Phase II Study of Arginine Butyrate With or Without Epoetin Alfa in Patients With Thalassemia Intermedia
NCT ID: NCT00006136
Last Updated: 2005-11-08
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
PHASE2
15 participants
INTERVENTIONAL
1999-03-31
Brief Summary
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II. Determine whether a proportional increase in gamma-globin synthesis and mRNA and an improvement in nonalfa and alfaglobin chain imbalance by at least 10% over baseline correlate with improved hematologic response in these patients when treated with this regimen.
III. Determine whether a decrease in hemolysis, as assayed by a decrease in LDH, compared to baseline levels correlates with improved hematologic response in these patients when treated with this regimen.
IV. Determine whether any particular genotypes are more responsive than others to this therapy in these patients.
V. Determine whether baseline epoetin alfa levels, gender, and/or baseline reticulocyte counts (or percent circulating nucleated erythroblasts) correlate with improved hematologic response in these patients when treated with this regimen.
Detailed Description
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Patients who have no medical contraindications (e.g., paraspinal extramedullary hematopoiesis, hypertension, or poorly controlled congestive heart failure) may continue therapy. Patients receive arginine butyrate IV over 6-14 hours on days 1-4 of weeks 27, 29, 31, 33, 35, 37, and 39 and epoetin alfa intramuscularly (IM) or subcutaneously (SC) three times weekly on weeks 27-40.
Patients may continue to receive epoetin alfa IM or SC alone three times weekly on weeks 41-52. Patients with severe anemia (hemoglobin less than 7 g/dL) may receive epoetin alfa alone on weeks 1-12 before arginine butyrate induction therapy.
Patients who complete therapy at week 26 are followed every 2 weeks for 2 months. Patients who complete therapy at week 40 are followed monthly for 2 months.
Conditions
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Keywords
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Study Design
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TREATMENT
Interventions
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arginine butyrate
epoetin alfa
Eligibility Criteria
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Inclusion Criteria
--Disease Characteristics--
* Diagnosis of thalassemia intermedia with hemoglobin no greater than 10 g/dL Two beta thalassemia mutations
* Must have undergone prior splenectomy or have no palpable spleen
--Prior/Concurrent Therapy--
* At least 3 months since prior red blood cell transfusion
--Patient Characteristics--
* Performance status: SWOG 0-2
* Hematopoietic: No severe iron overload or ferritin greater than 5,000 ng/mL
* Hepatic: Normal hepatic function No active hepatitis
* Renal: Normal renal function
* Other: Not pregnant or nursing Negative pregnancy test Fertile patients must use effective contraception Must be willing to have vascular access placed No viral disease No contraindication to study compliance
3 Years
ALL
No
Sponsors
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Boston University
OTHER
Principal Investigators
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Susan Park Perrine
Role: STUDY_CHAIR
Boston University
Locations
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Children's Hospital of Oakland
Oakland, California, United States
Yale Comprehensive Cancer Center
New Haven, Connecticut, United States
Clinical Hematology Branch
Bethesda, Maryland, United States
Boston University School of Medicine
Boston, Massachusetts, United States
Cancer Research Center
Boston, Massachusetts, United States
Mount Sinai School of Medicine
New York, New York, United States
University College London
London, England, United Kingdom
Countries
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Other Identifiers
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BUSM-4839
Identifier Type: -
Identifier Source: secondary_id
199/15337
Identifier Type: -
Identifier Source: org_study_id