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NCT00005934 · ClinicalTrials.gov registry record · Phase 2
5-Azacytidine and Phenylbutyrate to Treat Severe Thalassemia
A Phase 2 study, sponsored by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK).
- Completed
- Registry status
- Phase 2
- Development phase
- 24
- Enrollment target
NCT00005934: Completed Phase 2 study, sponsored by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK).
NCT00005934 is a Phase 2 study that has completed, run by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK). The registered enrollment target is 24 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (82% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00005934, a Phase 2 study, has completed, sponsored by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK).
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 24 participants
- Enrollment target
Study Summary
This study will evaluate the safety and effectiveness of 5-azacytidine and phenylbutyrate for treating thalassemia major. Patients with this disease have abnormal production of hemoglobin (the oxygen-carrying protein in red blood cells), which leads to red blood cell destruction. As a result, patients require frequent red cell transfusions over many years. Because of these transfusions, however, excess iron is deposited in various body organs-such as the heart, liver, thyroid gland and, in men, the testes-impairing their function. Fetal hemoglobin-a type of hemoglobin that is produced during fetal and infant life-can substitute for adult hemoglobin and increase the levels of red cells in the body. After infancy, however, this type of hemoglobin is no longer produced in large quantities. 5-azacytidine can increase fetal hemoglobin levels, but this drug can damage DNA, which in turn can increase the risk of cancer. This study will try to lessen the harmful effects of 5-azacytidine by using only one or two doses of it, followed by long-term therapy with phenylbutyrate, a drug that may be as effective as 5-azacytidine with less harmful side effects. Patients 18 years of age and older with severe thalassemia major may be eligible for this study. Before beginning treatment, candidates will have a medical history and physical examination, blood tests, chest X-ray, electrocardiogram (EKG), bone marrow biopsy (removal of a small sample of bone marrow from the hip for microscopic examination) and whole-body magnetic resonance imaging (MRI). For the biopsy, the area of the hip is anesthetized and a special needle is inserted to draw bone marrow from the hipbone. For the MRI scan, a strong magnetic field is used to produce images that will identify sites where the body is making red blood cells. During this procedure, the patient lies on a table in a narrow cylinder containing a magnetic field. Earplugs are placed in the ears to muffle the loud thumping sounds the machine mak
Interventions
- DRUG 5-Azacytidine
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 24 participants |
| Start Date | 2000-06 |
| Est. Completion | 2003-06 |
| Phase | Phase 2 |
What the finished NCT00005934 record still lists
NCT00005934 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 24 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (82% lower).
The record links to 0 conditions, and to 1 intervention - of which 5-Azacytidine is the first listed.
NCT00005934 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00005934 about?
NCT00005934 is a clinical study titled "5-Azacytidine and Phenylbutyrate to Treat Severe Thalassemia". This study will evaluate the safety and effectiveness of 5-azacytidine and phenylbutyrate for treating thalassemia major. Patients with this disease have abnormal production of hemoglobin (the oxygen-carrying protein in red blood cells), which leads to red blood cell destruction. As a result, patien...
What is the current status of trial NCT00005934?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 24 participants. The study started on 2000-06. Estimated completion is 2003-06.
What interventions are being tested in trial NCT00005934?
The interventions under investigation include: 5-Azacytidine (DRUG).
Who is sponsoring clinical trial NCT00005934?
This trial is sponsored by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), which has 490 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT00005934's enrollment target sits among peer trials
24 1756th of 2000 higher than 217 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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