Actively Recruiting
Study to Assess Safety and Preliminary Efficacy of Orally Administered JBI-802 in Subjects With Myeloproliferative Neoplasms (MPN) and Myelodysplastic/Myeloproliferative Neoplasms (MDS/MPN) With Thrombocytosis
Led by Jubilant Therapeutics Inc. · Updated on 2026-06-03
30
Participants Needed
7
Research Sites
13 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and preliminary effectiveness of JBI-802, an oral drug, in adults diagnosed with Myeloproliferative Neoplasms (MPN) and Myelodysplastic/Myeloproliferative Neoplasms (MDS/MPN) who have thrombocytosis. This phase 1/2 open-label study aims to find the best dose that is safe and tolerable and then assess how well the drug works for these conditions. JBI-802 targets specific enzymes involved in gene regulation and cancer development, and has shown promising preclinical results. Participants will take JBI-802 capsules daily on an empty stomach in 28-day treatment cycles for up to 2 years, or until the drug is no longer tolerated, disease progresses, or other reasons lead to stopping treatment. The study includes a dose escalation phase using a 3+3 design starting at 5 mg/day, with possible dose increases or decreases based on safety and tolerability. After establishing the recommended dose, an expansion phase will enroll more participants at that dose to further evaluate safety and efficacy. During the study, participants will undergo assessments including medical history reviews, physical exams, vital signs, ECGs, symptom questionnaires, imaging scans, bone marrow biopsies, and various blood and urine tests. Safety and side effects will be monitored at the start and end of each cycle, and participants will keep a dosing diary to track compliance. Follow-up visits will continue after treatment ends, and compassionate use of JBI-802 may be considered after study completion based on clinical judgment and availability.
CONDITIONS
Brief Title
Phase 1/ Phase 2 Study to Assess Safety and Efficacy of Orally Administered JBI-802 in Subjects With Myeloproliferative Neoplasms (MPN) and Myelodysplastic/Myeloproliferative Neoplasms (MDS/MPN) With Thrombocytosis
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or female subjects aged 18 years or older at screening.
- Diagnosed with Essential Thrombocythemia (ET) requiring treatment to lower platelet count based on age over 60 or history of thrombosis.
- Diagnosed with morphologically confirmed MDS/MPN neoplasms excluding JMML, CMML, and aCML, relapsed, refractory, or intolerant to standard care with no available therapies.
- Diagnosed with MDS/MPN unclassifiable or MDS/MPN-RS-T.
- For dose expansion: diagnosed with ET, Polycythemia Vera (PV), or pre-fibrotic myelofibrosis relapsed or refractory to standard care.
- Failed at least one standard therapy or intolerant to standard care.
- Discontinued prior therapy at least 1 week before study drug (4 weeks for interferon).
- Screening labs within defined ranges for hemoglobin, neutrophil count, platelet count, liver function, kidney function, and coagulation.
- Resolved prior therapy toxicities to grade 0 or 1 except specified exceptions.
- ECOG performance status of 2 or less.
- Able to swallow oral medication and comply with study requirements.
- Willing to undergo bone marrow biopsy during screening and study.
- Agree to use effective contraception during treatment and for 3 months after last dose.
You will not qualify if you...
- Received systemic anticancer, biological, or investigational therapy within 2 weeks or 5 half-lives prior to study drug.
- Underwent hematopoietic stem cell transplantation within 60 days or currently on immunosuppressive therapy post-transplant with active graft-versus-host disease.
- Had major surgery within 21 days before study drug or not recovered from surgery effects.
- Surgery or condition affecting absorption of medication.
- Radiotherapy within 2 weeks before study drug or not recovered from related toxicities.
- Known malignant central nervous system disease except stable treated brain metastases.
- Severe or unstable medical conditions including certain heart diseases, uncontrolled hypertension or diabetes, psychiatric conditions, or recent myocardial infarction.
- Congenital long QT syndrome or prolonged QT interval.
- History of other cancers interfering with study assessments except in remission or localized skin cancer.
- Received live vaccines within 30 days before study drug.
- Use of glucocorticoids except for specific indications.
- Initiation of bisphosphonates or RANKL inhibitors after consent.
- Prophylactic use of antidiarrheals, antiemetics, anti-inflammatory, antipyretic drugs, or colony-stimulating factors before first dose.
- Use of strong inhibitors or inducers of CYP3A or CYP2D6 within specified timeframes before dosing.
- Active HIV, hepatitis B or C infection.
- Active gastrointestinal diseases affecting drug absorption.
- Acute illness or active infection requiring systemic antibiotics within 14 days before dosing.
- Pregnant, breastfeeding, or planning pregnancy during study and 90 days after last dose.
- Participation in another investigational study.
- COVID vaccine within 7 days before dosing.
- Prior treatment with JBI-802.
- Any condition posing unacceptable risk or limiting participation as judged by Investigator.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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 2 years
Participants take the study drug JBI-802 orally once daily in 28-day cycles. Dose escalation starts at 5 mg daily and may be adjusted based on tolerability. Treatment may continue up to 2 years as long as clinical benefit is observed without unacceptable side effects.
1 baseline visit at start of treatment cycle 1, then visits every 28 days for each treatment cycle
Duration - 28 days after last dose
Participants are monitored for safety after treatment ends to observe any late or ongoing effects.
1 visit (in-person)
Duration - Up to approximately 2 years after treatment ends
Participants are followed with visits every 3 months to monitor overall survival status.
Visits every 3 months
Trial Site Locations
Total: 7 locations
1
St. Vincent's Hospital Sydney Limited
Sydney, New South Wales, Australia, 2010
Actively Recruiting
2
Macquarie University
Sydney, New South Wales, Australia, 2109
Actively Recruiting
3
Royal Adelaide Hospital
Adelaide, South Australia, Australia, 5000
Actively Recruiting
4
St Vincent's Hospital (Melbourne)
Melbourne, Victoria, Australia, 3065
Actively Recruiting
5
Monash Medical Centre
Melbourne, Victoria, Australia, 3168
Actively Recruiting
6
The Perth Blood Institute Limited
Perth, Western Australia, Australia, 6000
Actively Recruiting
7
Hollywood Private Hospital
Nedlands, Australia, 6009
Actively Recruiting
Research Team
M
Melda Dolan
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
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