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NCT01362595 · ClinicalTrials.gov registry record · Phase 1
Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia
A Phase 1 study, sponsored by Northwell Health.
- Completed
- Registry status
- Phase 1
- Development phase
- 55
- Enrollment target
NCT01362595: Completed Phase 1 study, sponsored by Northwell Health.
NCT01362595 is a Phase 1 study that has completed, run by Northwell Health. The registered enrollment target is 55 participants, roughly in line with the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01362595, a Phase 1 study, has completed, sponsored by Northwell Health.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 55 participants
- Enrollment target
Study Summary
This study will determine the safety and possibility of giving the amino acid, leucine, in patients with Diamond Blackfan anemia(DBA)who are on dependent on red blood cell transfusions. The leucine is expected to produce a response in patients with DBA to the point where red blood cell production is increased. Red cell transfusions can then be less frequent or possibly discontinued. The investigators will study the side effects, if any, of giving leucine to DBA patients. Leucine levels of leucine will be obtained at baseline and during the study. The drug leucine will be provided in capsule form and taken 3 times a day for a total of 9 months.
Primary Outcome
The primary outcome is the type of response observed at 9 months. Response to treatment can be one of the following: 1. Complete response (CR): Hb \> 9 gm/dL and transfusion-independence as defined in DBA 2. Partial response (PR): Hb \< 9 gm/dL and increased reticulocyte count to greater than baseline. 3. No response (NR): no change in transfusion requirements and no significant change reticulocyte count from baseline 4. Progression: worsening of disease as defined by the need for more frequent
Interventions
- DRUG leucine
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 55 participants |
| Start Date | 2013-06 |
| Est. Completion | 2020-11-30 |
| Phase | Phase 1 |
What the finished NCT01362595 record still lists
NCT01362595 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 55 participants, a relatively small participant target, roughly in line with the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target.
The record links to 0 conditions, and to 1 intervention - of which leucine is the first listed.
NCT01362595 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01362595 about?
NCT01362595 is a clinical study titled "Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia". This study will determine the safety and possibility of giving the amino acid, leucine, in patients with Diamond Blackfan anemia(DBA)who are on dependent on red blood cell transfusions. The leucine is expected to produce a response in patients with DBA to the point where red blood cell production i...
What is the current status of trial NCT01362595?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 55 participants. The study started on 2013-06. Estimated completion is 2020-11-30.
What interventions are being tested in trial NCT01362595?
The interventions under investigation include: leucine (DRUG).
Who is sponsoring clinical trial NCT01362595?
This trial is sponsored by Northwell Health, which has 370 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT01362595's enrollment target sits among peer trials
55 918th of 2000 higher than 1,070 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 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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