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ScholarsBetaWeill Cornell Medicine

Overview

Dr. Roboz is directly involved in developing and implementing clinical trials of novel regimens in acute myeloid leukemia, acute lymphoid leukemia, myelodysplastic syndrome, chronic myeloid leukemia and myeloproliferative disorders.

Highlights

  1. 1
    The New England journal of medicine · 2020
    GJGail J Roboz
    PMID: 33369355DOISenior author409 citations
  2. 2
    Blood · 2020
    GJGail J Roboz
    PMID: 31841594PMCID: PMC7019193DOIFirst author351 citations
  3. 3
    Blood · 2022
    GJGail J Roboz
    PMID: 34995344DOIFirst author68 citations

Publications

280 publications

Funding

9 grants

Active

2 grants
  • Co-I
    Pre-malignant mutation landscape and risk factors for progression to hematologic cancers
    National Cancer Institute · 2021–2027
    R01CA248747-04
  • PI
    Janssen Biopharma Agreement
    Janssen Biotech Inc. · 2020–2025BioPharma Alliance
    BP 212999-03
Completed grants (7)
  • PI
    The VALIDATE study
    Yale University · 2022–2025Contract
    SA 242085-01
  • Co-I
    3D co-culture system to characterize and target leukemia stem cells
    National Cancer Institute · 2020–2024
    R21CA245454-01A1
  • Co-I
    Integrative Single-cell Analysis of Hematological Malignancies
    Daiichi Sankyo Co., Ltd. · 2018–2020Contract
    DS 192276-01
  • Co-I
    Preclinical evaluation of UCART (allogeneic engineered CAR-T Cells) activity against primary Acute Myeloid Leukemia (AML) samples (CLL Agreement)
    Cellectis, Inc · 2019–2020Contract
    BP 193410-01
  • Co-I
    Pharmacological Modulation of Epigenetic Changes in AML
    National Cancer Institute · 2013–2018 · via Beckman Research Institute of the City of Hope· 2 renewals
    R01CA102031-10
  • KP
    Delineating Clinical Response with Single-Cell Omics
    AbbVie Inc. · 2016–2017Contract
    Abbvie RAA
  • KP
    Identification of Novel Therapies to Ablate Chemoresistant Myeloid Leukemia Stem Cells during Remission
    Office of the Director, NIH · 2010–2015
    DP2OD007399-05

