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Active, not recruitingNCT06717126OCULE-01Updated Jan 16, 2026

A Randomised Phase II Study of Roginolisib in Patients With Advanced/Metastatic Uveal Melanoma

A Phase 2 interventional study of roginolisib and Investigator choice of standard therapy in Uveal Melanoma and Ocular Melanoma, sponsored by iOnctura. Active, not recruiting at 16 sites in 3 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-01-16.

Sponsored by iOnctura · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
85
Allocation
Randomized
Ages
18 Years and older
Sex
All
01

Study 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 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).

02

Conditions studied

  • Uveal Melanoma
  • Ocular Melanoma

Keywords

  • Uveal
  • Ocular
  • Melanoma
03

In context

Uveal Melanoma

103 studies on the registry are indexed under Uveal Melanoma; 38 are open to participants now.

This study's planned enrollment of 85 is above the median of 42 across 93 interventional studies indexed under Uveal Melanoma.

Browse Uveal Melanoma studies →

Lead sponsor

iOnctura is the lead sponsor of 6 studies on the registry; 1 is open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  1. Male or female aged 18 years or older;
  2. Histologically or cytologically proven diagnosis of advanced or metastatic UM or ocular melanoma (arising from ocular melanocytes regardless of intraocular location)
  3. 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;
  4. 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);
  5. 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;
  6. ECOG performance status of 0 to 1;
  7. 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)
  8. 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;
  9. Provision of signed and dated, written informed consent prior to any study specific procedures, sampling and analyses.

Exclusion criteria

Exclusion Criteria:

  1. Inability to swallow oral medication;
  2. 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;
  3. 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;

  4. Abnormal liver enzymes defined as:

    1. ALT or AST ≥ 3× upper limit of normal (ULN) (≥ 5× ULN in patients with liver metastases);
    2. 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);
  5. Any other clinically significant out of range laboratory values;
  6. 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;
  7. Evidence of interstitial lung disease or active, non-infectious pneumonitis, pulmonary fibrosis;
  8. 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;
  9. Active hepatitis B virus (HBV) or hepatitis C virus (HCV) infection per institutional protocol;
  10. 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;
  11. 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;
  12. 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;
  13. 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);
  14. 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;
  15. Pregnant, likely to become pregnant, or lactating women.
05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
None (open label)
Enrollment
85 participants (estimated)

Study arms

  • Experimental
    Arm 1 - Roginolisib 80mg

    IOA-244: 80 mg (corresponding to 72 mg roginolisib) daily

    Drug: roginolisib

  • Active comparator
    Arm 2 - Investigator choice of standard of care

    Investigator´s choice of therapy

    Drug: Investigator choice of standard therapy

  • Experimental
    Arm 3 - Roginolisib 40mg

    IOA-244: 40 mg (corresponding to 36 mg roginolisib) daily

    Drug: roginolisib

Interventions

  • Drugroginolisib

    rognolisib

    Also known as: IOA-244

  • DrugInvestigator choice of standard therapy

    Investigator will choose the most appropriate treatment standardly given to patients

06

What researchers measure

Primary outcomes

  1. Overall survival

    To evaluate clinical efficacy of roginolisib as single agent, against Investigator's choice of therapy by assessment of overall survival (OS)

    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

Secondary outcomes

  1. 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

    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

  2. Objective response rate (ORR)

    ORR defined as percentage of patients with a Complete Response (CR) or Partial Response (PR)

    Time frame: Every 8 weeks whilst on treatment anticipated to be 52 weeks

  3. 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

    Time frame: Every 8 weeks up to 96 weeks from start of treatment

  4. 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

    Time frame: Every 8 weeks whilst on treatment anticipated to be 52 weeks

  5. 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),

    Time frame: Every 8 weeks whilst on treatment anticipated to be 52 weeks

  6. 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

    Time frame: Measured at Cycle 5 - approximately 16 weeks from start of dosing

  7. Safety and tolerability

    Assessed by AEs, laboratory parameters, vital signs, physical exam, ECG and ECOG status

