Close
Close

Hydroxyurea for the Treatment of Patients With Sickle Cell Anemia

Access Programs

Disease Information

Descriptive Information
Brief Title † Hydroxyurea for the Treatment of Patients With Sickle Cell Anemia
Official Title † Effect of Hydroxyurea on Fetal Hemoglobin Synthesis in Patients With Sickle Cell Anemia
Brief Summary A total of fifty severely affected patients with homozygous sickle cell disease or other sickling disorders (e.g. B negative or B positive Thalassemia/Sickle) who are greater than 18 years of age will be eligible for treatment. Such patients must be able to tolerate an extensive period without blood transfusion and have relatively well preserved renal and hepatic function (creatinine less than 1.5 mg/dl and normal liver function test with exception of a mild elevation in transaminase). Evidence of severe sickle cell anemia will include recurrent pain crisis, chronic bone oain, evidence of aseptic necrosis with symptoms, and intractable leg ulcer, etc. On admission to the study, each patient will receive a complete history and physical examination. These data and standard laboratory evaluation, including a test for pregnancy if appropriate, will be adequate to ascertain whether any of the criteria for exclusion are present. Each patient must accept responsibility for for using an effective means of contraception. Patients who are found to be HIV positive will be excluded from the study.
Detailed Description Hydroxyurea is a cell-cycle specific agent that blocks DNA synthesis by inhibiting ribonucleotide reductase, the enzyme that converts ribonucleotides to deoxyribonucleotides. Hydroxyurea has been shown to induce the production of HbF, initially in non-human primates, and now in more than fifty patients with sickle cell anemia. The majority of patients with sickle cell disease respond to the drug with a more than two-fold increase in HbF levels; in some patients the percent of HbF exceeds 10 or 15 percent. It is estimated that levels of 20 percent are required to substantially reduce the sickling propensity of red cells and to modulate disease severity. We propose now to treat several patients chronically with hydroxyurea to monitor the durability of the response, to examine for unanticipated long term sided effects and to determine hematological changes occurring longitudinally. Such patients will be candidates for protocols determining the ability of other agents to enhance HbF synthesis, especially in hydroxyurea non-responders. Finally, a series of in vitro studies are planned to attempt to develop predictors of response.
Study Phase Phase 2
Study Type † Interventional
Study Design †
Primary Outcome Measure † An increment in fetal hemoglobin production as a result of hydroxyurea.
Secondary Outcome Measure †
Condition † Sickle Cell Anemia
Intervention † DrugHydroxyurea
Study Arms / Comparison Groups
Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information
Recruitment Status † Drug
Estimated Enrollment † 41
Start Date † February 7, 1984
Completion Date May 18, 2015
Primary Completion Date December 21, 2003
Eligibility Criteria † - INCLUSION CRITERIA: A total of fifty severely affected patients with homozygous sickle cell disease or other sickling disorders (e.g., B negative or B positive Thalassemia/Sickle) who are greater than 18 years of age will be eligible for treatment. Such patients must be able to tolerate an extensive period without blood transfusion and have relatively well preserved renal and hepatic function (creatinine less than 1.5 mg/dl and normal liver function test with exception of a mild elevation in transaminase). Evidence of severe sickle cell anemia will include recurrent pain crisis, chronic bone pain, evidence of aseptic necrosis with symptoms, and intractable leg ulcers, etc. EXCLUSION CRITERIA: Patients who are found to be HIV positive will be excluded from the study.
Gender All
Ages 18 Years - N/A
Accepts Healthy Volunteers No
Contacts ††
Location Countries † United States
Administrative Information
NCT ID † NCT00001197
Organization ID 840029
Secondary IDs †† 84-H-0029
Responsible Party Sponsor
Study Sponsor † National Heart, Lung, and Blood Institute (NHLBI)
Collaborators ††
Investigators † Principal Investigator: Griffin P Rodgers, M.D., National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Information Provided By
Verification Date May 18, 2015
First Received Date † November 3, 1999
Last Updated Date April 19, 2017
† Required WHO trial registration data element.
†† WHO trial registration data element that is required only if it exists.
Find a Clinical Trial
Related Videos
by Abidemi Uruejoma
167 views
by Abidemi Uruejoma
663 views
by Abidemi Uruejoma
10,110 views
Free Newsletter