Clinical research

49 studies

Active

6 studies
  • PI
    Compassionate Use of Zelavespib (PU-H71) for Treatment of Relapsed/Refractory Acute Myeloid Leukemia (AML) in Patient C-B
    Weill Medical College of Cornell University · OPEN TO ACCRUAL (as of 2026)
  • PI
    Phase 3 Randomized, Double-blind, Placebo-controlled Studies Assessing Ziftomenib in Combination With Either Standard of Care Nonintensive (Venetoclax+Azacitidine) or Intensive (7+3) Therapy in Patients With Untreated NPM1 Mutated or KMT2A Rearranged Acute Myeloid Leukemia
    PHASE3 · Kura Oncology · OPEN TO ACCRUAL (as of 2026)
    Acute Myeloid Leukemia (AML)
    NCT07007312
  • PI
    A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Study of Bleximenib, Venetoclax and Azacitidine for the Treatment of Participants With Newly Diagnosed Acute Myeloid Leukemia Harboring KMT2A Rearrangements or NPM1 Mutations Who Are Ineligible for Intensive Chemotherapy
    PHASE3 · Janssen Research & Development, LLC · OPEN TO ACCRUAL (as of 2025)
    Leukemia, Myeloid, Acute
    NCT06852222
  • PI
    Phase 1 Study to Determine the Safety and Tolerability of Ziftomenib Combinations for the Treatment of KMT2A-rearranged or NPM1-mutant Relapsed/Refractory Acute Myeloid Leukemia
    PHASE1 · Kura Oncology · OPEN TO ACCRUAL (as of 2025)
    AML; AML With Mutated NPM1; Hematologic Malignancy; KMT2Ar; NPM1 Mutation; MLL Rearrangement; Leukemia; Acute Myeloid Leukemia; Leukemia, Myeloid; Leukemia, Myeloid, Acute; Acute Leukemia; Neoplasms by Histologic Type
    NCT06001788
  • PI
    Phase 1 Study of Venetoclax/Azacitidine or Venetoclax in Combination With Ziftomenib or Standard Induction Cytarabine/Daunorubicin (7+3) Chemotherapy in Combination With Ziftomenib for the Treatment of Patients With Acute Myeloid Leukemia
    PHASE1 · Kura Oncology · OPEN TO ACCRUAL (as of 2023)
    Acute Myeloid Leukemia; Mixed Lineage Leukemia Gene Mutation; Refractory AML; AML With Mutated NPM1; Acute Myeloid Leukemia Recurrent; Acute Myeloid Leukemia, in Relapse; NPM1 Mutation; KMT2Ar; Myeloid Sarcoma; Nucleophosmin 1-mutated Acute Myeloid Leukemia
    NCT05735184
  • PI
    The VALIDATE Study - A Multicenter, International, Retrospective Study in Patients with MDS and AML Who Have Received HMA-based Therapy
    Yale University · OPEN TO ACCRUAL (as of 2022)
Completed & closed trials (43)
  • PI
    A Phase 1/2, Multicenter, Open-Label, Randomized Dose Ranging and Expansion Study of the Combination of Gilteritinib, Venetoclax and Azacitidine in Patients With Newly Diagnosed FLT3 Mutated Acute Myeloid Leukemia (AML) Not Eligible for Intensive Induction Chemotherapy
    PHASE1; PHASE2 · Astellas Pharma Inc. · CLOSED TO ACCRUAL (as of 2026)
    Acute Myeloid Leukemia (AML); FLT3-mutated Acute Myeloid Leukemia
    NCT05520567
  • PI
    A Phase I/Ib Study of BP1002 (a Liposomal Bcl-2 Antisense Oligodeoxynucleotide) in Patients With Refractory/Relapsed Acute Myeloid Leukemia (AML)
    PHASE1 · Bio-Path Holdings, Inc. · IRB STUDY CLOSURE (as of 2026)
    Acute Myeloid Leukemia, in Relapse; Acute Myeloid Leukemia Refractory
    NCT05190471
  • PI
    A Phase 1/2 First in Human Study of the Menin-MLL(KMT2A) Inhibitor KO-539 in Patients With Relapsed or Refractory Acute Myeloid Leukemia
    PHASE1; PHASE2 · Kura Oncology · IRB STUDY CLOSURE (as of 2026)
    Advanced Malignant Neoplasm; Acute Myeloid Leukemia; Mixed Lineage Leukemia; Mixed Lineage Acute Leukemia; Acute Leukemia of Ambiguous Lineage; Mixed Phenotype Acute Leukemia; Acute Lymphoblastic Leukemia
    NCT04067336
