Highlights
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Publications
99 publications
Funding
14 grantsView NIH portfolio on RePORTER ↗
Active
1 grant- Sub-PI5 U1AMC28549-09-00Improving Access to Comprehensive Thalassemia Care - Eastern Region NetworkHealth Resources and Services Administration · 2021–2026 · via Children's Hospital of Philadelphia
Completed grants (13)
- PINU58DD000005-01 00The New York Comprehensive Thalassemia Center - Expanded NetworkCenters for Disease Control and Prevention · 2019–2025No publications yet
- KPS10OD027039-01A17T Magnetic Resonance Imaging System for Basic, Translational and Clinical ResearchOffice of the Director, NIH · 2021–2025
- Co-I75N92019D00037Recipient Epidemiology and Donor Evaluation Study IV - Pediatric (REDS-IV-P} -Domestic Hubs - Phase 3National Heart, Lung, and Blood Institute · 2024–2025 · via Columbia University
- KPR01DK116126-05S1QSM to Guide Iron Chelating Therapy in Transfusional Iron Overload - Administrative SupplementNational Institute of Diabetes and Digestive and Kidney Diseases · 2019–2025· 2 renewals
- Co-I75N92019D00037Recipient Epidemiology and Donor Evaluation Study IV - Pediatric (REDS-IV-P} -Domestic Hubs - Phase 2National Heart, Lung, and Blood Institute · 2021–2024 · via Columbia University
- PISRA 201879-01Burden of Illness of Beta ThalassemiaCelgene Corporation · 2019–2024Contract
- Co-I75N92019D00037Recipient Epidemiology and Donor Evaluation Study IV - Pediatric (REDS-IV-P} -Domestic Hubs - Phase 2 Budget BNational Heart, Lung, and Blood Institute · 2021–2024 · via Columbia University
- Sub-PIR33HL147845-03Development of a Clinical Hemoglobin ModulatorNational Heart, Lung, and Blood Institute · 2021–2023 · via Phoenicia BioSciences, Inc.
- PI6 U1AMC28551-03-01The New York Comprehensive Thalassemia Center Expanded Regional Comprehensive Care NetworkHealth Resources and Services Administration · 2018–2022
- Co-I75N92019D00037REDS-IV-P Domestic HubsNational Heart, Lung, and Blood Institute · 2019–2022 · via Columbia University
- KPASH 204940-01Honors Program Offer- CSOM StudentAmerican Society of Hematology Inc · 2020–2021
- PI4 U1AMC28551-02-02The New York Thalassemia Center - Expanded Regional Comprehensive Care NetworkHealth Resources and Services Administration · 2015–2018
- PIK23HL004275-05MENTORED PATIENT ORIENTED HEMATOLOGY RESEARCH TRAININGNHLBI · 2000–2006 · via NIH RePORTER
Clinical research
15 studies
Active
5 studies- PINCT07517133A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Subjects With α- or β-Non-Transfusion-Dependent ThalassemiaPHASE3 · Agios Pharmaceuticals, Inc. · OPEN TO ACCRUAL (as of 2026)Non-Transfusion-dependent Alpha-Thalassemia; Non-Transfusion-dependent Beta-Thalassemia
- PINCT07506863A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Subjects With α- or β- Transfusion-Dependent ThalassemiaPHASE3 · Agios Pharmaceuticals, Inc. · OPEN TO ACCRUAL (as of 2026)Transfusion-dependent Alpha-Thalassemia; Transfusion-dependent Beta-Thalassemia
- PINCT06421636A PHASE 2, TWO-PART, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTICENTER STUDY TO EVALUATE THE EFFICACY, SAFETY, AND TOLERABILITY OF SUBCUTANEOUSLY ADMINISTERED REGN7999 (AN INHIBITOR OF TMPRSS6) IN PARTICIPANTS WITH IRON OVERLOAD DUE TO NON-TRANSFUSION DEPENDENT β-THALASSEMIAPHASE2 · Regeneron Pharmaceuticals, Inc. · OPEN TO ACCRUAL (as of 2025)Non-Transfusion Dependent Beta-Thalassemia (NTDT)
