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NCT00592540 · ClinicalTrials.gov registry record · NA
Unrelated Donor BMT for Treatment of Patients With PGK Deficiency
A NA study, sponsored by Vanderbilt University.
- Completed
- Registry status
- NA
- Development phase
- 2
- Enrollment target
NCT00592540: Completed NA study, sponsored by Vanderbilt University.
NCT00592540 is a NA study that has completed, run by Vanderbilt University. The registered enrollment target is 2 participants, below the 1,404-participant average among 58,714 other NA trials with a reported enrollment target (100% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00592540, a NA study, has completed, sponsored by Vanderbilt University.
- COMPLETED
- Registry status
- NA
- Development phase
- 2 participants
- Enrollment target
Study Summary
Phosphoglycerate kinase (PGK) deficiency is a rare x-linked disorder characterized by hemolytic anemia, seizures, muscle fatigue, and progressive neurological dysfunction. The disease is caused by the deficiency of PGK, an enzyme required for ATP formation through the glycolytic pathway. PGK is an enzyme that is ubiquitous to all cells of the human body, but red blood cells, muscles, and nerve cells are most severely affected by the absence of PGK due to their reliance upon the glycolytic pathway. Mutations of the PGK gene are highly variable and result in diverse phenotypes, ranging from mild hemolytic anemia only to severe mental retardation and early death in childhood. The more severe phenotypes show progressive neurologic deterioration between infancy and adolescence. This is a 2 patient study aimed at studying the role of stem cell transplant in PGK deficiency. Because the disease is so rare, the study will be limited to the 2 sibling patients followed by our group, though it would be open to other participants who would meet inclusion/exclusion criteria if such presented to us. The objective of this study is to evaluate the feasibility and efficacy of stem cell transplants to treat patients with PGK deficiency, Amiens subtype.
Interventions
- PROCEDURE Unrelated Donor BMT
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 2 participants |
| Start Date | 2006-06 |
| Est. Completion | 2011-02 |
| Phase | NA |
What the finished NCT00592540 record still lists
NCT00592540 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 2 participants, a relatively small participant target, below the 1,404-participant average among 58,714 other NA trials with a reported enrollment target (100% lower).
The record links to 0 conditions, and to 1 intervention - of which Unrelated Donor BMT is the first listed.
NCT00592540 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00592540 about?
NCT00592540 is a clinical study titled "Unrelated Donor BMT for Treatment of Patients With PGK Deficiency". Phosphoglycerate kinase (PGK) deficiency is a rare x-linked disorder characterized by hemolytic anemia, seizures, muscle fatigue, and progressive neurological dysfunction. The disease is caused by the deficiency of PGK, an enzyme required for ATP formation through the glycolytic pathway. PGK is an e...
What is the current status of trial NCT00592540?
This trial is currently completed. It is a NA study. The enrollment target is 2 participants. The study started on 2006-06. Estimated completion is 2011-02.
What interventions are being tested in trial NCT00592540?
The interventions under investigation include: Unrelated Donor BMT (PROCEDURE).
Who is sponsoring clinical trial NCT00592540?
This trial is sponsored by Vanderbilt University, which has 430 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT00592540's enrollment target sits among peer trials
2 1997th of 2000 the lowest of 2,000 other NA trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other NA 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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