Research Studies

Division of Hematology Research Studies

Find research studies available to children cared for by the Division of Hematology team.
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Recruitment Status
Non-recruiting (3)
Recruiting (1)

Related conditions
(-) Beta Thalassemia (Cooley's Anemia) (2)
(-) Hemophilia (2)
Alpha Thalassemia (1)
Anemia (1)
Idiopathic Thrombocytopenic Purpura (ITP) (1)

Study categories
Adults (4)
Children (3)
MRI/Imaging Studies (2)

Specialty
Cellular Therapy and Transplant (1)
General Pediatrics (1)
Hematology (4)

Eligible age

Clinical trial phase
Phase I (1)
Phase II (2)
Phase III (1)
N/A (1)

Gene Therapy Trial for Hemophilia A
This clinical trial study uses a modified adeno-associated virus (AAV) to carry the human factor VIII gene to the liver. In the liver is where factor VIII protein is made. The study product, SPK-8011, is available only to participants in this research study. This product has not been approved yet for use in the general public. This will be the first hemophilia A gene transfer research study done in the United States. To learn more about the benefits and risks related to this study, please contact Dr. Lindsey George at (267) 425-0132.In order to participate in this study, you must be male, 18 years or older, have hemophilia A with FVIII activity levels less or equal to 2%, a history of bleeding that requires prophylaxis or on demand therapy, and no history of inhibitor to factor VIII. This study requires a number of visits over the course of 12 months. All costs related to participation in the study will either be covered or reimbursed by the Sponsor of the study, Spark Therapeutics, and participants will be compensated for their time.

Phase: Phase I, Phase II

Actively recruiting: No

Category: Adults, MRI/Imaging Studies

Conditions: Hemophilia

Gladiolus Study

This Phase 2 clinical research study is evaluating the safety and effectiveness of a once-daily oral investigational medicine that may improve anemia and reduce the need for red blood cell (RBC) transfusions among some patients who are chronically transfused. All participants enrolled in the study will receive the investigational medication.

We are currently enrolling individuals who meet the following criteria:*

  • Regular RBC transfusions to prevent stroke or recurrence of stroke (Cohort A)
  • Thalassemia and receiving regular RBC transfusions (Cohort B)
  • Thalassemia and not receiving regular RBC transfusions (Cohort C)

There are additional eligibility requirements, which the investigator will explain to you.

Phase: Phase II

Actively recruiting: No

Category: Adults, Children

Conditions: Alpha Thalassemia, Beta Thalassemia (Cooley's Anemia), Anemia

HGB-212: Phase 3 Beta Thalassemia LentiGlobin BB305 Gene Therapy Trial

Individuals with a certain type of beta thalassemia that is treated with regular transfusions, and who are 50 years old and younger, may be able to participate. A number of screening tests will be done to determine if subjects can participate. These include review of your past medical history, blood and urine tests, bone marrow aspirate/biopsy, bone age/ DEXA scan, heart ultrasound, electrocardiogram, magnetic resonance imaging (MRI) of the heart and liver, and liver biopsy. If eligible, blood stem cells will be collected by apheresis or by bone marrow harvest. These stem cells will then have a healthy beta globin gene inserted. You will then be hospitalized and chemotherapy will be given to empty out the bone marrow. The stem cells, with the inserted gene, will then be given back to you. You will be followed for side effects and to see if the inserted gene helps you make hemoglobin.

Phase: Phase III

Actively recruiting: No

Category: Adults, Children, MRI/Imaging Studies

Conditions: Beta Thalassemia (Cooley's Anemia)

Regeneron for Hemophilia B

This research study is seeking participants who have hemophilia B and need regular preventive treatment with factor IX protein (FIX) replacement therapy to prevent and also control bleeding events. To join the study , you must be male as assigned at birth, and between the ages of 16 and 65 years at the time of signing the informed consent form. The purpose of this study is to gather at least 6 months of information on bleeding events for each individual participant while they continue to use their usual FIX replacement therapy. There is no experimental drug or treatment being tested in this study. The study is informational, and part of a larger program to understand and treat hemophilia B with a potential experimental new therapy in the future. After participants complete this research study, should they be interested and eligible, they may be offered the opportunity to take part in a future study with this new therapy. There is no obligation to agree to taking part in this future study.

Phase: N/A

Actively recruiting: Yes

Category: Adults, Children

Conditions: Hemophilia