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Phase 2
3 Years
N/A
Not Enrolling
Both
Beta-Thalassemia

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Trial Information


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.

Inclusion 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

Type of Study:

Interventional

Study Design:

Primary Purpose: Treatment

Principal Investigator

Susan Park Perrine

Investigator Role:

Study Chair

Investigator Affiliation:

Boston University

Authority:

United States: Food and Drug Administration

Study ID:

199/15337

NCT ID:

NCT00006136

Start Date:

March 1999

Completion Date:

Related Keywords:

  • Beta-Thalassemia
  • genetic diseases and dysmorphic syndromes
  • hematologic disorders
  • rare disease
  • thalassemia intermedia
  • Beta-Thalassemia
  • Thalassemia

Name

Location

Mount Sinai School of MedicineNew York, New York  10029
Yale Comprehensive Cancer CenterNew Haven, Connecticut  06520-8028
Children's Hospital of OaklandOakland, California  94609-1809
Boston University School of MedicineBoston, Massachusetts  02118
Cancer Research CenterBoston, Massachusetts  02118
Clinical Hematology BranchBethesda, Maryland  20892