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.

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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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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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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 2 years of age
  • No prior therapy with allogeneic stem cell transplantation
  • No HIV infection
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Collaborative Clinical Trials

AGT4HB: Gene Therapy for Hemophilia B

Dose-Escalation Study of a Self-Complementary Adeno-Associated Viral Vector for Gene Transfer in Hemophilia B

Diseases Treated:

Hemophilia

Eligibility:

  • 18 years old or older
  • Diagnosis of severe hemophilia B
  • Male
  • Treated/exposed to FIX products (e.g., concentrates or fresh, frozen plasma) for at least 10 years or 50 exposure days
  • At least 3 bleeding episodes per year requiring FIX infusions or prophylactic FIX infusions because of frequent prior bleeding episodes
  • Currently free of inhibitor and no history of inhibitors to FIX protein
  • Negative family history for development of an inhibitor
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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
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Go-8: Gene Therapy for Hemophilia A Using a Novel Serotype 8 Capsid Pseudotyped Adeno-Associated Viral Vector Encoding Factor VIII-V3

Diseases Treated:

Hemophilia A

Eligibility:

  • Male
  • At least 18 years old
  • Severe hemophilia A (less than 1% FVIII activity at baseline)
  • On factor prophylaxis or at least 4 bleeds per year or chronic hemophilic arthropathy
  • More than 50 exposure days to factor product
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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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