Hematologic Disorders

Hematologic disorders are conditions found in the blood and blood forming organs. This includes blood cell cancers, genetic disorders, anemia, HIV related conditions, sickle cell disease, and complications do to chemotherapy treatment or transfusions. Our hematology team leads clinical trials that evaluate effective treatments through medicines, compare new therapies against existing protocols, and study groups of patients with hematologic conditions to decrease pain and reduce patient’s dependency on current treatments.

A Comprehensive Program to Increase Sickle Cell Trait Knowledge and Awareness Among Parents of Young Children Identified by Newborn Screening

Protocol ID: SCTaware Disease: Sickle Cell Trait Who can participate?: Biological parents of young children (<3 years old) with SCT identified by NBS who are referred and present for in-person SCT education at NCH Lead Researcher: Susan E. Creary

A Natural History Cohort Study of the Safety, Effectiveness, and Practice of Treatment for People with Severe Von Willebrand Disease (VWD)

Protocol ID: ATHN9 Disease: VWD Who can participate?: Participants with severe Von Willebrand Disease with Type 3 VWD or VWF:RCo, VWF:GPlbM or VWF:Ag ≤30% of pooled normal control plasma on more than one occasion; Participants with clinically severe VWD as defined by VWF:RCo, VWF:GPlbM or VWF:Ag ≤40% of normal with severe bleeding phenotype defined as requiring recurrent use of factor concentrates; and Co-enrollment in the ATHNdataset. Lead Researcher: Amy L. Dunn

A Phase 3 Study of Eltrombopag vs. Standard First-Line Management for Newly Diagnosed Immune Thrombocytopenia (ITP) in Children

Protocol ID: ICON 3 Disease: Thrombocytopenia Who can participate?: Age: 1- <18 years. Newly diagnosed ITP (<3 months from diagnosis (first abnormal platelet count). Platelets <30 x109/L at screening Lead Researcher: Melissa J. Rose

A Phase 3b, Prospective, Open-Label, Uncontrolled, Multicenter Study on Long-Term Safety and Efficacy of rVWF in Pediatric and Adult Subjects with Severe Von Willebrand Disease

Protocol ID: SHP677-304 Disease: Von Willebrand Disease (VWD) Who Can Participate?: Ages 12 to 99 Lead Researcher: Amy L. Dunn, MD

A Prospective Natural History Study of Diagnosis, Treatment and Outcomes of Children with SCID Disorders

Protocol ID: PIDTC 6901 Disease: SCID Disorders Who can participate?: SCID being treated with transplant or PEG-ADA or gene therapy Lead Researcher: Hemalatha Geetharani Rangarajan

Assessing the Effect of Intravenous Iron on Fatigue, Cognitive Function and Exercise Capacity in Iron-Deficient Children with Inflammatory Bowel Disease

Protocol ID: IV Iron Disease: IBD Who Can Participate?: Ages 8 to 21 Lead Researcher: Amanda E. Jacobson-Kelly, MD

Development of Multi-Platform Mobile App Technology for Real-Time Measurement of Menstrual Cycle Characteristics in Adolescents

Protocol ID: Mobile App Disease: Heavy Menstrual Bleeding (HMB) Who Can Participate?: Ages 10 to 14 Lead Researcher: Sarah H. O’Brien. MD

Engaging Parents of Children with Sickle Cell Disease and their Providers in Shared-Decision Making for Hydroxyurea (ENGAGE HU)

Protocol ID: ENGAGE Disease: Sickle Cell Who can participate?: Ages 0 to 5 (inclusive) with SCD diagnosis Lead Researcher: Susan E. Creary

Genetic and Biological Determinants of Immune Thrombocytopenia

Protocol ID: ICON Bank Disease: Thrombocytopenia Who can participate?: Patients diagnosed with immune thrombocytopenia (including Evans syndrome if ITP is present). Lead Researcher: Melissa J. Rose

Genotypic and Phenotypic Analysis of Adolescents with Heavy Menstrual Bleeding and Low Von Willebrand Activity

Protocol ID: HMB & Low VWF H-39295 Disease: Menorrhagia Who can participate?: Post-menarchal females < 21 years of age with HMB (defined as PBAC score >100) Lead Researcher: Sarah H. O’Brien

Hydroxyurea Optimization through Precision Study (HOPS)

Protocol ID: HOPS Disease: Sickle Cell Anemia Who can participate?: Children with confirmed (HbSS, HbSD or Hbβ0-thalassemia) who are initiating hydroxyurea therapy, following a discussion with their local clinical team, will be eligible for this study. Lead Researcher: Susan E. Creary

Identifying Copper Deficiency and Associated Hematologic Complications in an At-Risk Population