  • PI
    Multicenter, Open-label, Randomized, Phase 2 Study of Venetoclax and Azacitidine Plus Cusatuzumab Versus Venetoclax and Azacitidine Alone in Newly Diagnosed AML Patients Who Are Not Candidates for Intensive Therapy
    PHASE2 · OncoVerity, Inc. · CLOSED TO ACCRUAL (as of 2026)
    Leukemia, Myeloid, Acute
    NCT06384261
  • PI
    Long-Term Follow-up Study (LTFS): An Observational Long-term Follow-up Study of Patients Who Received Prior Caribou Cell Therapy
    Caribou Biosciences, Inc. · IRB STUDY CLOSURE (as of 2026)
  • PI
    Venetoclax In Combination With ASTX727, an All-ORal TherapY for Chronic Myelomonocytic Leukemia and Other MDS/MPN With Excess Blasts (VICTORY-MDS/MPN): a Randomized, Phase 2 Trial
    PHASE2 · National Cancer Institute · IRB STUDY CLOSURE (as of 2026)
    Chronic Myelomonocytic Leukemia; Myelodysplastic Syndrome; Myelodysplastic Syndrome With Excess Blasts; Myelodysplastic/Myeloproliferative Neoplasm; Myeloproliferative Neoplasm
    NCT05600894
  • PI
    The ASH Research Collaborative Data Hub COVID-19 Registry for Hematologic Malignancy Protocol
    American Society of Hematology Inc · IRB STUDY CLOSURE (as of 2025)
  • PI
    Phase I, Open Label Dose Escalation and Dose-Expansion Study to Evaluate the Safety, Expansion, Persistence, and Clinical Activity of UCART123 (Allogeneic Engineered T-cells Expressing Anti-CD123 Chimeric Antigen Receptor), Administered in Patients With Relapsed/Refractory Acute Myeloid Leukemia
    PHASE1 · Cellectis, Inc · IRB STUDY CLOSURE (as of 2025)
    Relapsed/Refractory Acute Myeloid Leukemia
    NCT03190278
  • PI
    A Phase 1 First-in-Human Study Evaluating Safety, Pharmacokinetics and Efficacy of ABBV-787 in Adult Subjects With Acute Myeloid Leukemia (AML)
    PHASE1 · AbbVie Inc. · IRB STUDY CLOSURE (as of 2025)
    Acute Myeloid Leukemia
    NCT06068868
  • PI
    A Phase 1, Multicenter, Open-Label Study of CB-012, a CRISPR-Edited Allogeneic Anti-CLL-1 CAR-T Cell Therapy in Patients With Relapsed/Refractory Acute Myeloid Leukemia
    PHASE1 · Caribou Biosciences, Inc. · IRB STUDY CLOSURE (as of 2025)
    Acute Myeloid Leukemia, in Relapse; Acute Myeloid Leukemia Refractory
    NCT06128044
  • PI
    A Phase II Multi-center, Single Arm, Safety and Efficacy Study of MBG453 in Combination With Azacitidine and Venetoclax for the Treatment of Acute Myeloid Leukemia (AML) in Adult Patients Unfit for Chemotherapy
    PHASE2 · Novartis Pharmaceuticals Corporation · IRB STUDY CLOSURE (as of 2025)
    Acute Myeloid Leukemia
    NCT04150029
  • PI
    Phase 1b/2 Clinical Study of the Safety, Tolerability, and Pharmacokinetic and Pharmacodynamic Profiles of CFI-400945 as a Single Agent or in Combination With Azacitidine in Patients With AML, MDS or CMML
    PHASE1; PHASE2 · Treadwell Therapeutics, Inc. · CLOSED TO ACCRUAL (as of 2025)
    Acute Myeloid Leukemia; Myelodysplastic Syndromes; Chronic Myelomonocytic Leukemia; AML; MDS; CMML
    NCT04730258
  • PI
    A Phase I/II Study of Venetoclax and Lintuzumab-Ac225 in Patients With Refractory or Relapsed AML
    PHASE1; PHASE2 · Actinium Pharmaceuticals, Inc. · IRB STUDY CLOSURE (as of 2025)
    Acute Myeloid Leukemia; Relapsed Adult AML
    NCT03867682
  • PI
    A Phase 1, Multicenter, Open-Label, Dose-Escalation and Expansion, Safety, Pharmacokinetic, Pharmacodynamic, and Clinical Activity Study of Intravenously Administered IO-202 and IO-202 + Azacitidine ± Venetoclax in Acute Myeloid Leukemia (AML) Patients With Monocytic Differentiation and in Chronic Myelomonocytic Leukemia (CMML) Patients
