- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00001958
Hydroxyurea to Treat Beta-Thalassemia (Cooley's Anemia)
Effect of Hydroxyurea on the Level of Ineffective Erythropoiesis, Transfusion Requirement, and Fetal Hemoglobin Synthesis in Patients With Beta-Thalassemia-Intermedia
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.
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
Study Type
Enrollment
Phase
- Phase 2
Contacts and Locations
Study Locations
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Maryland
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Bethesda, Maryland, United States, 20892
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
- Child
- Adult
- Older Adult
Accepts Healthy Volunteers
Genders Eligible for Study
Description
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.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
Collaborators and Investigators
Publications and helpful links
General Publications
- 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.
- 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.
- 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.
Study record dates
Study Major Dates
Study Start
Study Completion
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Estimate)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Hematologic Diseases
- Genetic Diseases, Inborn
- Anemia
- Anemia, Hemolytic, Congenital
- Anemia, Hemolytic
- Thalassemia
- beta-Thalassemia
- Hemoglobinopathies
- Molecular Mechanisms of Pharmacological Action
- Nucleic Acid Synthesis Inhibitors
- Enzyme Inhibitors
- Antineoplastic Agents
- Antisickling Agents
- Hydroxyurea
Other Study ID Numbers
- 000040
- 00-DK-0040
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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Editas Medicine, Inc.RecruitingHemoglobinopathies | Thalassemia Major | Thalassemia Intermedia | Transfusion Dependent Beta ThalassemiaUnited States, Canada
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University of British ColumbiaCompletedSickle Cell Disease | Beta-Thalassemia | Sickle Cell Trait | Sickle Cell-Beta Thalassemia | Sickle Cell-SS DiseaseCanada, Nepal
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Children's Hospital of PhiladelphiaNot yet recruiting
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Bristol-Myers SquibbRecruiting
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Assiut UniversityRecruiting
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Clinical Trials on Hydroxyurea
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UNICANCERCompletedBrain and Central Nervous System TumorsFrance
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St. Jude Children's Research HospitalCompleted
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St. Jude Children's Research HospitalCompletedAnemia, Sickle CellUnited States
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National Heart, Lung, and Blood Institute (NHLBI)CompletedHematologic Diseases | Anemia, Sickle Cell | Hemoglobinopathies