- PINCT06609226An Open-label, Multi-centre, Rollover Study to Characterise Long-term Safety and Efficacy of Etavopivat in Adults, Adolescents and Children Who Have Sickle Cell Disease or Thalassaemia and Have Completed a Treatment Period in an Etavopivat StudyPHASE3 · Novo Nordisk Pharmaceuticals, Inc. · OPEN TO ACCRUAL (as of 2025)Sickle Cell Disease; Thalassemia
- PINCT04143724A Phase 2a Study to Evaluate the Safety and Pharmacokinetics of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-ThalassemiaPHASE2 · Celgene Corporation · OPEN TO ACCRUAL (as of 2024)Beta-Thalassemia
Completed & closed trials (10)
- PINCT04987489A Phase 2 Open-Label Study to Evaluate Safety and Clinical Activity of Etavopivat in Patients With Thalassemia or Sickle Cell DiseasePHASE2 · Forma Therapeutics · IRB STUDY CLOSURE (as of 2026)Sickle Cell Disease; Thalassemia
- PINCT05144256A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Subjects With Pyruvate Kinase Deficiency Who Are Regularly Transfused, Followed by a 5-Year Open-label Extension PeriodPHASE3 · Agios Pharmaceuticals, Inc. · CLOSED TO ACCRUAL (as of 2023)Pediatric Pyruvate Kinase Deficiency; Pediatric Hemolytic Anemia
- PINCT04064060A Phase 3b, Open-label, Single-arm, Rollover Study to Evaluate Long-term Safety in Subjects Who Have Participated in Other Luspatercept (ACE-536) Clinical TrialsPHASE3 · Celgene Corporation · IRB STUDY CLOSURE (as of 2023)Myelodysplastic Syndromes (MDS); Beta-thalassemia; Myeloproliferative Neoplasm(MPN)-Associated Myelofibrosis
- PINCT04432623A Phase 1b Sequential Open Label Dose-Ranging Study of Safety, Pharmacokinetics, and Preliminary Activity of Benserazide in Subjects With Beta Thalassemia Intermedia and Sickle Cell DiseasePHASE1; PHASE2 · Phoenicia BioSciences, Inc. · IRB STUDY CLOSURE (as of 2023)Beta Thalassemia Intermedia; Sickle Cell Disease
- PINCT04770779A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy and Safety of Mitapivat in Subjects With Transfusion-Dependent Alpha- or Beta-Thalassemia (ENERGIZE-T)PHASE3 · Agios Pharmaceuticals, Inc. · CLOSED TO ACCRUAL (as of 2023)Transfusion-dependent Alpha-Thalassemia; Transfusion-dependent Beta-Thalassemia
- PICollecting Patient-reported Outcome (PRO) Data from Patients with Transfusion Dependent à -ThalassemiaBluebird Bio · IRB STUDY CLOSURE (as of 2023)
- PINCT04770753A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy and Safety of Mitapivat in Subjects With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (ENERGIZE)PHASE3 · Agios Pharmaceuticals, Inc. · CLOSED TO ACCRUAL (as of 2023)Non-Transfusion-dependent Alpha-Thalassemia; Non-Transfusion-dependent Beta-Thalassemia
- PINCT04411082A Phase 2 Study to Evaluate the Safety and Tolerability of IMR-687 in Subjects With Beta ThalassemiaPHASE2 · Imara Inc · IRB STUDY CLOSURE (as of 2022)β Thalassemia
- PINCT03637556A Multi-Center, Open-Label, Two-Period Cross-Over, Patient-Pilot Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Iron Chelating Activity of DST-0509 (Deferasirox) Tablets in Thalassemia Patients With Inadequate Response to Standard Chelation TherapyPHASE2 · DisperSol Technologies · IRB STUDY CLOSURE (as of 2022)Thalassemia Major
- PINCT03802916Safety and Acceptability of Deferiprone Delayed Release Tablets in Patients With Systemic Iron OverloadPHASE2 · ApoPharma, Inc. · IRB STUDY CLOSURE (as of 2020)Iron Overload Due to Repeated Red Blood Cell Transfusions
External relationships
Advisory/Scientific Board Member
Agios Pharmaceuticals, Inc. · Vertex Pharmaceuticals, Inc.
Other
Agios Pharmaceuticals, Inc. · Bristol-Myers Squibb Company · Chiesi Pharmaceuticals · Novo Nordisk Pharmaceuticals, Inc.