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ScholarsBetaWeill Cornell Medicine

Publications

26 publications

Funding

10 grants

Active

4 grants
  • PI
    ATHN Transcends ATHN2020-TS-052-01
    American Thrombosis & Hemostasis Network, Inc. · 2020–2035Contract
    SRA 214042-01
  • Sub-PI
    Hemophilia Treatment Centers (SPRANS)
    Health Resources and Services Administration · 2022–2027 · via Icahn School of Medicine at Mount Sinai
    5 H30MC24048-12-00
  • PI
    2026 ATHN Data Quality Counts Grant Award - Round 17
    American Thrombosis & Hemostasis Network, Inc. · 2026–2026
    ATHN 262023-01
  • Sub-PI
    Public Health Surveillance for the Prevention of Complications of Bleeding Disorders
    Centers for Disease Control and Prevention · 2020–2026 · via Icahn School of Medicine at Mount Sinai
    N U27 DD000020-05-02
Completed grants (6)
  • PI
    2021 ATHN Data Quality Counts (DQC)
    American Thrombosis & Hemostasis Network, Inc. · 2021–2024
    ATHN 220300-01
  • PI
    ATHN 10 - Leveraging the ATHNdataset to Document the State of Rare Coagulation Disorders in the United States
    American Thrombosis & Hemostasis Network, Inc. · 2020–2024Contract
    SA 204660-04
  • Sub-PI
    A Natural History Cohort Study of the Safety, Effectiveness, and Practice of Treatment for People with Hemophilia (ATHN 7)
    Genentech, Inc. · 2019–2024 · via American Thrombosis & Hemostasis Network, Inc.
    ATHN2017-ATHN7
  • Sub-PI
    Hemophilia Inhibitor Prevention and Eradication (INHIBIT) Trials Platform
    Health Resources and Services Administration · 2020–2022 · via University of Pittsburgh
    H30MC24050
  • Sub-PI
    Comprehensive Care in Patients With Hemophilia
    United States Department of Health & Human Services · 2012–2022 · via Mount Sinai School of Medicine
    6 H30 MC24048-06
  • Sub-PI
    Community Counts: Public Health Surveillance for Bleeding Disorders
    Centers for Disease Control and Prevention · 2015–2020 · via Mount Sinai School of Medicine
    No publications yet
    U27DD001155-05-00

