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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Clinical trial
Adults (5)
Children (4)
MRI/Imaging Studies (2)

Clinical Trial Phases
Phase I (1)
Phase II (2)
Phase III (3)
Phase IV (1)
N/A (1)

Recruitment Status
Non-recruiting (4)
Recruiting (2)

Related conditions
Hemophilia (2)

Related Specialties and Programs
Division of Hematology (6)
Novel Therapeutics for Bleeding Disorders (NoT Bleeding) Program (2)
Clinical In Vivo Gene Therapy (1)
Department of Pediatrics (1)
Gene therapy for hematological disorders (1)
Hemophilia and Bleeding Disorders Program (1)

Specialty
Cellular Therapy and Transplant (1)
General Pediatrics (1)
Hematology (6)

ENERGY Study

TheENERGY study is for adults (ages 18 and up) with warm Autoimmune HemolyticAnemia (wAIHA) who are currently receiving treatment or have previously receivedtreatment. Thegoal of the study is to see if the study drug, Nipocalimab is safe andeffective in the treatment of wAIHA. Nipocalimab is an investigationalmedication designed to stop your immune system from destroying your red blood cells.

You may be able to join the study if you are: 
  •          18 years of age or older.
  •          Diagnosed with wAIHA for at least 3months.
  •          Currently receiving treatment orhave previously received treatment for wAIHA
  •          Have platelet counts of more than30,000/ L and Hemoglobin less than 10 g/dL

 Other studyrequirements will apply.

Phase: Phase II, Phase III

Actively recruiting: No

Category: Adults

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

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

LUNA 3
The LUNA 3 study is for teenagers and adults (ages 12 and up) with primary immune thrombocytopenia (ITP) for which standard medications are no longer working. The goal of the study is to see if the study drug, Rilzabrutinib can safely increase platelet counts and improve symptoms for people with ITP. Rilzabrutinib is an investigational medication designed to stop your immune system from destroying your platelets. You may be able to join the study if you are:
  • 12 years of age or older.
  • Diagnosed with primary ITP for at least 6 months for ages 12 through 17 or for at least 3 months for ages 18 and up.
  • Have taken IVIg or steroids, but they are no longer working.
  • Have platelet counts of less than 30,000. 
Other study requirements will apply.

Phase: Phase III

Actively recruiting: Yes

Category: Adults, Children

Panzyga Study
Immune Thrombocytopenia (ITP) is a blood disorder in which the immune system produces antibodies that attack and destroy the body's own blood platelets. The Panzyga Study is looking at a potential medicine (experimental drug) to see if it can result in increasing platelet counts and may potentially treat ITP. You will be infused with the experimental drug once or twice during the treatment period and will be followed up for 32 days. Testing will include blood tests, urine tests, pregnancy tests and physical exams.You may be able to take part in the study if you are below 18 years of age and have been diagnosed with chronic ITP.

Phase: Phase IV

Actively recruiting: No

Category: Children

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