Search / Trial NCT00001958

Hydroxyurea to Treat Beta-Thalassemia (Cooley's Anemia)

Launched by NATIONAL INSTITUTE OF DIABETES AND DIGESTIVE AND KIDNEY DISEASES (NIDDK) · Jan 18, 2000

Trial Information

Current as of December 27, 2024

Completed

Keywords

Gamma Gene Hemoglobin Switching Erythropoiesis Hb E Hemoglobin Chain Synthesis Imbalance Cooley's Anemia Beta Thalassemia Intermedia

ClinConnect Summary

Individuals with homozygous beta thalassemia are either severely anemic or dependent on blood transfusion to sustain life. Deficient synthesis of the beta chain leads to imbalanced chain synthesis with an excess of alpha globin. This alpha globin precipitates, causing ineffective erythropoiesis and shortened red cell survival. Hydroxyurea is a cell-cycle specific agent that blocks DNA synthesis by inhibiting ribonuclease reductase, the enzyme that converts ribonucleotides to deoxyribonucleotides. Administration of hydroxyurea to primates and more than 300 patients with sickle cell anemia ha...

Gender

ALL

Eligibility criteria

  • Beta-Thalassemia Intermedia patients.
  • Steady-state Hb values greater than 6.5gm/dl (unrelated to transfusion)
  • Males and females.
  • Patients greater than 15 years of age.
  • Patients who are transfusion-requiring but not dependent will be offered the opportunity to enroll.
  • Stable renal and hepatic function
  • Willingness to use appropriate birth control measures.
  • Ability to give informed consent.
  • No beta-thalassemia major.
  • No blood transfusion requirement greater than 1 unit every 2 months over the last 12 months.
  • No patients with WBC less than 4000/micrograms.
  • No one with a platelet count less than 150,000/micrograms.
  • No evidence of active viral infective, including viral hepatitis.
  • No abnormal liver function test (ALT or AST greater than 2.5 x normal).
  • No abnormal renal function test (creatinine greater 1.5 mg/dl).
  • No HIV positive blood test.

About National Institute Of Diabetes And Digestive And Kidney Diseases (Niddk)

The National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) is a prominent research institution within the National Institutes of Health (NIH) dedicated to advancing scientific knowledge and promoting health in the areas of diabetes, endocrine and metabolic disorders, obesity, digestive diseases, and kidney diseases. Through rigorous clinical trials and innovative research initiatives, NIDDK aims to improve prevention, diagnosis, and treatment strategies, ultimately enhancing patient outcomes and quality of life. The institute fosters collaboration among researchers, healthcare professionals, and community stakeholders to drive impactful discoveries and translate findings into effective healthcare solutions.

Locations

Bethesda, Maryland, United States

People applied

Timeline

First submit

Trial launched

Trial updated

Estimated completion

Not reported

Discussion 0

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