Clinical research

15 studies

Active

5 studies
  • PI
    ATHNdataset Registry- American Thrombosis and Hemostasis Network
    American Thrombosis & Hemostasis Network, Inc. · OPEN TO ACCRUAL (as of 2026)
  • PI
    CDC Public Health Surveillance for Bleeding Disorders ? Registry for Bleeding Disorders Surveillance
    American Thrombosis & Hemostasis Network, Inc. · OPEN TO ACCRUAL (as of 2026)
  • PI
    A Phase 3, Prospective, Open-label, Uncontrolled, Multicenter Study on Efficacy and Safety of Prophylaxis With Vonicog Alfa (rVWF) in Children Diagnosed With Severe Von Willebrand Disease
    PHASE3 · Takeda Development Center Americas, Inc. · OPEN TO ACCRUAL (as of 2025)
    Von Willebrand Disease (VWD)
    NCT05582993
  • PI
    A PHASE 3, NON-INVESTIGATIONAL PRODUCT, MULTI COUNTRY COHORT STUDY TO DESCRIBE THE LONG-TERM SAFETY AND EFFECTIVENESS OF A PRIOR SINGLE-DOSE TREATMENT WITH INVESTIGATIVE GIROCTOCOGENE FITELPARVOVEC OR FIDANACOGENE ELAPARVOVEC IN PARTICIPANTS WITH HEMOPHILIA A OR HEMOPHILIA B, RESPECTIVELY
    PHASE3 · Pfizer, Inc. · OPEN TO ACCRUAL (as of 2024)
    Hemophilia A; Hemophilia B
    NCT05568719
  • PI
    ATHN Transcends: A Natural History Cohort Study of the Safety, Effectiveness, and Practice of Treatment in People With Non-Neoplastic Hematologic Disorders
    American Thrombosis & Hemostasis Network, Inc. · OPEN TO ACCRUAL (as of 2021)
    Hematologic Disorder; Bleeding Disorder; Connective Tissue Disorder; Hemophilia; Thrombosis; Von Willebrand Diseases; Thrombophilia; Rare Bleeding Disorder; Platelet Disorder; Factor IX Deficiency; Factor VIII Deficiency; Thalassemia; Sickle Cell Disease
    NCT04398628
Completed & closed trials (10)
  • PI
    Prospective, Observational , Multicenter Study of Effectiveness of Efanesoctocog Alfa on Long-term Joint Health in Patients With Hemophilia A
    Sanofi S.A. · CLOSED TO ACCRUAL (as of 2026)
    Hemophilia A
    NCT05911763
  • PI
    American Thrombosis & Hemostasis Network (ATHN) / "Leveraging the ATHNdataset to Document the State of Rare Coagulation Disorders in the United States"
    American Thrombosis & Hemostasis Network, Inc. · CLOSED TO ACCRUAL (as of 2025)
  • PI
    A Phase 3 Open-label, Multicenter Study of the Long-term Safety and Efficacy of Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein (rFVIIIFc-VWF-XTEN; BIVV001) in Previously Treated Patients With Severe Hemophilia A
    PHASE3 · Bioverativ · IRB STUDY CLOSURE (as of 2025)
    Hemophilia A
    NCT04644575
  • PI
    A Natural History Cohort Study of the Safety, Effectiveness, and Practice of Treatment for People With Hemophilia
    American Thrombosis & Hemostasis Network, Inc. · IRB STUDY CLOSURE (as of 2024)
    Hemophilia A With Inhibitor; Haemophilia A Without Inhibitor; Hemophilia B With Inhibitor; Haemophilia B Without Inhibitor
    NCT03619863
  • PI
    A Phase 3 Open-Label, Multicenter Study of the Safety, Efficacy, and Pharmacokinetics of Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein (rFVIIIFc-VWF-XTEN; BIVV001) in Previously Treated Patients ≥12 Years of Age With Severe Hemophilia A
    PHASE3 · Bioverativ · IRB STUDY CLOSURE (as of 2023)
    Factor VIII Deficiency
    NCT04161495
  • PI
    Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors
    PHASE1; PHASE2 · Spark Therapeutics · IRB STUDY CLOSURE (as of 2023)
    Adeno-Associated Virus (AAV); Blood Coagulation Disorder; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Factor VIII (FVIII); Factor VIII (FVIII) Deficiency; Factor VIII (FVIII) Gene; Factor VIII (FVIII) Protein; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Gene Therapy; Gene Transfer; Hematologic Diseases; Hemorrhagic Disorders; Recombinant; Vector; Inhibitors
    NCT03734588
  • PI
    Phase III Multi-Center, Randomized, Controlled Inhibitor Eradication Trial, Comparing Eloctate Immune Tolerance Induction (ITI) Plus Emicizumab vs. Eloctate ITI Alone to Eradicate Inhibitor Formation in Severe Hemophilia A
    PHASE3 · University of Pittsburgh · IRB STUDY CLOSURE (as of 2022)
    Hemophilia A With Inhibitor
    NCT04303572
  • PI
    Multicenter, Randomized Phase III Inhibitor Prevention Trial, Comparing Eloctate vs. Emicizumab to Prevent Inhibitor Formation in Severe Hemophilia A
    PHASE3 · University of Pittsburgh · IRB STUDY CLOSURE (as of 2022)
    Hemophilia A Without Inhibitor
    NCT04303559
  • PI
    A Phase IV, Multicenter, Single-Arm, Open-Label Study of Emicizumab Prophylaxis in Patients With Hemophilia A With or Without Inhibitors Undergoing Minor Surgical Procedures
    PHASE4 · Genentech, Inc. · IRB STUDY CLOSURE (as of 2020)
    Hemophilia A
    NCT03361137
  • PI
    AN OPEN-LABEL, NON-INVESTIGATIONAL PRODUCT, LEAD-IN STUDY TO EVALUATE AT LEAST 6 MONTHS OF PROSPECTIVE EFFICACY AND SAFETY DATA OF FACTOR IX OR FACTOR VIII PROPHYLAXIS REPLACEMENT THERAPY IN THE USUAL CARE SETTING OF MODERATELY SEVERE TO SEVERE ADULT HEMOPHILIA B SUBJECTS (FIX:C≤2%) WHO ARE NEGATIVE FOR nAb TO AAV VECTOR-SPARK100 AND MODERATELY SEVERE TO SEVERE HEMOPHILIA A ADULT SUBJECTS (FVIII:C≤1%) WHO ARE NEGATIVE FOR nAb TO AAV VECTOR SB-525 CAPSID (AAV6), PRIOR TO THE RESPECTIVE THERAPEUTIC PH 3 GENE THERAPY STUDIES (See Detailed Description Section for Official Protocol Title)
    PHASE3 · Pfizer, Inc. · IRB STUDY CLOSURE (as of 2020)
    Hemophilia B; Hemophilia A
    NCT03587116

External relationships

Advisory/Scientific Board Member

Genentech, Inc.

Other

Takeda Pharmaceutical Company Limited