Supportive Hematological Disorders Studies

Non-therapeutic protocols address such issues as side effects or long-term effects of therapy, quality of life, making testing easier on the children, prevention of pain, and issues related to nutrition and stress. There are also tissue and blood banking protocols for laboratory researchers who are studying diseases and searching for underlying causes and cures. The research at St. Jude is designed to include all aspects related to the care of sick children and their families.

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.

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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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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)
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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.
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