    Time frame: Every 4 weeks whilst on treatment anticipated to be 52 weeks

  8. Pharmacokinetics (PK)

    Concentration of roginolisib at pre-dose and steady state levels (including Area under the curve \[AUC\], population PK)

    Time frame: Every 4 weeks for 52 weeks from start of treatment

  9. Safety of 40 vs 80 mg of roginolisib

    Assessed by AEs, laboratory parameters, vital signs, physical exam, ECG and ECOG status

    Time frame: Every 4 weeks whilst on treatment anticipated to be 52 weeks

  10. Health care utilisation

    Assessed by health resource used

    Time frame: Every 4 weeks whilst on treatment anticipated to be 52 weeks

  11. Quality of Life

    Changes in Patient Reported Outcomes (PRO) relative to baseline. Patients to complete EuroQoL Research Foundation EQ-5D-5L Health questionnaire

    Time frame: Every 4 weeks for 52 weeks from start of treatment

  12. 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)

    Time frame: Every 4 weeks for 52 weeks from start of treatment

  13. Quality of Life

    Changes in Patient Reported Outcomes (PRO) relative to baseline. Patients to complete Epworth Sleepiness Scale (ESS) questionnaire

    Time frame: Every 4 weeks for 52 weeks from start of treatment

  14. Quality of Life

    Changes in Patient Reported Outcomes (PRO) relative to baseline. Patients to complete Fatigue Severity Scale (FSS)

    Time frame: Every 4 weeks for 52 weeks from start of treatment

Other outcomes

  1. Circulating Tumour Deoxyribonucleic Acid (ctDNA)

    To assess any treatment-related changes in the pre and on treatment levels of circulating DNA from blood

    Time frame: Every 4 weeks for 52 weeks from start of treatment

07

Study locations

16 sites
  • SSD Tumori Rari e Melanoma Viale Orazio Flacco
    Bari, 70124, Italy
  • IRCSS National Cancer Institute, "G.Pascale" Foundation Dip. CORP-S di Ricerca ed Assistenziale Cute, Melanoma lmmunologia Oncologica Sperimentale e Terapie Innovative
    Naples, 80131, Italy
  • IRCSS Istituto Oncologico Veneto UOS Oncologia 2 del Melanoma Ospedale Busonera
    Padova, 35128, Italy
  • IRCCS Istituto Clinico Humanitas
    Rozzano, 20089, Italy
  • A.O.U.S. Santa Maria delle Scotte
    Siena, 53100, Italy
  • Institut Catala d'Oncologia - ICO L'Hospitalet
    Barcelona, 08908, Spain
  • Hospital Universitario La Paz
    Madrid, 28046, Spain
  • Complejo Hospitalario Universitario de Santiago - CHUS
    Santiago de Compostela, 15706, Spain
  • Hospital Universitario Virgen Macarena, University of Seville
    Seville, Spain
  • Consorcio Hospital General Universitario de València - CHGUV
    Valencia, 46014, Spain
  • East and North Hertfordshire NHS Trust (Mount Vernon Cancer Centre)
    Northwood, Middlesex HA6 2RN, United Kingdom
  • The Clatterbridge Cancer Centre NHS Foundation Trust
    Bebington, Wirral CH63 4JH, United Kingdom
  • Beatson West of Scotland Cancer Centre, Glasgow (NHS Greater Glasgow & Clyde)
    Glasgow, G12 0YN, United Kingdom
  • University College London Hospital NHS
    London, NW1 2PG, United Kingdom
  • Royal Marsden Hospital
    London, SW3 6JJ, United Kingdom
  • University Hospital Southampton NHS Foundation
    Southampton, SO16 6YD, United Kingdom
08

References and documents

Individual participant data

Plan to share: Undecided

No publications or documents are linked to this record.

09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jan 16, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT06717126
Lead sponsor
iOnctura
Responsible party
Sponsor
First posted
Dec 4, 2024
Start date
Feb 27, 2025
Primary completion
Dec 2027 (estimated)
Completion
Dec 2028 (estimated)
Last update
Jan 16, 2026

Study contacts

Michael Lahn, MD
study director · iOnctura

Oversight

Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is active, not recruiting, as verified in Jan 2026. You cannot join it, but the record below documents what was studied.

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