Hematological Disorders Clinical Trials

 

St. Jude Clinical Trials

ED0157: Phase 1 Pediatric PK/PD Study

A Phase I, Open-Label, Single-Dose, Non-Randomized Study to Evaluate Pharmacokinetics and Pharmacodynamics of Edoxaban in Pediatric Patients

Diseases Treated:

For patients at risk for blood clots

Eligibility:

  • Participant has a diagnosis requiring anticoagulant therapy.
  • Participant has completed their standard anticoagulant therapy.
  • Participant is not receiving active therapy for a malignant condition.
  • Participant is less than 18 years of age at the time of consent.
  • Participant and legal guardian agree to food and drug restrictions during study.
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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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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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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

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