    PHASE1 · Immune-Onc Therapeutics, Inc. · IRB STUDY CLOSURE (as of 2025)
    AML With Monocytic Differentiation; CMML
    NCT04372433
  • PI
    Randomized Phase 2 Study of Daunorubicin and Cytarabine Liposome + Pomalidomide Versus Daunorubicin and Cytarabine Liposome in Newly Diagnosed AML With MDS-Related Changes
    PHASE2 · National Cancer Institute · CLOSED TO ACCRUAL (as of 2025)
    Acute Myeloid Leukemia; Acute Myeloid Leukemia Post Cytotoxic Therapy; Acute Myeloid Leukemia With Multilineage Dysplasia; Chronic Myelomonocytic Leukemia; Myelodysplastic Syndrome; Myeloproliferative Neoplasm
    NCT04802161
  • PI
    A Phase 1/2 Open-Label Study of the Safety, Tolerability and Efficacy of the Selective Inhibitor of Nuclear Export (SINE) Compound Eltanexor (KPT-8602) in Patients With Newly Diagnosed and Relapsed/Refractory Cancer Indications
    PHASE1; PHASE2 · Karyopharm Therapeutics, Inc. · IRB STUDY CLOSURE (as of 2024)
    Relapsed/Refractory Multiple Myeloma (RRMM); Metastatic Colorectal Cancer (mCRC); Metastatic Castration-Resistant Prostate Cancer (mCRPC); Higher-Risk Myelodysplastic Syndrome (HR-MDS); Acute Myeloid Leukemia (AML); Newly Diagnosed Intermediate/High-Risk MDS
    NCT02649790
  • PI
    A Phase 1b/2 Study of IMGN632 as Monotherapy or Combination With Venetoclax and/or Azacitidine for Participants With CD123-Positive Acute Myeloid Leukemia
    PHASE1; PHASE2 · AbbVie Inc. · CLOSED TO ACCRUAL (as of 2024)
    Acute Myeloid Leukemia
    NCT04086264
  • PI
    A Multi-phase, Pharmacokinetics, Safety, and Efficacy Study of ASTX030 (Azacitidine and Cedazuridine) as Monotherapy in Subjects With Myeloid Neoplasm or in Combination With Venetoclax in Subjects With AML (AZTOUND Study)
    PHASE2; PHASE3 · Astex Pharmaceuticals, Inc. · IRB STUDY CLOSURE (as of 2024)
    Myelodysplastic Syndromes; Acute Myeloid Leukemia; Myelodysplastic Syndrome/Neoplasm; Chronic Myelomonocytic Leukemia
    NCT04256317
  • PI
    A Single-Arm, Open-Label Pharmacokinetic, Safety, and Efficacy Study of ASTX727 in Combination With Venetoclax in Adult Patients With Acute Myeloid Leukemia
    PHASE1; PHASE2 · Taiho Oncology, Inc. · CLOSED TO ACCRUAL (as of 2024)
    Acute Myeloid Leukemia
    NCT04657081
  • PI
    A Phase 1B, Open-label, Global, Multicenter, Dose Determination Study to Evaluate Safety, Tolerability, and Preliminary Efficacy of CC-486 (ONUREG®) in Combination Therapy in Subjects With Acute Myeloid Leukemia (AML)
    PHASE1 · Celgene Corporation · IRB STUDY CLOSURE (as of 2024)
    Leukemia, Myeloid, Acute
    NCT04887857
  • PI
    A Modular Phase I/II, Open-Label, Multi-Centre Study to Assess the Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of AZD0466 Monotherapy or in Combination in Patients With Advanced Haematological Malignancies
    PHASE1 · AstraZeneca, PLC · IRB STUDY CLOSURE (as of 2023)
    Haematological Malignancies
    NCT04865419
  • PI
    A Phase 1 Study of Safety, Pharmacokinetics and Preliminary Activity of TAS1553 in Subjects With Relapsed or Refractory (R/R) Acute Myeloid Leukemia (AML) and Other Myeloid Neoplasms
    PHASE1 · Astex Pharmaceuticals, Inc. · IRB STUDY CLOSURE (as of 2023)
    Acute Myeloid Leukemia; Myeloproliferative Neoplasm; Myelodysplastic/Myeloproliferative Neoplasm
    NCT04637009
  • PI
    Treatment of single patient infected with Acute Lymphocytic Leukemia (ALL) using Navitoclax and Venetoclax
    Weill Medical College of Cornell University · IRB STUDY CLOSURE (as of 2023)
  • PI
