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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)

NCT ID: NCT06343805

Last Updated: 2025-12-24

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1

Total Enrollment

76 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-10-23

Study Completion Date

2027-02-15

Brief Summary

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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.

Detailed Description

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This is a phase 1, non-randomized, open-label study utilizing a 3+3 sequential dose escalation design followed by an expansion phase. The primary objective will be to evaluate the safety and tolerability of AJ1-11095, and establish a Maximally Tolerated Dose (MTD) and/or inform the establishment of a candidate Recommended Phase 2 dose (RP2D). The RP2D may be the maximally tolerated dose (MTD) or may be a dose below the MTD. The candidate RP2D will be based on AE pattern, PK and biomarker information, in addition to all available safety and efficacy data. Expansion cohorts will be enrolled to gather additional safety and efficacy information and to further refine input for future RP2D discussions. Eligible participants will have PMF, PPV-MF or PET-MF and will have either have relapsed after a response, or be refractory to, at least one prior type I JAK2 inhibitor therapy, either administered as monotherapy or in combination with another drug.

Conditions

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Primary Myelofibrosis Post-Essential Thrombocythemia Myelofibrosis Post-Polycythemia Vera Myelofibrosis PMF PPV-MF PET-MF

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

This dose escalation study will follow a 3+3 cohort design until the RP2D and/or MTD is determined.
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Cohort 1

Dose A of AJ1-11095 taken orally by patients.

Group Type EXPERIMENTAL

AJ1-11095

Intervention Type DRUG

Type II JAK2 Inhibitor

Cohort 2

Dose B of AJ1-11095 taken orally by patients.

Group Type EXPERIMENTAL

AJ1-11095

Intervention Type DRUG

Type II JAK2 Inhibitor

Cohort 3

Dose C of AJ1-11095 taken orally by patients.

Group Type EXPERIMENTAL

AJ1-11095

Intervention Type DRUG

Type II JAK2 Inhibitor

Cohort 4

Dose D of AJ1-11095 taken orally by patients.

Group Type EXPERIMENTAL

AJ1-11095

Intervention Type DRUG

Type II JAK2 Inhibitor

Cohort 5

Dose E of AJ1-11095 taken orally by patients.

Group Type EXPERIMENTAL

AJ1-11095

Intervention Type DRUG

Type II JAK2 Inhibitor

Dose Expansion Cohort 1

Candidate RP2D of AJ1-11095 taken orally by patients.

Group Type EXPERIMENTAL

AJ1-11095

Intervention Type DRUG

Type II JAK2 Inhibitor

Dose Expansion Cohort 2

Alternative candidate RP2D of AJ1-11095 taken orally by patients.

Group Type EXPERIMENTAL

AJ1-11095

Intervention Type DRUG

Type II JAK2 Inhibitor

Interventions

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AJ1-11095

Type II JAK2 Inhibitor

Intervention Type DRUG

Eligibility Criteria

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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 Type I JAK2 inhibitor must have been discontinued for at least 5 days or 5 half-lives prior to dosing (whichever is longer).
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.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Ajax Therapeutics, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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John Mascarenhas, M.D.

Role: PRINCIPAL_INVESTIGATOR

Mt. Sinai

Locations

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Stanford Cancer Institute

Palo Alto, California, United States

Site Status RECRUITING

Moffitt Cancer Cancer Center

Tampa, Florida, United States

Site Status RECRUITING

University of Kansas Medical Center

Kansas City, Kansas, United States

Site Status RECRUITING

Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status RECRUITING

Dana Farber Cancer Institute

Boston, Massachusetts, United States

Site Status RECRUITING

University of Michigan

Ann Arbor, Michigan, United States

Site Status RECRUITING

Washington University School of Medicine

St Louis, Missouri, United States

Site Status RECRUITING

David H. Koch Center for Cancer Care at Memorial Sloan Kettering

New York, New York, United States

Site Status RECRUITING

Icahn School of Medicine at Mount Sinai

New York, New York, United States

Site Status RECRUITING

Levine Cancer Institute

Charlotte, North Carolina, United States

Site Status RECRUITING

University of Cincinnati

Cincinnati, Ohio, United States

Site Status RECRUITING

The Ohio State University Comprehensive Cancer Center

Columbus, Ohio, United States

Site Status RECRUITING

MD Anderson Cancer Center

Houston, Texas, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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David Steensma, M.D.

Role: CONTACT

Phone: 917-410-7250

Email: [email protected]

Facility Contacts

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William Shomali, MD

Role: primary

Andrew Kuykendall, MD

Role: primary

Abdulraheem Yacoub, MD

Role: primary

Gabriela Hobbs, MD

Role: primary

Jacqueline Garcia, MD

Role: primary

Kristen Pettit, MD

Role: primary

Stephen Oh, MD

Role: primary

Raajit Rampal, MD

Role: primary

John Mascarenhas, MD

Role: primary

Michael Grunwald, MD

Role: primary

Eric Vick, MD

Role: primary

Uma Borate, MD

Role: primary

Prithviraj Bose, MD

Role: primary

Other Identifiers

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AJX-101

Identifier Type: -

Identifier Source: org_study_id