- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00006136
Phase II Study of Arginine Butyrate With or Without Epoetin Alfa in Patients With Thalassemia Intermedia
OBJECTIVES: I. Determine whether arginine butyrate with or without epoetin alfa can stimulate gamma-globin chain production to a degree that decreases anemia and results in hematologic improvement in patients with thalassemia intermedia.
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.
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
PROTOCOL OUTLINE: This is a multicenter study. Patients receive arginine butyrate IV over 6-14 hours on days 1-5 of weeks 1-4 and 7-10. Patients then receive maintenance arginine butyrate IV over 6-14 hours on days 1-4 of weeks 13, 15, 17, 19, 21, 23, and 25.
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.
Study Type
Enrollment
Phase
- Phase 2
Contacts and Locations
Study Locations
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England
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London, England, United Kingdom, W1W 7EJ
- University College London
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California
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Oakland, California, United States, 94609
- Children's Hospital of Oakland
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Connecticut
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New Haven, Connecticut, United States, 06520-8028
- Yale Comprehensive Cancer Center
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Maryland
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Bethesda, Maryland, United States, 20892
- Clinical Hematology Branch
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Massachusetts
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Boston, Massachusetts, United States, 02118
- Boston University School of Medicine
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Boston, Massachusetts, United States, 02118
- Cancer Research Center
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New York
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New York, New York, United States, 10029
- Mount Sinai School of Medicine
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
PROTOCOL ENTRY 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
Study Plan
How is the study designed?
Design Details
- Primary Purpose: TREATMENT
Collaborators and Investigators
Sponsor
Investigators
- Study Chair: Susan Park Perrine, Boston University
Study record dates
Study Major Dates
Study Start
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
Other Study ID Numbers
- 199/15337
- BUSM-4839
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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