Phase II Study of Arginine Butyrate With or Without Epoetin Alfa in Patients With Thalassemia Intermedia

NCT00006136 | Completed Phase 2

• 2 other identifiers: 199/15337BUSM-4839
• Study Type: interventional
• Target: 15
• Locations: 2 countries
• Sites: 7

Brief Summary

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.

Conditions

Beta-Thalassemia

Keywords

genetic diseases and dysmorphic syndromes; hematologic disorders; rare disease; thalassemia intermedia

Interventions

arginine butyrate DRUG

epoetin alfa DRUG

Eligibility Criteria

• Age: 3 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

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

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• Boston University: lead

Study Sites (7)

Children's Hospital of Oakland

Oakland, California, 94609, United States

Location

Yale Comprehensive Cancer Center

New Haven, Connecticut, 06520-8028, United States

Location

Clinical Hematology Branch

Bethesda, Maryland, 20892, United States

Location

Boston University School of Medicine

Boston, Massachusetts, 02118, United States

Location

Cancer Research Center

Boston, Massachusetts, 02118, United States

Location

Mount Sinai School of Medicine

New York, New York, 10029, United States

Location

University College London

London, England, W1W 7EJ, United Kingdom

Location

MeSH Terms

Conditions

beta-Thalassemia; Genetic Diseases, Inborn; Hematologic Diseases; Rare Diseases

Interventions

arginine butyrate; Epoetin Alfa

Condition Hierarchy (Ancestors)

Thalassemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hemic and Lymphatic Diseases; Hemoglobinopathies; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Erythropoietin; Colony-Stimulating Factors; Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors

Study Officials

• Susan Park Perrine

  Boston University

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 2
• Purpose: TREATMENT
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: August 3, 2000
• First Posted: August 4, 2000
• Study Start: March 1, 1999
• Last Updated: November 8, 2005

Record last verified: 2004-07

Locations

US (6); GB (1)