    A Phase 1 Study to Evaluate the Safety and Pharmacokinetic Profiles of CB-5339 in Participants With Relapsed/Refractory Acute Myeloid Leukemia or Relapsed/Refractory Intermediate or High Risk Myelodysplastic Syndrome
    PHASE1 · Cleave Therapeutics, Inc. · IRB STUDY CLOSURE (as of 2023)
    Acute Myeloid Leukemia, in Relapse; Myelodysplastic Syndromes
    NCT04402541
  • PI
    An Open-label, Multicenter, Phase 1b Study of OV-1001 (Cusatuzumab; Anti-CD70 Monoclonal Antibody) in Combination With Background Therapy for the Treatment of Subjects With Acute Myeloid Leukemia
    PHASE1 · Janssen Research & Development, LLC · IRB STUDY CLOSURE (as of 2023)
    Leukemia, Myeloid, Acute
    NCT04150887
  • PI
    An Open-Label, Multicenter, Phase 1b/2 Study of the Safety and Efficacy of KRT-232 When Administered Alone and in Combination With Low-Dose Cytarabine (LDAC) or Decitabine in Patients With Acute Myeloid Leukemia (AML)
    PHASE1; PHASE2 · Kartos Therapeutics, Inc. · IRB STUDY CLOSURE (as of 2023)
    Relapsed or Refractory Acute Myeloid Leukemia (AML); Acute Myeloid Leukemia (AML), Secondary to Myeloproliferative Neoplasms (MPN)
    NCT04113616
  • PI
    An Open-Label, Multicenter, Phase 1b/2 Study of the Safety and Efficacy of TL-895 Combined With KRT-232 in Patients With Relapsed/Refractory (R/R) FLT3+ Acute Myeloid Leukemia (AML)
    PHASE1; PHASE2 · Telios Pharma, Inc. · IRB STUDY CLOSURE (as of 2023)
    Acute Myeloid Leukemia
    NCT04669067
  • PI
    Multicenter, Phase 3 Study of Venetoclax and Azacitidine as Maintenance Therapy for Patients With Acute Myeloid Leukemia in First Remission After Conventional Chemotherapy
    PHASE3 · AbbVie Inc. · IRB STUDY CLOSURE (as of 2023)
    Acute Myeloid Leukemia (AML)
    NCT04102020
  • PI
    A Phase I/II Open Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Clinical Efficacy of ONO-7475 in Patients With Acute Leukemias or Myelodysplastic Syndromes
    PHASE1; PHASE2 · Ono Pharmaceutical Co., Ltd · IRB STUDY CLOSURE (as of 2023)
    Acute Leukemia; Myelodysplastic Syndromes
    NCT03176277
  • PI
    A Sequential Two-Stage Dose Escalation Study to Evaluate the Safety and Efficacy of Ruxolitinib for the Treatment of Chronic Myelomonocytic Leukemia (CMML): A Phase 2 Expansion
    PHASE2 · H. Lee Moffitt Cancer Center & Research Institute · IRB STUDY CLOSURE (as of 2022)
    Chronic Myelomonocytic Leukemia; Leukemia
    NCT03722407
  • PI
    Phase I Study of APR-246 in Combination With Venetoclax and Azacitidine in TP53-Mutant Myeloid Malignancies
    PHASE1 · Aprea Therapeutics AB · IRB STUDY CLOSURE (as of 2022)
    Myeloid Malignancy
    NCT04214860
  • PI
    A Phase I, Multicenter, Open-Label, Dose-Escalation and Expansion, Safety, Pharmacokinetic, Pharmacodynamic, and Clinical Activity Study of Orally Administered AG-120 in Subjects With Advanced Hematologic Malignancies With an IDH1 Mutation
    PHASE1 · Agios Pharmaceuticals, Inc. · IRB STUDY CLOSURE (as of 2022)
    Relapsed or Refractory Acute Myeloid Leukemia (AML); Untreated AML; Other IDH1-mutated Positive Hematologic Malignancies; Myelodysplastic Syndromes
    NCT02074839
  • PI
    Open Label Dose-escalation and Dose-expansion Study to Evaluate the Safety, Expansion, Persistence and Clinical Activity of UCART22 (Allogeneic Engineered T-cells Expressing Anti-CD22 Chimeric Antigen Receptor) in Patients With Relapsed or refractoryCD22+ B-cell Acute Lymphoblastic Leukemia (B-ALL)
    PHASE1; PHASE2 · Cellectis, Inc · IRB STUDY CLOSURE (as of 2022)
    B-cell Acute Lymphoblastic Leukemia
    NCT04150497
  • PI
