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NCT00001749 · ClinicalTrials.gov registry record · Phase 2

Medical Treatment for Diamond Blackfan Anemia

A Phase 2 study, sponsored by National Heart, Lung, and Blood Institute (NHLBI).

Completed
Registry status
Phase 2
Development phase
25
Enrollment target

NCT00001749: Completed Phase 2 study, sponsored by National Heart, Lung, and Blood Institute (NHLBI).

NCT00001749 is a Phase 2 study that has completed, run by National Heart, Lung, and Blood Institute (NHLBI). The registered enrollment target is 25 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (81% lower). According to ClinicalTrials.gov, the official US trial registry.

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The verdict

NCT00001749, a Phase 2 study, has completed, sponsored by National Heart, Lung, and Blood Institute (NHLBI).

COMPLETED
Registry status
Phase 2
Development phase
25 participants
Enrollment target

Study Summary

Diamond Blackfan anemia (DBA) is a condition in which the bone marrow is underdeveloped. DBA is considered a congenital disease, meaning patients are born with it. In DBA there is a lack of cells that give rise to red blood cells. The other elements produced in the bone marrow, such as white blood cells and platelets, are normal. Standard treatments used for this disorder such as steroids and bone marrow transplants are associated with failure, relapse, side-effects, increased morbidity, and even death. Two drugs, antithymocyte globulin (ATG) and cyclosporin have been used to treat DBA, but have only provided occasional responses. No study has ever combined these two drugs for the treatment of DBA. This study is designed to explore the combined use of ATG and cyclosporine as a rational approach to the treatment of DBA.

Interventions

  • DRUG Cyclosporine
  • DRUG Antithymocyte globulin

Trial Details

FieldValue
Enrollment Target 25 participants
Start Date 1998-07
Est. Completion 2005-07
Phase Phase 2

What the finished NCT00001749 record still lists

NCT00001749 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 25 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (81% lower).

The record links to 0 conditions, and to 2 interventions - of which Cyclosporine is the first listed.

NCT00001749 does not publish any study locations in the registry export this page uses.

Frequently Asked Questions

What is clinical trial NCT00001749 about?

NCT00001749 is a clinical study titled "Medical Treatment for Diamond Blackfan Anemia". Diamond Blackfan anemia (DBA) is a condition in which the bone marrow is underdeveloped. DBA is considered a congenital disease, meaning patients are born with it. In DBA there is a lack of cells that give rise to red blood cells. The other elements produced in the bone marrow, such as white blood c...

What is the current status of trial NCT00001749?

This trial is currently completed. It is a Phase 2 study. The enrollment target is 25 participants. The study started on 1998-07. Estimated completion is 2005-07.

What interventions are being tested in trial NCT00001749?

The interventions under investigation include: Cyclosporine (DRUG), Antithymocyte globulin (DRUG).

Who is sponsoring clinical trial NCT00001749?

This trial is sponsored by National Heart, Lung, and Blood Institute (NHLBI), which has 501 total clinical trials registered on ClinicalTrials.gov.

How this trial's enrollment target compares

Where NCT00001749's enrollment target sits among peer trials

25 1719th of 2000 higher than 245 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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Source: ClinicalTrials.gov NCT00001749, the US trial registry maintained by the National Library of Medicine. NCT00001749 (small enrollment · none site footprint · completed) retrieved and formatted by PlainTrial, see methodology.