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Sponsored by: |
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) |
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Information provided by: | Office of Rare Diseases (ORD) |
ClinicalTrials.gov Identifier: | NCT00007072 |
OBJECTIVES:
I. Determine the safety and efficacy of azacitidine and phenylbutyrate in treatment of patients with thalassemia major.
Condition | Intervention | Phase |
---|---|---|
Thalassemia Major |
Drug: azacitidine Drug: phenylbutyrate |
Phase II |
Study Type: | Interventional |
Study Design: | Treatment |
Estimated Enrollment: | 24 |
Study Start Date: | November 2000 |
PROTOCOL OUTLINE: Patients receive azacitidine IV continuously on days 1-4 and oral phenylbutyrate three times a day on days 14-42. Bone marrow needle aspiration is performed on days 6, 14, and 42 to assess disease response to treatment. If no response on day 42, a second course of azacitidine and phenylbutyrate begins 7 days later.
Patients are followed weekly for 3 months and then monthly thereafter.
Ages Eligible for Study: | 18 Years and older |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics--
Diagnosis of thalassemia major Progressive disease defined as: Increasing transfusion requirement or difficulty in maintenance of hemoglobin levels greater than 7 g/dL as a consequence of autologous or allogeneic antibodies OR Increasing extramedullary hematopoiesis causing compression phenomena OR Disease with complications of iron overload despite traditional transfusion and iron chelation therapy (e.g., heart failure, decreased cardiac ejection fraction, endocrinopathy, or evidence of progressive liver dysfunction)
Standard transfusion therapy or iron chelation therapy must be contraindicated
--Prior/Concurrent Therapy--
See Disease Characteristics
--Patient Characteristics--
Performance status: ECOG 0-2
Life expectancy: Greater than 10 days Not moribund
Hematopoietic: See Disease Characteristics
Hepatic: See Disease Characteristics AST or ALT no greater than 3 times upper limit of normal (ULN) Bilirubin no greater than 1.5 times ULN (unless due to hemolysis or Gilbert's disease) Albumin at least 3 g/dL No severe concurrent hepatic disease
Renal: Creatinine no greater than 2 mg/dL Creatinine clearance at least 60 mL/min No severe concurrent renal disease
Cardiovascular: See Disease Characteristics No New York Heart Association class III or IV
Other: Not pregnant or nursing No severe concurrent metabolic disease No severe sepsis or septic shock No concurrent altered mental status or seizure disorder No concurrent myelodysplastic syndrome or leukemia
United States, Maryland | |
Clinical Hematology Branch | |
Bethesda, Maryland, United States, 20892 |
Study Chair: | Griffin Platt Rodgers | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) |
Study ID Numbers: | 199/15578, NIDDK-00-DK-0166, NCI-1311 |
Study First Received: | December 6, 2000 |
Last Updated: | June 23, 2005 |
ClinicalTrials.gov Identifier: | NCT00007072 |
Health Authority: | United States: Federal Government |
genetic diseases and dysmorphic syndromes hematologic disorders rare disease thalassemia major |
4-phenylbutyric acid Hematologic Diseases Beta-thalassemia Rare Diseases Anemia Anemia, Hemolytic Thalassemia |
Anemia, Hemolytic, Congenital Thalassemia minor Genetic Diseases, Inborn Beta-Thalassemia Hemoglobinopathies Azacitidine Hemoglobinopathy |
Antimetabolites Antimetabolites, Antineoplastic Molecular Mechanisms of Pharmacological Action Antineoplastic Agents |
Therapeutic Uses Enzyme Inhibitors Pharmacologic Actions |