Protocol ID: Copper Deficiency Disease: J/GJ Tube Dependency Who Can Participate?: Ages 6 months to 21 years Lead Researcher: Amanda E. Jacobson-Kelly, MD

Leveraging the ATHNdataset to Document the State of Rare Coagulation Disorders in the United States

Protocol ID: ATHN10 Diseases: Rare Coagulation Disorders Who Can Participate?: Ages 0 to 99 Lead Researcher: Amy L. Dunn, MD

Phase 3, Prospective, Multicenter, Uncontrolled, Open-Label Clinical Study to Determine the Efficacy, Safety, and Tolerability of rVWF with or without ADVATE in the treatment and control of bleeding episodes, the efficacy and safety of rVMF in Elective and Emergency Surgeries, and the PK or rVWF in Children Diagnosed with Severe von Willebrand Disease

Protocol ID: Baxalta 071102 rVWF (Von Vendi) Disease: Von Willebrand Disease Who can participate?: 3 age cohorts:

Cohort 1: >12 to <18 years Cohort 2: >6 to <12 years (after 6 subjects from Cohort 1) Cohort 3: <6 years (after 6 subjects in Cohort 2) Severe VWD

Lead Researcher: Amy L. Dunn

Post-Marketing Study to Evaluate the Efficacy and Safety of PANZYGA in Pediatric Patients with Chronic Immune Thrombocytopenia (ITP)

Protocol ID: NGAM-10 Disease: Immune Thrombocytopenia (ITP) Who Can Participate?: Ages 1 to 17 Lead Researcher: Melissa J. Rose, DO

Randomization of Cytarabine Monotherapy vs. Standard of care Vinblastine/Prednisone for Frontline Treatment of Langerhans Cell Histiocytosis

Protocol ID: LCH REASON Disease: Langerhans cell histiocytosis (LCH) Who Can Participate?: Confirmed diagnosis of Langerhans cell histiocytosis, ages 0 to 21 Lead Researcher: Amanda E. Jacobson-Kelly, MD

Reduced Intensity Conditioning for Haploidentical Bone Marrow Transplantation in Patients with Symptomatic Sickle Cell Disease

Protocol ID: BMT CTN 1507 Disease: SCD Who can participate?: Must be b/t 5 – 14 yrs and had a stroke. Must be b/t 15 – 45yrs and meet additional protocol criterion. Have a donor who is a half match. Lead Researcher: Hemalatha Geetharani Rangarajan

Reproductive Care in Women with Sickle Cell Disease- Use of Secondary Data to Investigate Risk of Contraception-Related Thrombosis and the Impact of Hydroxyurea on Pregnancy Outcomes

Protocol ID: Medicaid Data in SCD Disease: Sickle Cell Disease (SCD) Lead Researcher: Sarah H. O’Brien. MD

Safety, tolerability, pharmacokinetics, and pharmacodynamics of single and multiple subcutaneous doses of NNC0365-3769 (Mim8) in healthy subjects and in subjects with hemophilia A with or without factor VIII inhibitors

Protocol ID: FRONTIER1 Disease: Hemophilia A Who Can Participate?: Ages 12 to 64 Lead Researcher: Amy L. Dunn, MD

Screening and Lead-In Observational Protocol to Determine Potential Patient Eligibility for Inclusion in AAV Gene Therapy Clinical Trials in Hemophilia B

Protocol ID: ECLIPSE Disease: Hemophilia B Who Can Participate?: Ages 16 to 99 Lead Researcher: Amy L. Dunn, MD

Surveillance Epidemiology of Coronavirus Under Research Exclusion (SECURE)-SCD Registry

Protocol ID: SECURE-SCD Registry Disease: Sickle Cell Disease (SCD) Who Can Participate?: Ages 0 to 99 Lead Researcher: Anthony D. Villella, MD

Adolescents Presenting to Young Women’s Hematology Clinics with Heavy Menstrual Bleeding: A Prospective Cohort Study and Research Registry

Protocol ID: HMB Registry Disease: Menorrhagia Who can participate?: Ages < 21, patients who are new referrals to a Young Women Hematology Clinic or General Hematology Clinic for evaluation of HMB Lead Researcher: Sarah H. O’Brien, MD

Sever Chronic Neutropenia International Registry Protocol

Protocol ID: SCN Registry Disease: SCN Who can participate?: Age greater than three months Lead Researcher: Melissa J. Rose, DO

Our hematology team leads clinical trials that evaluate effective treatments through medicines, compare new therapies against existing protocols, and study groups of patients with hematologic conditions to decrease pain and reduce patient’s dependency on current treatments.