    A Phase III Multicenter, Randomized, Open Label Study of APR-246 in Combination With Azacitidine Versus Azacitidine Alone for the Treatment of (Tumor Protein) TP53 Mutant Myelodysplastic Syndromes
    PHASE3 · Aprea Therapeutics AB · IRB STUDY CLOSURE (as of 2022)
    MDS
    NCT03745716
  • PI
    A Randomized, Double-Blind, Phase 3 Study Evaluating the Safety and Efficacy of Venetoclax in Combination With Azacitidine in Patients Newly Diagnosed With Higher-Risk Myelodysplastic Syndrome (Higher-Risk MDS)
    PHASE3 · AbbVie Inc. · CLOSED TO ACCRUAL (as of 2022)
    Myelodysplastic Syndrome (MDS)
    NCT04401748
  • PI
    A First-in-Human Phase 1 Study to Assess the Safety, Tolerability, Efficacy, and Pharmacokinetics of FF-10101-01 in Subjects With Relapsed or Refractory Acute Myeloid Leukemia
    PHASE1 · FujiFilm · IRB STUDY CLOSURE (as of 2022)
    AML, Adult
    NCT03194685
  • PI
    Managed Access Programs for ABL001, Asciminib
    Weill Medical College of Cornell University · IRB STUDY CLOSURE (as of 2022)
    Chronic Myeloid Leukemia in Chronic Phase
    NCT04360005
  • PI
    A Phase 1b Dose-escalation Study to Assess the Safety, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of PLX2853 in Subjects With Relapsed or Refractory Acute Myeloid Leukemia or High-risk Myelodysplastic Syndrome
    PHASE1 · Plexxikon, Inc. · IRB STUDY CLOSURE (as of 2022)
    Relapsed Acute Myeloid Leukemia (AML); Refractory Acute Myeloid Leukemia (AML); High-risk Myelodysplastic Syndrome (MDS)
    NCT03787498
  • PI
    A Phase 1 Trial to Evaluate the Potential Impact of Renal Impairment on the Pharmacokinetics and Safety of CPX-351 (Daunorubicin and Cytarabine) Liposome for Injection Treatment in Adult Patients With Hematologic Malignancies
    PHASE1 · Jazz Pharmaceuticals · IRB STUDY CLOSURE (as of 2021)
    Hematologic Malignancy; Acute Myeloid Leukemia; Acute Lymphocytic Leukemia; Acute Lymphoblastic Leukemia; Myelodysplastic Syndromes
    NCT03555955
  • PI
    A Randomized Phase II/III Study of Conventional Chemotherapy +/- Uproleselan (GMI-1271) in Older Adults With Acute Myeloid Leukemia Receiving Intensive Induction Chemotherapy
    PHASE2; PHASE3 · Alliance for Clinical Trials in Oncology · IRB STUDY CLOSURE (as of 2021)
    Acute Myeloid Leukemia
    NCT03701308
  • PI
    A Phase 2 Study of Tipifarnib in Subjects With Chronic Myelomonocytic Leukemia, Other Myelodysplastic /Myeloproliferative Neoplasias, and Acute Myeloid Leukemia
    PHASE2 · Kura Oncology · IRB STUDY CLOSURE (as of 2021)
    Leukemia, Myelomonocytic, Chronic
    NCT02807272
  • PI
    Single Patient Use of Asciminib for Patient LR
    Weill Medical College of Cornell University · IRB STUDY CLOSURE (as of 2021)
  • PI
    Single-Patient Compassion Use of PU-H71 in Patient S-N
    Weill Medical College of Cornell University · IRB STUDY CLOSURE (as of 2019)

News mentions

2 mentionsView all WCM news ↗

Media highlights

2 clips

External relationships

Other

AbbVie Inc. · Amgen, Inc. · AstraZeneca Pharmaceuticals LP · Bristol-Myers Squibb Company · Caribou Biosciences, Inc. · Celgene Corporation · Daiichi Sankyo, Inc. · Ellipses Pharma · Geron Corporation · GlaxoSmithKline · Janssen Pharmaceutical Company · Jasper Therapeutics, Inc · Jazz Pharmaceuticals, Inc. · Kyowa Kirin, Inc. · Molecular Partners AG · MorphoSys AG · NeoGenomics Laboratories, Inc. · Novartis Pharma AG · OncoVerity, Inc. · Pfizer, Inc. · Rigel Pharmaceuticals, Inc. · Roche Pharmaceuticals, Inc. · Servier Pharmaceuticals LLC · Syndax Pharmaceuticals, Inc.