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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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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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SCDHCT: Reduced Intensity Related Donor HCT for Patients with Severe Sickle Cell Disease

Reduced Intensity Related Donor Peripheral Blood Derived Hematopoietic Progenitor Cell Transplantation for Patients with Severe Sickle Cell Disease

Diseases Treated:

Sickle Cell Disease

Eligibility:

  • 25 years old or younger
  • Diagnosed with severe sickle cell disease
  • Has a suitable HLA-matched sibling donor or single haplotype matched (half matched) family member donor (parents or siblings)
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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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