We're building a better ClinicalTrials.gov. Check it out and tell us what you think!
Try the New Site
We're building a modernized ClinicalTrials.gov! Visit Beta.ClinicalTrials.gov to try the new functionality.
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00006136
Recruitment Status : Completed
First Posted : August 4, 2000
Last Update Posted : November 8, 2005
Sponsor:
Information provided by:
Office of Rare Diseases (ORD)

Study Description
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
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.


Condition or disease Intervention/treatment Phase
Beta-Thalassemia Drug: arginine butyrate Drug: epoetin alfa Phase 2

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 Design
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Enrollment : 15 participants
Primary Purpose: Treatment
Study Start Date : March 1999

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Thalassemia

Arms and Interventions
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information


Outcome Measures
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Eligibility Criteria
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   3 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
Contacts and Locations
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

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


Locations
Layout table for location information
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
United Kingdom
University College London
London, England, United Kingdom, W1W 7EJ
Sponsors and Collaborators
Boston University
Investigators
Layout table for investigator information
Study Chair: Susan Park Perrine Boston University
More Information
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
Layout table for additonal information
ClinicalTrials.gov Identifier: NCT00006136    
Other Study ID Numbers: 199/15337
BUSM-4839
First Posted: August 4, 2000    Key Record Dates
Last Update Posted: November 8, 2005
Last Verified: July 2004
Keywords provided by Office of Rare Diseases (ORD):
genetic diseases and dysmorphic syndromes
hematologic disorders
rare disease
thalassemia intermedia
Additional relevant MeSH terms:
Layout table for MeSH terms
Thalassemia
beta-Thalassemia
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
 Hemoglobinopathies
Genetic Diseases, Inborn
Arginine butyrate
Epoetin Alfa
Hematinics
Antineoplastic Agents