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.

Dose escalation study to characterize pharmacokinetics after oral admin of eltrombopag in pediatric patient with refractory, relapsed, or treatment naïve severe aplastic anemia or recurrent aplastic anemia

Protocol ID: Eltromopag

Disease: Hematology

Who can participate?: Age 1-18y; New diagnosed or relapsed AA; bone marrow 4weeks prior to treatment; BMT not an option; normal FISH chromosome.

Lead Researcher: Melissa J. Rose

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

Disease: Sickle Cell

Who can participate?: Ages 0 to 5 (inclusive) with SCD diagnosis

Lead Researcher: Susan I. Vear

Exercise versus DDAVP in patients with mild hemophilia A-is one inferior to the  other and do they work additively in improving hemostasis?

Protocol ID: DDAVP-Exercise

Disease: Hemophilia A

Who can participate?:

Mild Hemophilia A

Ages >13 and <21

FVIII: C level of >6% to <25%

Lead Researcher: Riten Kumar, MD

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

Protocol ID: Genotypic and Phenotypic Analysis of Adolescents with HMB & VWF

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

ISTH Database and DNA Biorespository for Congenital Antithrombin Deficiency- pilot/feasibility study

Protocol ID: ISTH Database

Disease: Thrombosis

Who can participate?:

Ages 0-21 years

AT deficiency confirmed

Lead Researcher: Riten Kumar, 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: rVWF-Baxalta

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

Prospective Non-Interventional Study of the Bleeding Episodes, Factor VIII Infusions, and Patient- Reported Outcomes in Individuals with Severe Hemophilia A

Protocol ID: Biomarin 902

Disease: Hematology 

Who can participate?: Males >18, hemophilia A

Lead Researcher: Amy L. Dunn

Safety Of Rivipansel (GMI-1070) In The Treatment Of One or More Vaso-occlusive Crises In Hospitalized Subjects With Sickle Cell Disease

Protocol ID: B5201003- Rivipansel Extension

Disease: Sickle Cell

Who can participate?: ≥ 6 years of age.

Lead Researcher: Anthony D. Villella

Community Counts Surveillance study

Protocol ID: Community Counts

Disease: Multiple Bleeding Disorders

Who can participate?:

Lead Researcher: Amy L. Dunn, MD

Prospective Multi-Center Evaluation of the Duration of Therapy for Thrombosis in Children (the "Kids-DOTT" Trial)

Protocol ID: KidsDott

Disease: Thrombosis

Who can participate?: Children (birth to <21 years of age) with radiologically-confirmed acute venous thrombosis in the past 30 days

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