Ignite Creation Date:
2025-12-25 @ 3:21 AM
Ignite Modification Date:
2026-01-13 @ 5:14 PM
Study NCT ID:
NCT06343805
Status:
RECRUITING
Last Update Posted:
2025-12-24
First Post:
2024-03-13
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
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)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D055728', 'term': 'Primary Myelofibrosis'}], 'ancestors': [{'id': 'D009196', 'term': 'Myeloproliferative Disorders'}, {'id': 'D001855', 'term': 'Bone Marrow Diseases'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SEQUENTIAL', 'interventionModelDescription': 'This dose escalation study will follow a 3+3 cohort design until the RP2D and/or MTD is determined.'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 76}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2024-10-23', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-12', 'completionDateStruct': {'date': '2027-02-15', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-12-18', 'studyFirstSubmitDate': '2024-03-13', 'studyFirstSubmitQcDate': '2024-03-26', 'lastUpdatePostDateStruct': {'date': '2025-12-24', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2024-04-03', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2026-10-15', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of patients with treatment-emergent adverse events as assessed by CTCAE v 5.0.', 'timeFrame': 'Baseline through study completion, an average of 1 year', 'description': 'Treatment Emergent AEs will be assessed during routine study visits and compared to Baseline to continuously evaluate safety and tolerability of AJ1-11095.'}, {'measure': 'Number of patients with Dose Limiting Toxicities (DLTs)', 'timeFrame': 'Baseline through study completion, an average of 1 year', 'description': 'Protocol-defined potential DLTs will be assessed by the Safety Review Committee at routine intervals.'}, {'measure': 'To establish the maximum tolerated dose (MTD) and/or recommended phase 2 dose (RP2D) of AJ1-11095', 'timeFrame': 'Baseline through study completion, an average of 1 year', 'description': '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.'}], 'secondaryOutcomes': [{'measure': 'To assess clinical response to AJ1-11095 evaluated by the Total Symptom Score (TSS).', 'timeFrame': 'Baseline through Week 24', 'description': '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.'}, {'measure': 'To assess clinical response to AJ1-11095 evaluated by spleen volume assessments.', 'timeFrame': 'Baseline through Week 24', 'description': 'Spleen volume reduction (SVR) of ≥35% from Baseline measured by magnetic resonance imaging (MRI) or computed tomography (CT).'}, {'measure': 'To assess clinical response to AJ1-11095 evaluated by spleen length assessments.', 'timeFrame': 'Baseline through Week 24', 'description': 'Proportion of subjects with ≥50% reduction in length of spleen assessed by palpation.'}, {'measure': 'To assess clinical response to AJ1-11095 evaluated through spleen size improvement.', 'timeFrame': 'Baseline through Week 24', 'description': 'Time to spleen size improvement response measured by patient and across all patients.'}, {'measure': 'To evaluate the Area Under the Curve (AUC) of AJ1-11095', 'timeFrame': 'Pre dose and post dose Cycle 1 (Day 1, and Day 2 (24hrs post), Day 8, 15, 22, and Cycle 2 (Day 1 and 24 hrs post).', 'description': 'AUC time curve from 0 to 24 hrs post dose and percent difference between intervals will be evaluated.'}, {'measure': 'To evaluate the Cmax of AJ1-11095', 'timeFrame': 'Pre dose and post dose Cycle 1 (Day 1, and Day 2 (24hrs post), Day 8, 15, 22, and Cycle 2 (Day 1 and 24 hrs post).', 'description': 'The maximum observed plasma concentration will be evaluated.'}, {'measure': 'To evaluate the Tmax of AJ1-11095', 'timeFrame': 'Pre dose and post dose Cycle 1 (Day 1, and Day 2 (24hrs post), Day 8, 15, 22, and Cycle 2 (Day 1 and 24 hrs post).', 'description': 'The duration of time taken to reach Cmax will be evaluated.'