AJX-101 is a first-in-human (FIH), phase 1, non-randomized, multi-center, open-label clinical trial designed to investigate the safety, tolerability, pharmacokinetics (PK), clinical activity and changes in biomarkers of an orally administered type II JAK2 inhibitor, AJ1-11095, in subjects with primary or secondary myelofibrosis previously treated with at least one type I JAK2 inhibitor.
18 years of age or older.
Diagnosis of PMF, post-PV MF, or post-ET MF.
DIPSS Intermediate-2 or High-risk MF with ≤10% blasts, regardless of JAK2 mutation status.
Estimated spleen volume ≥450cm3.
Prior splenectomy.
Splenic irradiation within 3 months prior to first dose of study drug.
Ongoing use of systemic corticosteroids at dose equivalent to >10mg/day of prednisone.
Uncontrolled intercurrent illness such as an acute infection.
Sequential
AJ1-11095
DrugType II JAK2 Inhibitor
7
Treatment groupsSee each treatment group below.
Number of patients with treatment-emergent adverse events as assessed by CTCAE v 5.0.
Treatment Emergent AEs will be assessed during routine study visits and compared to Baseline to continuously evaluate safety and tolerability of AJ1-11095.
Number of patients with Dose Limiting Toxicities (DLTs)
Protocol-defined potential DLTs will be assessed by the Safety Review Committee at routine intervals.
To establish the maximum tolerated dose (MTD) and/or recommended phase 2 dose (RP2D) of AJ1-11095
Safety evaluations will occur consistently for each patient and across patients to assess MTD or RP2D. See description of safety evaluations described in outcomes 1 and 2 mentioned above.
To assess clinical response to AJ1-11095 evaluated by the Total Symptom Score (TSS).
Number and proportion of patients with an improvement of ≥50% from Baseline in Total TSS as well as time to TSS response and duration of TSS response using the Myelofibrosis Symptom Assessment Form (MFSAF) v4.0. The TSS is a 7 question assessment form with lower scores indicating better outcomes.
To assess clinical response to AJ1-11095 evaluated by spleen volume assessments.
Spleen volume reduction (SVR) of ≥35% from Baseline measured by magnetic resonance imaging (MRI) or computed tomography (CT).
To assess clinical response to AJ1-11095 evaluated by spleen length assessments.
Proportion of subjects with ≥50% reduction in length of spleen assessed by palpation.
To assess clinical response to AJ1-11095 evaluated through spleen size improvement.
Time to spleen size improvement response measured by patient and across all patients.
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