy of roginolisib as single agent, against Investigator's choice of therapy by assessment of overall survival (OS)
Measured: Patients will be followed up for overall survival every 12 weeks, for 96 weeks from last patient enrolled, until their death or end of the study
Secondary outcomes (14)
Progression free survival (PFS)
PFS measured from the time from the date of the first dose of IMP until the earliest date of disease progression as determined by radiographic/objective disease assessment as per RECIST v1.1
Measured: Patients will be followed up for progression free survival every 8 weeks, for 96 weeks from last patient enrolled, until progression of disease, their death or end of the study
Objective response rate (ORR)
ORR defined as percentage of patients with a Complete Response (CR) or Partial Response (PR)
Measured: Every 8 weeks whilst on treatment anticipated to be 52 weeks
Duration of response (DOR)
DOR defined as the time from the date of first documented response (CR, PR) by RECIST v1.1 until the date of documented progression or death in the absence of disease progression
Measured: Every 8 weeks up to 96 weeks from start of treatment
Time to response
Time to Response is defined as the time from date of first dose of IMP until the date of first documented objective response
Measured: Every 8 weeks whilst on treatment anticipated to be 52 weeks
Disease control rate (DCR)
DCR is defined as the proportion of patients with a Best Objective Response (BOR) of CR or PR or Stable disease (SD) recorded at ≥8 weeks (±1 week),
Measured: Every 8 weeks whilst on treatment anticipated to be 52 weeks
Clinical benefit rate (CBR)
CBR is defined as the proportion of patients with a BOR of CR or PR or SD recorded at Cycle 5 Day 1
Measured: Measured at Cycle 5 - approximately 16 weeks from start of dosing
Safety and tolerability
Assessed by AEs, laboratory parameters, vital signs, physical exam, ECG and ECOG status
Measured: Every 4 weeks whilst on treatment anticipated to be 52 weeks
Pharmacokinetics (PK)
Concentration of roginolisib at pre-dose and steady state levels (including Area under the curve [AUC], population PK)
Measured: Every 4 weeks for 52 weeks from start of treatment
Safety of 40 vs 80 mg of roginolisib
Assessed by AEs, laboratory parameters, vital signs, physical exam, ECG and ECOG status
Measured: Every 4 weeks whilst on treatment anticipated to be 52 weeks
Health care utilisation
Assessed by health resource used
Measured: Every 4 weeks whilst on treatment anticipated to be 52 weeks
Quality of Life
Changes in Patient Reported Outcomes (PRO) relative to baseline. Patients to complete EuroQoL Research Foundation EQ-5D-5L Health questionnaire
Measured: Every 4 weeks for 52 weeks from start of treatment
Quality of Life
Changes in Patient Reported Outcomes (PRO) relative to baseline. Patients to complete European Organisation for Research and Treatment of Cancer (EORTC QlQ-C30)
Measured: Every 4 weeks for 52 weeks from start of treatment
Quality of Life
Changes in Patient Reported Outcomes (PRO) relative to baseline. Patients to complete Epworth Sleepiness Scale (ESS) questionnaire
Measured: Every 4 weeks for 52 weeks from start of treatment
Quality of Life
Changes in Patient Reported Outcomes (PRO) relative to baseline. Patients to complete Fatigue Severity Scale (FSS)
Measured: Every 4 weeks for 52 weeks from start of treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Dec 2024
- Study start27 Feb 2025
- Primary completionDec 2027 (estimated)
- Study completionDec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Uveal MelanomaStudies 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
- Randomised
- Intervention model
- Parallel groups
- Model description
- Randomised parallel-arm, open-label
- Masking
- None (open label)
- Enrolment
- 85 participants (estimated)
Sponsor and investigators
- Lead sponsor
- iOnctura
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Michael Lahn, MD, Study director, iOnctura
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06717126
- Sponsor's protocol ID
- IOA-244-201
- Acronym
- OCULE-01
- Status last verified
- Jan 2026
- First submitted
- 18 Nov 2024
- Last updated
- 16 Jan 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 16 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II, Multi-centre, Open Label, Randomised Study to Evaluate the Anti-tumour Activity of Roginolisib in Patients With Advanced/Metastatic Ocular/Uveal Melanoma
- ClinicalTrials.gov ID
- NCT06717126
- Acronym / sponsor ID
- OCULE-01 / IOA-244-201
- Conditions
- Uveal Melanoma, Ocular Melanoma
- Keywords
- Uveal, Ocular, Melanoma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 85 participants (estimated)
- Lead sponsor
- iOncturaIndustry (pharma, biotech, devices)
- Study start
- 27 Feb 2025 (actual)
- Primary completion
- Dec 2027 (estimated)
- Study completion
- Dec 2028 (estimated)
- First posted
- 4 Dec 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The goal of this clinical trial is to learn how roginolisib works in comparison to standard treatment in adult patients with uveal/ocular melanoma. The main questions it aims to answer are:
Does roginolisib extend overall survival compared to standard treatment? How does dosing of roginolisib impact quality of life compared to standard treatment?
Detailed description
A Phase II open-label, randomised, parallel-arm study, which will assess the clinical efficacy of oral roginolisib (IOA 244 [roginolisib hemi-fumarate]) as monotherapy against a control of Investigator´s treatment choice in patients with advanced or metastatic uveal melanoma (UM).
