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

NCT00001958 | Completed Phase 2

• 2 other identifiers: 00004000-DK-0040
• Study Type: interventional
• Target: 100
• Locations: 1 country
• Sites: 1

Brief Summary

This 12-month study will evaluate the safety and effectiveness of hydroxyurea in treating beta-thalassemia, a type of anemia caused by defective hemoglobin (the oxygen-carrying pigment in blood). Hemoglobin is composed of two protein chains-alpha globin chains and beta globin chains; patients with beta-thalassemia do not make beta globin. Patients often require frequent red blood cell transfusions. This leads to iron overload, which, in turn, requires iron chelation therapy (removal of iron from the blood). Some drugs, including hydroxyurea, can stimulate production of a third type of protein chain called gamma chains. In the womb, the fetus makes this type of protein instead of beta globin. It is not until after birth, when the fetus no longer produces gamma globin that the beta globin deficiency becomes apparent. Gamma chain synthesis improves hemoglobin and red blood cell production, correcting the anemia. This study will determine if and at what dose hydroxyurea treatment reduces patients' need for red blood cell transfusions and whether certain factors might predict which patients are likely benefit from this treatment. Patients 15 years and older with moderately severe beta-thalassemia may be eligible for this study. Participants will take hydroxyurea daily at a dose calculated according to the patient's body size. Blood will be drawn weekly to measure blood cell and platelet counts. The drug dosage may be increased after 12 weeks of treatment and again after 24 weeks if the white cell and platelet counts remain stable. Patients who respond dramatically to treatment may continue to receive hydroxyurea for up to 3 years.

Conditions

Beta Thalassemia; Hemoglobinopathy

Keywords

Gamma Gene; Hemoglobin Switching; Erythropoiesis; HbE Hemoglobin Chain Synthesis Imbalance; Cooley's Anemia; Beta-Thalassemia Intermedia

Interventions

Hydroxyurea DRUG

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

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.

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK): lead

Study Sites (1)

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Bethesda, Maryland, 20892, United States

Location

Related Publications (3)

• Modell B, Petrou M. Management of thalassaemia major. Arch Dis Child. 1983 Dec;58(12):1026-30. doi: 10.1136/adc.58.12.1026. No abstract available.

  PMID: 6660889 BACKGROUND

• Wolfe L, Olivieri N, Sallan D, Colan S, Rose V, Propper R, Freedman MH, Nathan DG. Prevention of cardiac disease by subcutaneous deferoxamine in patients with thalassemia major. N Engl J Med. 1985 Jun 20;312(25):1600-3. doi: 10.1056/NEJM198506203122503.

  PMID: 4000198 BACKGROUND

• Thomas ED, Buckner CD, Sanders JE, Papayannopoulou T, Borgna-Pignatti C, De Stefano P, Sullivan KM, Clift RA, Storb R. Marrow transplantation for thalassaemia. Lancet. 1982 Jul 31;2(8292):227-9. doi: 10.1016/s0140-6736(82)90319-1. No abstract available.

  PMID: 6124668 BACKGROUND

MeSH Terms

Conditions

beta-Thalassemia; Hemoglobinopathies

Interventions

Hydroxyurea

Condition Hierarchy (Ancestors)

Thalassemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Urea; Amides; Organic Chemicals

Study Design

• Study Type: interventional
• Phase: phase 2
• Purpose: TREATMENT
• Sponsor Type: NIH

Study Record Dates

• First Submitted: January 18, 2000
• First Posted: January 19, 2000
• Study Start: December 1, 1999
• Study Completion: February 1, 2002
• Last Updated: March 4, 2008

Record last verified: 2002-02

Locations

US (1)