Blood Disorders Clinical Trials

Full title:

10-CBA: A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs) for Transplantation in Pediatric and Adult Patients with Hematologic Malignancies and Other Indications

Study goal:

The primary objective of this study is to examine the incidence of neutrophil recovery after cord transplantation.

In participants receiving a non-licensed CBU: 1) to determine the frequency of graft rejection; 2) to determine the frequency of infection; 3) to determine the frequency of serious infusion reactio; 4) to determine the 1 year survival rate.

Diagnosis:

Blood disorders, childhood cancer

Full title:

A Phase 1/2 Study Evaluating the Safety and Efficacy of a Single Dose of Autologous CD34+ Base Edited Hematopoietic Stem Cells (BEAM-101) to Increase Fetal Hemoglobin (HbF) Production in Patients with Severe Sickle Cell Disease

Study goal:

The main goal of this study is to see if a single dose of BEAM-101 allows your body to increase the amount of hemoglobin F while reducing the painful effects of sickle cell disease.

Diagnosis:

Sickle cell disease

Age:

18 to 35 years old

Full title:

Needs Assessment of Knowledge, Beliefs, and Attitudes of Patients With Hemophilia B About Gene Therapy

Study goal:

To find out how much patients with hemophilia B, their caregivers, and health care workers know about gene therapy for this disease, and how they feel about gene therapy. This will help us better educate patients and caregivers about  gene therapy for hemophilia B.

Diagnosis:

Hemophilia B

Age:

12 years and older

Full title:

A Phase 3 Study to Evaluate the Safety and Efficacy of a Single Dose of CTX001 in Pediatric Subjects With Severe Sickle Cell Disease

Study goal:

The main goal of this study is to see if a single dose of exa-cel allows your child’s body to increase the amount of hemoglobin F while reducing the painful effects of sickle cell disease.

Diagnosis:

Sickle cell disease

Age:

2 to 11 years old

Full title:

A Prospective, Multicenter, Single-Arm Pilot Study of CPX-351 (VYXEOS) in Individuals < 22 Years with Secondary Myeloid Neoplasms 

Study goal:

This study will help us understand the effects of CPX-351 treatment in patients with MDS and AML.

Diagnosis:

Acute myeloid leukemia, myelodysplastic syndromes

Age:

1-22 years old

Full title:

Functional Neuroimaging of Cognitive Deficits in Pediatric Sickle Cell Disease

Study goal:

To find out how gene therapy and bone marrow transplant affect the brain function of patients with sickle cell disease

Diagnosis:

Sickle cell disease

Age:

8–25 years old

Study goal:

To learn more about participants beliefs, attitudes, and questions about gene therapy to help make web-based resources to share information. This could help patients make treatment decisions.

Diagnosis:

Neurological disorders, sickle cell disease, hemophilia, inherited genetic diseases

Age:

18 to 35 years old with rare genetic diseases

Full title:

Haploidentical Donor Hematopoietic Cell Transplantation for Patients with Severe Aplastic Anemia

Study goal:

The main goal of this study is to learn more about the effects (good and bad) of this treatment in children and young adults with high-risk blood disorders.

Diagnosis:

Aplastic anemia

Age:

21 years old and younger

Full title:

Examining Cognition, Health Literacy, Transition Readiness, and Educational Experiences in Patients with Hemophilia

Study goal:

This study will help researchers understand how hemophilia affects how patients think, feel, and experience school.

Diagnosis:

Hemophilia

Age:

6 to 18 years old

Full title:

Investigating the Genetics of Hematologic Diseases

Study goal:

The main goal of this trial is to collect DNA from individuals with non-cancerous blood diseases and their family members. Researchers will use the DNA to study how genes cause and influence these diseases. All research data will be confidential.

Diagnosis:

Non-malignant blood diseases (non-therapeutic)

Full title:

Longitudinal Examination of Predictors and Outcomes of Sickle Cell Disease Health Care Transition

Study goal:

The main goal of this study is to find out what a successful health care transition looks like and to look at connections between health care transition and factors such as hospitalizations, patients’ disease knowledge and general adjustment. Researchers also want to know how these factors change over time.

Diagnosis:

Sickle cell disease

Age:

16 to 20 years old

Full title:

National Marrow Donor Program: A Research Database for Allogeneic Unrelated Hematopoietic Stem Cell Transplantation

Study goal:

The purpose of this study is to collect information from the medical records of patients receiving an unrelated transplant. Information in this research database may be helpful towards improving the results of future patients receiving transplants.

Full title:

Pain Management of Vaso-Occlusive Crisis in Children and Young Adults With Sickle Cell Disease-Effect of Virtual Reality Technology

Study goal:

The main goal of this clinical trial is to find out how well virtual reality therapy lessens acute pain in sickle cell patients.

Diagnosis:

Sickle cell disease

Age:

Between 6 and 25 years old

Full title:

Sickle Cell Research and Intervention Program

Study goal:

The purpose of this research study is to collect data on sickle cell disease participants from birth to end of life. With these data, researchers will better understand problems caused by sickle cell disease both in children and in adults. 

Diagnosis:

Sickle cell disease, sickle cell anemia

Full title:

SCD Stem Cell Mobilization and Apheresis Using Motixafortide

Study goal:

To see if motixafortide is safe and helps increase collection of stem cells in adults with sickle cell disease. Learn more about the study.

Diagnosis:

Sickle cell disease

Age:

Age 18 and older

Full title:

SKITS2: School Readiness Intervention for Preschool Children with Sickle Cell Disease

Study goal:

This study will help us find out how to make the intervention program better and test it with a larger group of caregivers and their children.

Diagnosis:

Sickle cell disease

Age:

4–6

Full title:

TBANK: Protocol for Collecting, Banking, and Distributing Human Tissue Samples: St. Jude Children’s Research Hospital Biorepository

Study goal:

To provide a high-quality repository of tumor and normal samples to facilitate translational research performed by St Jude faculty and their collaborators

Diagnosis:

All diseases

Full title:

A Phase 3 Randomized Trial Comparing Unrelated Donor Bone Marrow Transplantation with Immune Suppressive Therapy for Newly Diagnosed Pediatric and Young Adult Patients with Severe Aplastic Anemia

Study goal:

Determine the optimal, upfront therapy for pediatric SAA in the absence of a matched sibling donor. 

Diagnosis:

Aplastic Anemia

Age:

Up to 25 years old