This study will enrol approximately 85 male and female patients aged over 18 years with advanced or metastatic UM, who have progressed following at least 1 prior immunotherapy treatment. The disease must be measurable (i.e., at least 1 measurable lesion) as per RECIST v1.1 by Computerised Tomography (CT) scan or Magnetic Resonance Imaging (MRI).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
- Model description
- Randomised parallel-arm, open-label
| Arm | Type | Receives |
|---|---|---|
| Arm 1 - Roginolisib 80mg | Experimental | Drug: roginolisibIOA-244: 80 mg (corresponding to 72 mg roginolisib) daily |
| Arm 2 - Investigator choice of standard of care | Active comparator | Drug: Investigator choice of standard therapyInvestigator´s choice of therapy |
| Arm 3 - Roginolisib 40mg | Experimental | Drug: roginolisibIOA-244: 40 mg (corresponding to 36 mg roginolisib) daily |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Male or female aged 18 years or older;
- Histologically or cytologically proven diagnosis of advanced or metastatic UM or ocular melanoma (arising from ocular melanocytes regardless of intraocular location)
- Patients who have progressed following at least 1 prior immunotherapy treatment for advanced or metastatic UM. For patients who are HLA-A*02:01 positive prior treatment should have included tebentafusp, if available or patients clinically suitable. Patients who have also received prior melphalan hepatic infusion may be included;
- Presence of at least one lesion suitable for biopsy. Biopsies will be mandatory at Screening and C5D1 (see Sections 8.1.3 and 8.6 for more information);
- Presence of at least one measurable lesion as per RECIST v1.1. Any lesion that is biopsied cannot be used as a measurable lesion for the purposes of RECIST v1.1 assessments;
Key exclusion criteria (17 listed)
- Inability to swallow oral medication;
- a). History of a prior Grade 3 or 4 irAE or any grade ocular irAE from prior immunotherapy which did not respond to corticosteroid therapy or resolved with treatment interruptions and returned to at least Grade 1; b). Have not recovered from toxic effect(s) of prior therapy to ≤ Grade 1, other than alopecia or fatigue or neuropathy which must be ≤ Grade 1;
- Presence of symptomatic or untreated CNS metastases or CNS metastases that require doses of corticosteroids within the prior 3 weeks to first dose of roginolisib. Patients with brain metastases are eligible if lesions have been treated with localised therapy and there is no evidence of progressive disease for at least 4 weeks prior to the first dose of IMP;
- Abnormal liver enzymes defined as:
- Any other clinically significant out of range laboratory values;
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 |
|---|---|---|
| roginolisibAlso: IOA-244rognolisib | Drug | Arm 1 - Roginolisib 80mg, Arm 3 - Roginolisib 40mg |
| Investigator choice of standard therapyInvestigator will choose the most appropriate treatment standardly given to patients | Drug | Arm 2 - Investigator choice of standard of care |
Primary outcome
Overall survival
Time frame: Patients will be followed up for overall survival every 12 weeks, for 96 weeks from last patient enrolled, until their death or end of the study
To evaluate clinical efficacy of roginolisib as single agent, against Investigator's choice of therapy by assessment of overall survival (OS)
14 secondary outcomes
Progression free survival (PFS)
Time frame: Patients will be followed up for progression free survival every 8 weeks, for 96 weeks from last patient enrolled, until progression of disease, their death or end of the study
Objective response rate (ORR)
Time frame: Every 8 weeks whilst on treatment anticipated to be 52 weeks
Duration of response (DOR)
Time frame: Every 8 weeks up to 96 weeks from start of treatment
Time to response
Time frame: Every 8 weeks whilst on treatment anticipated to be 52 weeks
Disease control rate (DCR)
Time frame: Every 8 weeks whilst on treatment anticipated to be 52 weeks
Clinical benefit rate (CBR)
Time frame: Measured at Cycle 5 - approximately 16 weeks from start of dosing
Safety and tolerability
Time frame: Every 4 weeks whilst on treatment anticipated to be 52 weeks
Pharmacokinetics (PK)
Time frame: Every 4 weeks for 52 weeks from start of treatment
Safety of 40 vs 80 mg of roginolisib
Time frame: Every 4 weeks whilst on treatment anticipated to be 52 weeks
Health care utilisation
Time frame: Every 4 weeks whilst on treatment anticipated to be 52 weeks
Quality of Life
Time frame: Every 4 weeks for 52 weeks from start of treatment
Quality of Life
Time frame: Every 4 weeks for 52 weeks from start of treatment
Quality of Life
Time frame: Every 4 weeks for 52 weeks from start of treatment
Quality of Life
Time frame: Every 4 weeks for 52 weeks from start of treatment
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Jan 2026.
- Individual participant dataSharing planned: Undecided
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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.
Michael Lahn, MD study director
iOnctura
16 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing roginolisib and Investigator choice of standard therapy in people with Uveal Melanoma and Ocular Melanoma, run by iOnctura at 16 sites in 3 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 9 inclusion and 17 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: under way, not looking for participants, as of the sponsor's last update on 16 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 16 sites in United Kingdom, Italy, Spain. 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?
This study is still under way. Results are not available in the source record yet.
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?
Overall survival (time frame: Patients will be followed up for overall survival every 12 weeks, for 96 weeks from last patient enrolled, until their death or end of the study). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 16 Jan 2026.