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Hematological Disorders Clinical Trials

 

St. Jude Clinical Trials

CPXSMN: CPX-351 in Pediatric Patients with Secondary Myeloid Neoplasms

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

Diseases Treated:

Eligibility:

  • 1–22 years old
  • Treatment-related or secondary MDS/AML 
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DIRECT70: CAR T–Cell Therapy for Children with Blood Malignancies

DIRECT70: CAR T–Cell Therapy Directed to CD70 for Pediatric Patients with Hematological Malignancies (DIRECT70)

Diseases Treated:

ALL
AML
MDS
Lymphoma

Eligibility:

  • Up to 21 years old
  • CD70+ acute myeloid leukemia, acute lymphoblastic leukemia (B-ALL, T-ALL), lymphoma, or myelodysplastic syndrome
  • If prior allogeneic hematopoietic cell transplant, no graft vs. host disease
  • Has an identified hematopoietic cell transplantation donor
  • Has adequate organ function
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HAPSAA: Partially Matched Related Donor Bone Marrow Transplant for Patients with Aplastic Anemia

Haploidentical Donor Hematopoietic Cell Transplantation for Patients with Severe Aplastic Anemia

Diseases Treated:

Aplastic Anemia

Eligibility:

  • 21 years old and younger
  • Diagnosis of severe aplastic anemia
  • No available matched donor
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INSIGHT-HD: Investigating the Genetics of Hematologic Diseases

Diseases Treated:

Non-malignant blood diseases (non-therapeutic)

Eligibility:

  • Receiving therapy or a consultation for a non-malignant blood disorder
  • Biological relatives (with or without a non-malignant blood disorder) who agree to undergo genetic testing
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LEAPS: Understanding Sickle Cell Disease Transition to Adult Care

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

Diseases Treated:

Sickle Cell Disease

Eligibility:

This is a non-therapeutic study for patients at St. Jude Children’s Research Hospital and Methodist Adult Comprehensive Sickle Cell Center.

  • Diagnosis of sickle cell disease (SCD)
  • 16 to 20 years old
  • English is primary language
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LVXSCID-ND: Gene Therapy for X-Linked Severe Combined Immunodeficiency (SCID-X1) in Newly Diagnosed Infants

A Pilot Feasibility Study of Gene Transfer For X-Linked Severe Combined Immunodeficiency (SCID-X1) in Newly Diagnosed Infants Using a Self-Inactivating Lentiviral Vector To Transduce Autologous CD34 + Hematopoietic Stem Cells

Diseases Treated:

X-linked Severe Combined Immunodeficiency (SCID-X1)

Eligibility:

  • Diagnosis of Severe Combined Immunodeficiency, X-linked (SCID-X1)
  • Newborn to 2 years of age
  • No prior therapy with allogeneic stem cell transplantation
  • No HIV infection
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SKITS2: School Readiness Intervention for Sickle Cell Disease

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

Diseases Treated:

Sickle Cell

Eligibility:

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Collaborative Clinical Trials

BHEEM: Study of BEAM-101 in Patients with Severe Sickle Cell Disease

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

Diseases Treated:

Sickle Cell Disease

Eligibility:

  •  18 to 35 years old
  • Diagnosed with severe sickle cell disease
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