Clinical Trials Logo
  1. Home
  2. Thalassemia Major
  3. NCT00007072

Phase II Study of Azacitidine and Phenylbutyrate in Patients With Thalassemia Major

Thalassemia Major Clinical Trial

Clinical Trial Summary

OBJECTIVES:

I. Determine the safety and efficacy of azacitidine and phenylbutyrate in treatment of patients with thalassemia major.

Clinical Trial Description

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. ;

Study Design

Primary Purpose: Treatment

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT00007072
Study type Interventional
Source Office of Rare Diseases (ORD)
Contact
Status Terminated
Phase Phase 2
Start date November 2000
View Details
See also
  Status Clinical Trial Phase
Completed NCT02559648 - Denosumab vs Placebo in Patients With Thalassemia Major and Osteoporosis Phase 2
Recruiting NCT02126046 - Unrelated Umbilical Cord Blood Following HLA-haploidentical Hematopoietic Stem Cell Transplantation in Patients With β-thalassemia Major N/A
Completed NCT01125254 - Amlodipine Use in the Prevention and Treatment of Iron Overload in Patients With Thalassemia Major Phase 2/Phase 3
Terminated NCT00034528 - Stem Cell Transplantation After Reduced-Dose Chemotherapy for Patients With Sickle Cell Disease or Thalassemia Phase 2
Completed NCT00733811 - Efficacy Study of the Use of Sequential DFP-DFO Versus DFP Phase 4
Enrolling by invitation NCT02986698 - In Utero Hematopoietic Stem Cell Transplantation for Alpha-thalassemia Major (ATM) Phase 1
Not yet recruiting NCT06026839 - Longitudinal Study on the QoL of Pediatric Patients After HSCT and Its Influencing Factors
Completed NCT04292314 - Hydroxy Urea, Omega 3, Nigella Sativa,Honey on Oxidative Stress and Iron Chelation in Pediatric Major Thalassemia Phase 2/Phase 3
Completed NCT02049450 - Study of Efficacy and Safety of INC424 in Regularly Transfused Patients With Thalassemia. Phase 2
Active, not recruiting NCT04523376 - Pilot Study PBSCT With TCRab Depletion For Hemoglobinopathies N/A
Completed NCT03992001 - Impact of the Preparation Method of Red Cell Concentrates on Transfusion Indices in Thalassemic Patients Phase 4
Not yet recruiting NCT05777733 - NAC Effect on Iron Overload and Blood Transfusion in β-thalassemia Major Phase 1
Completed NCT01241357 - High-Tc Susceptometer to Monitor Transfusional Iron Overload Phase 2
Completed NCT00749515 - Pilot Study for Patients With Poor Response to Deferasirox Phase 4
Completed NCT04260516 - The Effect of N-acetylcysteine on Oxidative Stress Status and Iron Overload in Thalassemia Major Phase 1
Recruiting NCT02307786 - Long Term Outcomes in β Thalassemia Major N/A
Not yet recruiting NCT01323608 - The Effect of Vitamin D Supplementation on Calcium Excretion in Thalassemia: a Dose Response Study Phase 4
Completed NCT00005893 - Study of Allogeneic Bone Marrow Transplantation Using Matched, Related Donors in Patients With Nonmalignant Hematologic Disorders N/A
Recruiting NCT06137079 - "Iron Overload and Endocrinological Diseases"
Completed NCT00006138 - Diagnostic Pilot Study of Dual Energy Absorptiometry in the Detection of Osteopenia or Osteoporosis in Patients With Thalassemia Major N/A