A Phase 1 Study of AJ1-11095 in Patients With Primary Myelofibrosis (PMF), Post-Polycythemia Vera Myelofibrosis (PPV-MF), or Post-Essential Thrombocythemia Myelofibrosis (PET-MF) Who Have Been Failed by a Type I JAK2 Inhibitor (JAK2i)

ID#: NCT06343805

Age: 18 years - 66+

Gender: All

Healthy Subjects: No

Study Phase: Phase 1

Recruitment Status: Recruiting

Start Date: October 01, 2024

End Date: February 15, 2027

Contact Information:
David Steensma, M.D.
917-410-7250
Summary: 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.
Eligibility:

Inclusion Criteria:

1. 18 years of age or older.

2. Diagnosis of PMF, post-PV MF, or post-ET MF.

3. DIPSS Intermediate-2 or High-risk MF with ≤10% blasts, regardless of JAK2 mutation status.

4. Estimated spleen volume ≥450cm3.

5. MFSAF v.4.0 TSS ≥10, or at least 2 of 7 MFSAF-assessed symptoms with scores ≥3.

6. ECOG PS of 0, 1, 2, or 3.

7. Prior therapy with at least 1 type I JAK2 inhibitor, and either failed to achieve a response or relapsed after achieving a response.

8. ANC ≥1.0×10^9/L.

9. Platelet count ≥75×10^9/L.

10. eGFR ≥45 mL/min/1.73m2.

11. Serum total bilirubin ≤2.0 × upper limit of normal (ULN).

12. AST and ALT ≤3.0 × ULN.

13. QTcF ≤480 msec.

Exclusion Criteria:

1. Prior splenectomy.

2. Splenic irradiation within 3 months prior to first dose of study drug.

3. Ongoing use of systemic corticosteroids at dose equivalent to >10mg/day of prednisone.

4. Uncontrolled intercurrent illness such as an acute infection.

5. Chronic active or acute hepatitis B or C infection.

6. Chemotherapy in the previous 4 weeks prior to first dose of study drug (Hydrea is permitted until 5 days before starting protocol therapy).

7. Use of a JAK2 inhibitor in the previous 10 days.

8. Use of erythropoiesis stimulating agents (unless stable for >8 weeks).

9. Peripheral neuropathy ≥ Grade 2 (NCI CTCAE v 5.0).

10. Unable or unwilling to undergo CT or MRI for spleen size imaging.

11. Pregnant or breastfeeding.

12. Requirement for therapy with a medication that is a strong CYP3A4 inhibitor as a concomitant medication.