Hematological Disorders Clinical Trials

 

St. Jude Clinical Trials

CPPGAL: Characterization of the Patient Population with Galactosialidosis

Diseases Treated:

Galactosialidosis

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients.

View Trial

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.
View Trial

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
View Trial

LVXSCID-ND: Gene Transfer for X-Linked Severe Combined Immunodeficiency (SCID) in Newly Diagnosed Infants

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

Diseases Treated:

X-linked Severe Combined Immunodeficiency (SCID-X1)

Eligibility:

  • Diagnosis of Severe Combined Immunodeficiency Disease, X-linked (SCID-X1)
  • Newborn to 12 months of age
  • No prior therapy with allogeneic stem cell transplantation
  • No HIV infection
View Trial

Collaborative Clinical Trials

HIRS: Hemophilia Inhibitor Research Study Phase III

Diseases Treated:

Hemophilia (non-therapeutic)

Eligibility:

Study Arm I

  • Participant is male age two (2) or older (Study Arm I) or of any age and any race/ethnicity (Study Arm II)
  • Participant has a diagnosis of Hemophilia A (Study Arm I) or Hemophilia A or B (Study Arm II) with a baseline factor activity level less than 50% of normal.
  • Participant has not participated in HIRS Phase I or Phase II (Study Arm I)
View Trial

PKiDS: Pyruvate Kinase Deficiency (PKD) Natural History Study

Diseases Treated:

Pyruvate kinase deficiency (PKD)

Eligibility:

This is a non-therapeutic clinical trial that is only open to St. Jude patients.

  • Participants of all ages with biochemically or genetically diagnosed PKD.
  • Participants with a hemolytic anemia and a family member with genetically diagnosed PKD.
  • The participant or the guardian of the participant is willing and able to give written informed consent and/or assent.
View Trial