Phase II Study of Arginine Butyrate With or Without Epoetin Alfa in Patients With Thalassemia Intermedia
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|ClinicalTrials.gov Identifier: NCT00006136|
Recruitment Status : Completed
First Posted : August 4, 2000
Last Update Posted : November 8, 2005
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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.
|Condition or disease||Intervention/treatment||Phase|
|Beta-Thalassemia||Drug: arginine butyrate Drug: epoetin alfa||Phase 2|
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 :||Interventional (Clinical Trial)|
|Enrollment :||15 participants|
|Study Start Date :||March 1999|
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|Ages Eligible for Study:||3 Years and older (Child, Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
PROTOCOL ENTRY CRITERIA:
- 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
- At least 3 months since prior red blood cell transfusion
- 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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00006136
|United States, California|
|Children's Hospital of Oakland|
|Oakland, California, United States, 94609|
|United States, Connecticut|
|Yale Comprehensive Cancer Center|
|New Haven, Connecticut, United States, 06520-8028|
|United States, Maryland|
|Clinical Hematology Branch|
|Bethesda, Maryland, United States, 20892|
|United States, Massachusetts|
|Boston University School of Medicine|
|Boston, Massachusetts, United States, 02118|
|Cancer Research Center|
|Boston, Massachusetts, United States, 02118|
|United States, New York|
|Mount Sinai School of Medicine|
|New York, New York, United States, 10029|
|University College London|
|London, England, United Kingdom, W1W 7EJ|
|Study Chair:||Susan Park Perrine||Boston University|
|Other Study ID Numbers:||
|First Posted:||August 4, 2000 Key Record Dates|
|Last Update Posted:||November 8, 2005|
|Last Verified:||July 2004|
genetic diseases and dysmorphic syndromes
Anemia, Hemolytic, Congenital
Genetic Diseases, Inborn