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Hematological Disorders Clinical Trials

 

St. Jude Clinical Trials

FIX-GT: Factor IX Gene Therapy Study for Patients with Hemophilia B

A Factor IX Gene Therapy Study of a Novel Adeno-associated Viral Vector (FLT180a) in Patients with Hemophilia B

Diseases Treated:

Hemophilia B

Eligibility:

  • Male
  • At least 18 years old
  • Diagnosis of hemophilia B
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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

CASPER: A Study to Evaluate the Safety and Efficacy of CRISPR Cas9 Gene Editing in Patients with Severe Sickle Cell Disease

A Phase I/II Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects with Severe Sickle Cell Disease

Diseases Treated:

Sickle Cell Disease

Eligibility:

  • 18 to 35 years old (Ages 18 to 25 at St. Jude)
  • Diagnosis of severe sickle cell disease (SCD)
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EDO312: Edoxaban, a new Oral Anticoagulant, for Blood Clots in Children

Phase III Trial to Evaluate the Pharmacokinetics and Pharmacodynamics of Edoxaban and to Compare the Efficacy and Safety of Edoxaban with Standard Anticoagulant Therapy in Children with Venous Thromboembolism (VTE)

Diseases Treated:

Blood clots (venous thromboembolism)

Eligibility:

This clinical trial is open only to St. Jude patients.

  • Newborn to 17 years old (must be younger than 18)
  • Diagnosis of blood clot in a vein (venous thromboembolism, also called VTE)
  • Requires anticoagulant therapy for at least 90 days
  • Received at least 5 days of heparin therapy prior to study
View Trial

FIX-GT: Factor IX Gene Therapy Study for Patients with Hemophilia B

A Factor IX Gene Therapy Study of a Novel Adeno-associated Viral Vector (FLT180a) in Patients with Hemophilia B

Diseases Treated:

Hemophilia B

Eligibility:

  • Male
  • At least 18 years old
  • Diagnosis of hemophilia B
View Trial