The goal of this clinical trial is to learn how roginolisib works in comparison to standard treatment in adult patients with Myelofibrosis. The main questions it aims to answer is to evaluate the safety and tolerability of roginolisib when administered in combination with ruxolitinib.
A Phase I/II Open-Label, Single Arm Multi-centre Study to Assess the Safety and Tolerability of Roginolisib in Combination with Ruxolitinib in Patients with Myelofibrosis (MF) who are Unresponsive to JAK inhibitors (HEMA-MED). This study will enrol approximately 26 male and female patients aged over 18 years with MF, who have been treated with ruxolitinib for ≥ 3 months with a stable dose ≥ 10 mg for at least the last 8 weeks prior to Day 1 and no significant spleen reduction. The study will initially enrol 13 patients in Part 1 to assess the benefit/risk profile of roginolisib when combined with ruxolitinib. Part 2 will enrol an additional 13 patients to further characterize the benefit/risk.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
26
IOA-244: 80 mg (corresponding to 72 mg roginolisib) Ruxolitinib: up to 25 mg BD
Azienda Ospedaliero Universitaria Careggi Firenze, Struttura Complessa di Ematologia
Florence, Florence, Italy
RECRUITINGIRCCS Clinical Institute Humanitas
Rozzano, Milan, Italy
RECRUITINGIstituto di Ematolgia e Oncologia Medica
Bologna, Italy
Number of participants with treatment emergent adverse events
Safety measure by Adverse Event's
Time frame: Day 1 and 15 of cycle 1; Day 1 of each subsequent cycle whilst on treatment anticipated to be 52 weeks. Cycle length 28 days
Number of Participants with abnormal ECG recordings
Changes observed on 12-lead ECG
Time frame: Day 1 of each cycle whilst on treatment anticipated to be 52 weeks. Cycle length 28 days
Number of participants with abnormal laboratory parameters
Standard laboratory parameters (including clinical chemistry, heamatology)
Time frame: Day 1 and 15 of cycle 1; Day 1 of each subsequent cycle whilst on treatment anticipated to be 52 weeks. Cycle length 28 days
Number of participants with changes in blood pressure measurements
As measured by blood pressure measurements
Time frame: Day 1 and 15 of cycle 1; Day 1 of each subsequent cycle whilst on treatment anticipated to be 52 weeks. Cycle length 28 days
To evaluate biomarker responses (e.g., Treg reduction) when roginolisib is administered in combination with ruxolitinib in patients with MF.
Changes in peripheral blood Tregs from baseline at Week 12 and continued reduction over time
Time frame: Day 1 of Cycle 1, 2 and 3 and then Day 1 of every other cycle (C5, C7 etc) until end of treatment anticipated to be 52 weeks. Cycle length is 28 days
Spleen reduction responses of roginolisib when administered in combination with ruxolitinib in patients with MF.
Splenic response rate (SRR) of ≥15%, ≥25% and ≥35% reduction in spleen volume as assessed by MRI/CT Duration of spleen response
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Azienda Sanitario Universitaria Friuli Centrale
Udine, Italy
RECRUITINGVall d'Hebron University Hospital
Barcelona, Spain
RECRUITINGAvigunda Gran via de l'Hospitalet 199-203, 08908 L'Hospitalet de Llobregat
Barcelona, Spain
RECRUITINGHospital Universitario de Salamanca
Salamanca, Spain
RECRUITINGTime frame: Splenic response rate at baseline, 12 and 24 weeks. Duration of spleen response every 12 weeks whilst on treatment anticipated to be 52 weeks.
To evaluate preliminary signs of clinical efficacy of roginolisib when given in combination with ruxolitinib
Proportion of patients with transfusion independence at 12 and 24 weeks where applicable
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 determine the improvements of MF related symptoms when roginolisib is given in combination with ruxolitinib as assessed by the Myelofibrosis Symptom Assessment Form (MFSAF)
Proportion of patients who have any reduction in Total Symptom Score (TSS) at 12 and 24 weeks compared to baseline as measured by Myelofibrosis Symptom Assessment Form (MFSAF) Proportion of patients who have a reduction of ≥25% and ≥50% in TSS as measured by Myelofibrosis Symptom Assessment Form (MFSAF) Mean change in TSS as measured by Myelofibrosis Symptom Assessment Form (MFSAF) Time to the first ≥50% reduction compared to baseline in TSS as measured by Myelofibrosis Symptom Assessment Form (MFSAF) Duration of TSS response as measured by MFSAF (e.g., duration of TSS ≥25% and ≥50%)
Time frame: TTS and MFSAF measured at baseline and at 12 and 24 weeks.
To determine the pharmacokinetic (PK) parameters of roginolisib when given in combination with ruxolitinib to allow exposure/response and/or exposure/safety assessment
Concentration of roginolisib at pre-dose and steady state levels Area under the curve \[AUC\]
Time frame: Day 1 of each cycle whilst on treatment anticipated to be 52 weeks. Cycle length 28 days
To evaluate preliminary signs of clinical efficacy of roginolisib when given in combination with ruxolitinib
Overall Survival (OS) defined as the time from the date of the first dose of study treatment until death from any cause anticipated
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