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?
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 Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
85
rognolisib
Investigator will choose the most appropriate treatment standardly given to patients
SSD Tumori Rari e Melanoma Viale Orazio Flacco
Bari, Italy
IRCSS National Cancer Institute, "G.Pascale" Foundation Dip. CORP-S di Ricerca ed Assistenziale Cute, Melanoma lmmunologia Oncologica Sperimentale e Terapie Innovative
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
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
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
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
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
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),
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Naples, Italy
IRCSS Istituto Oncologico Veneto UOS Oncologia 2 del Melanoma Ospedale Busonera
Padova, Italy
IRCCS Istituto Clinico Humanitas
Rozzano, Italy
A.O.U.S. Santa Maria delle Scotte
Siena, Italy
Institut Catala d'Oncologia - ICO L'Hospitalet
Barcelona, Spain
Hospital Universitario La Paz
Madrid, Spain
Complejo Hospitalario Universitario de Santiago - CHUS
Santiago de Compostela, Spain
Hospital Universitario Virgen Macarena, University of Seville
Seville, Spain
Consorcio Hospital General Universitario de València - CHGUV
Valencia, Spain
...and 6 more locations
Time frame: 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
Time frame: 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
Time frame: 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)
Time frame: 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
Time frame: Every 4 weeks whilst on treatment anticipated to be 52 weeks
Health care utilisation
Assessed by health resource used
Time frame: 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
Time frame: 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)
Time frame: 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
Time frame: 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)
Time frame: Every 4 weeks for 52 weeks from start of treatment