}, {'measure': 'To evaluate the half-life of AJ1-11095', 'timeFrame': 'Pre dose and post dose Cycle 1 (Day 1, and Day 2 (24hrs post), Day 8, 15, 22, and Cycle 2 (Day 1 and 24 hrs post).', 'description': 'The depletion of AJ1-00195 in the body will be observed over time.'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Primary Myelofibrosis', 'Post-Essential Thrombocythemia Myelofibrosis', 'Post-Polycythemia Vera Myelofibrosis', 'PMF', 'PPV-MF', 'PET-MF']}, 'descriptionModule': {'briefSummary': '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.', 'detailedDescription': '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.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. 18 years of age or older.\n2. Diagnosis of PMF, post-PV MF, or post-ET MF.\n3. DIPSS Intermediate-2 or High-risk MF with ≤10% blasts, regardless of JAK2 mutation status.\n4. Estimated spleen volume ≥450cm3.\n5. MFSAF v.4.0 TSS ≥10, or at least 2 of 7 MFSAF-assessed symptoms with scores ≥3.\n6. ECOG PS of 0, 1, 2, or 3.\n7. Prior therapy with at least 1 type I JAK2 inhibitor, and either failed to achieve a response or relapsed after achieving a response.\n8. ANC ≥1.0×10\\^9/L.\n9. Platelet count ≥75×10\\^9/L.\n10. eGFR ≥45 mL/min/1.73m2.\n11. Serum total bilirubin ≤2.0 × upper limit of normal (ULN).\n12. AST and ALT ≤3.0 × ULN.\n13. QTcF ≤480 msec.\n\nExclusion Criteria:\n\n1. Prior splenectomy.\n2. Splenic irradiation within 3 months prior to first dose of study drug.\n3. Ongoing use of systemic corticosteroids at dose equivalent to \\>10mg/day of prednisone.\n4. Uncontrolled intercurrent illness such as an acute infection.\n5. Chronic active or acute hepatitis B or C infection.\n6. Chemotherapy in the previous 4 weeks prior to first dose of study drug (Hydrea is permitted until 5 days before starting protocol therapy).\n7. 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).\n8. Use of erythropoiesis stimulating agents (unless stable for \\>8 weeks).\n9. Peripheral neuropathy ≥ Grade 2 (NCI CTCAE v 5.0).\n10. Unable or unwilling to undergo CT or MRI for spleen size imaging.\n11. Pregnant or breastfeeding.\n12. Requirement for therapy with a medication that is a strong CYP3A4 inhibitor as a concomitant medication.'}, 'identificationModule': {'nctId': 'NCT06343805', 'briefTitle': '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)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Ajax Therapeutics, Inc.'}, 'officialTitle': 'A Multicenter, Open-Label, Phase 1 Study of AJ1-11095 Administered as Oral Monotherapy 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)', 'orgStudyIdInfo': {'id': 'AJX-101'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Cohort 1', 'description': 'Dose A of AJ1-11095 taken orally by patients.', 'interventionNames': ['Drug: AJ1-11095']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 2', 'description': 'Dose B of AJ1-11095 taken orally by patients.', 'interventionNames': ['Drug: AJ1-11095']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 3', 'description': 'Dose C of AJ1-11095 taken orally by patients.', 'interventionNames': ['Drug: AJ1-11095']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 4', 'description': 'Dose D of AJ1-11095 taken orally by patients.', 'interventionNames': ['Drug: AJ1-11095']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 5', 'description': 'Dose E of AJ1-11095 taken orally by patients.', 'interventionNames': ['Drug: AJ1-11095']}, {'type': 'EXPERIMENTAL', 'label': 'Dose Expansion Cohort 1', 'description': 'Candidate RP2D of AJ1-11095 taken orally by patients.', 'interventionNames': ['Drug: AJ1-11095']}, {'type': 'EXPERIMENTAL', 'label': 'Dose Expansion Cohort 2', 'description': 'Alternative candidate RP2D of AJ1-11095 taken orally by patients.', 'interventionNames': ['Drug: AJ1-11095']}], 'interventions': [{'name': 'AJ1-11095', 'type': 'DRUG', 'description': 'Type II JAK2 Inhibitor', 'armGroupLabels': ['Cohort 1', 'Cohort 2', 'Cohort 3', 'Cohort 4', 'Cohort 5', 'Dose