Actively Recruiting
A Phase I/II Open-Label Multi-Centre Study to Assess Safety and Tolerability of Roginolisib with Ruxolitinib in Adults with Myelofibrosis Unresponsive to JAK Inhibitors
Led by iOnctura · Updated on 2026-04-27
26
Participants Needed
11
Research Sites
17 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating how the drug roginolisib works when combined with ruxolitinib in adults with myelofibrosis (MF) who have not responded adequately to prior JAK inhibitor treatments. This Phase I/II open-label study aims to assess the safety and tolerability of this combination treatment in patients with intermediate or high-risk MF. About 26 male and female patients aged 18 or older, who have been on stable doses of ruxolitinib for at least 3 months without significant spleen size reduction, will participate. Participants will receive roginolisib at a dose of 80 mg (equivalent to 72 mg roginolisib) alongside ruxolitinib up to 25 mg twice daily. The study starts with 13 patients in Part 1 to evaluate initial safety and potential benefits, followed by 13 more patients in Part 2 to further understand the effects of this combination. The treatment cycles are 28 days long, and the treatment period is expected to last up to 52 weeks. During the study, participants will have regular assessments including monitoring of adverse events, ECGs, laboratory tests, and blood pressure at specific days within each cycle. Researchers will also measure biomarker responses, spleen size reduction, symptom improvements, and drug levels in the blood. Participants will be followed for overall survival every 12 weeks for up to 96 weeks after enrollment of the last patient, with symptom assessments at baseline, 12 and 24 weeks. Safety and tolerability will be closely monitored throughout the study period.
CONDITIONS
Brief Title
A Study of Roginolisib in Combination With Ruxolitinib in Patients With Myelofibrosis (MF) Who Are Unresponsive to JAK Inhibitors
Who Can Participate
Eligibility Criteria
You may qualify if you...
- 18 years of age or older at the time of consent
- Able to give signed informed consent
- Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2
- Diagnosed with myelofibrosis, post-polycythaemia vera MF, or post-essential thrombocythemia MF
- DIPSS risk category of intermediate-1, intermediate-2, or high
- Treated with ruxolitinib for at least 3 months with a stable dose of 10 mg or more for at least 8 weeks before Day 1
- Unsatisfactory spleen reduction with less than 25% decrease and palpable spleen at least 10 cm below the left costal margin
- No experimental or other effective MF drug treatments within 3 months before study drug start, except ruxolitinib
- Active MF symptoms with Total Symptom Score (TSS) of 10 or higher at screening
- Peripheral blast count less than 10%
- Women must be non-childbearing or use effective pregnancy prevention; men must use effective contraception from screening through safety follow-up
You will not qualify if you...
- Unable to swallow food or any condition preventing oral medication
- History of severe Grade 3 or 4 adverse events unresponsive to treatment except fatigue; Grade 2 neuropathy or alopecia allowed
- Active autoimmune diseases requiring systemic treatment
- Abnormal ECG considered clinically meaningful, including QTc over 480 ms or left bundle branch block
- Active significant cardiovascular disease within 6 months prior to enrollment
- Active malignancy requiring treatment or recent malignancy without remission for at least 2 years
- Serious or uncontrolled medical disorders or active infections increasing study risk
- Use of erythropoiesis stimulating agents within 4 weeks before roginolisib
- Splenic irradiation within 3 months before roginolisib
- Major surgery within 2 weeks before first study dose
- Immune-suppressive treatment including corticosteroids over 10 mg/day within 14 days before first dose
- Live vaccine within 30 days before starting study therapy
- Known allergy to study drugs or ingredients
- Currently breastfeeding
- Known alcohol or substance abuse
- Lab values out of defined ranges including low neutrophils, platelets, hemoglobin, abnormal kidney or liver function, abnormal coagulation tests
- Active or latent tuberculosis
- Evidence of active hepatitis B, hepatitis C, or HIV unless well controlled on stable therapy
AI-Screening
AI-Powered Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 52 weeks
Participants receive roginolisib in combination with ruxolitinib to assess safety and tolerability while monitoring clinical and biomarker responses.
Visits on Day 1 and 15 of Cycle 1, and Day 1 of each subsequent 28-day cycle
Duration - Up to 96 weeks after last patient enrolled
Participants are followed for overall survival and long-term safety after treatment ends.
Visits every 12 weeks
Trial Site Locations
Total: 11 locations
1
Azienda Ospedaliero Universitaria Careggi Firenze, Struttura Complessa di Ematologia
Florence, Florence, Italy, 50134
Actively Recruiting
2
IRCCS Clinical Institute Humanitas
Rozzano, Milan, Italy, 20089
Actively Recruiting
3
Istituto di Ematolgia e Oncologia Medica
Bologna, Italy, 40138
Actively Recruiting
4
Azienda Sanitario Universitaria Friuli Centrale
Udine, Italy, 33100
Actively Recruiting
5
Vall d'Hebron University Hospital
Barcelona, Spain, 08035
Actively Recruiting
6
Avigunda Gran via de l'Hospitalet 199-203, 08908 L'Hospitalet de Llobregat
Barcelona, Spain, 08908
Actively Recruiting
7
START Madrid CIOCC
Madrid, Spain, 28050
Not Yet Recruiting
8
Hospital Universitario de Salamanca
Salamanca, Spain, 37007
Actively Recruiting
9
United Lincolnshire Teach Hospitals NHS Trust, Pilgrim Hospital Boston
Boston, Lincolnshire, United Kingdom, PE21 9QS
Not Yet Recruiting
10
Belfast City Hospital
Belfast, United Kingdom, BT9&AB
Not Yet Recruiting
11
Guy´s and St. Thomas NHS Foundation Trust
London, United Kingdom, SE1 9RT
Not Yet Recruiting
Research Team
T
Tracey Hammett, RN
K
Karen Tonge
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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