Expansion Cohort 1', 'Dose Expansion Cohort 2']}]}, 'contactsLocationsModule': {'locations': [{'zip': '94304', 'city': 'Palo Alto', 'state': 'California', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'William Shomali, MD', 'role': 'CONTACT', 'phone': '650-498-6000'}], 'facility': 'Stanford Cancer Institute', 'geoPoint': {'lat': 37.44188, 'lon': -122.14302}}, {'zip': '33612', 'city': 'Tampa', 'state': 'Florida', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Andrew Kuykendall, MD', 'role': 'CONTACT', 'phone': '813-745-4639'}], 'facility': 'Moffitt Cancer Cancer Center', 'geoPoint': {'lat': 27.94752, 'lon': -82.45843}}, {'zip': '66160', 'city': 'Kansas City', 'state': 'Kansas', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Abdulraheem Yacoub, MD', 'role': 'CONTACT'}], 'facility': 'University of Kansas Medical Center', 'geoPoint': {'lat': 39.11417, 'lon': -94.62746}}, {'zip': '02114', 'city': 'Boston', 'state': 'Massachusetts', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Gabriela Hobbs, MD', 'role': 'CONTACT', 'phone': '617-724-1124'}], 'facility': 'Massachusetts General Hospital', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '02115', 'city': 'Boston', 'state': 'Massachusetts', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Jacqueline Garcia, MD', 'role': 'CONTACT', 'phone': '617-632-1906'}], 'facility': 'Dana Farber Cancer Institute', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '48109', 'city': 'Ann Arbor', 'state': 'Michigan', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Kristen Pettit, MD', 'role': 'CONTACT', 'phone': '734-647-2829'}], 'facility': 'University of Michigan', 'geoPoint': {'lat': 42.27756, 'lon': -83.74088}}, {'zip': '63110', 'city': 'St Louis', 'state': 'Missouri', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Stephen Oh, MD', 'role': 'CONTACT', 'phone': '314-362-8814'}], 'facility': 'Washington University School of Medicine', 'geoPoint': {'lat': 38.62727, 'lon': -90.19789}}, {'zip': '10021', 'city': 'New York', 'state': 'New York', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Raajit Rampal, MD', 'role': 'CONTACT'}], 'facility': 'David H. Koch Center for Cancer Care at Memorial Sloan Kettering', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '10029', 'city': 'New York', 'state': 'New York', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'John Mascarenhas, MD', 'role': 'CONTACT', 'phone': '212-241-8839'}], 'facility': 'Icahn School of Medicine at Mount Sinai', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '28204', 'city': 'Charlotte', 'state': 'North Carolina', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Michael Grunwald, MD', 'role': 'CONTACT', 'phone': '980-442-4363'}], 'facility': 'Levine Cancer Institute', 'geoPoint': {'lat': 35.22709, 'lon': -80.84313}}, {'zip': '45221', 'city': 'Cincinnati', 'state': 'Ohio', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Eric Vick, MD', 'role': 'CONTACT', 'phone': '513-213-3203'}], 'facility': 'University of Cincinnati', 'geoPoint': {'lat': 39.12711, 'lon': -84.51439}}, {'zip': '43210', 'city': 'Columbus', 'state': 'Ohio', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Uma Borate, MD', 'role': 'CONTACT', 'phone': '614-685-9828'}], 'facility': 'The Ohio State University Comprehensive Cancer Center', 'geoPoint': {'lat': 39.96118, 'lon': -82.99879}}, {'zip': '77030', 'city': 'Houston', 'state': 'Texas', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Prithviraj Bose, MD', 'role': 'CONTACT', 'phone': '713-792-7747'}], 'facility': 'MD Anderson Cancer Center', 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}], 'centralContacts': [{'name': 'David Steensma, M.D.', 'role': 'CONTACT', 'email': 'david@ajaxtherapeutics.com', 'phone': '917-410-7250'}], 'overallOfficials': [{'name': 'John Mascarenhas, M.D.', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Mt. Sinai'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Ajax Therapeutics, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}