Trial · NCT01362595
Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia
NCT01362595, Phase 1, Phase 2, Completed, Northwell Health; results first posted 2022-12-02
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia. Trials, record NCT01362595. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01362595
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia
- NCT number
- NCT01362595
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 55 participants, actual
- Lead sponsor
- Northwell Health
- Conditions
- Diamond Blackfan Anemia, Blackfan Diamond Syndrome, DBA, Congenital Hypoplastic Anemia, Pure Red Cell Aplasia
- Interventions
- leucine
- First posted
- 2011-05-30
- Last updated
- 2022-12-02
- Results first posted
- 2022-12-02
Source ClinicalTrials.gov
Registered outcomes2
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Response to Leucine in Transfusion Dependent Patients With Diamond Blackfan Anemia | 9 Months | 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 transfusions |
| Secondary | Severe Adverse Events Attributable to Leucine | 9 months | Adverse events occurring while participants were on Leucine |
Source ClinicalTrials.gov
Outcomes2
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Response to Leucine in Transfusion Dependent Patients With Diamond Blackfan Anemia | 9 Months | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Severe Adverse Events Attributable to Leucine | 9 months | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group6
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Response to Leucine in Transfusion Dependent Patients With Diamond Blackfan Anemia | Leucine | Complete Hematologic Response | 2 | Participants | |
| Response to Leucine in Transfusion Dependent Patients With Diamond Blackfan Anemia | Leucine | Partial hematologic response | 5 | Participants | |
| Response to Leucine in Transfusion Dependent Patients With Diamond Blackfan Anemia | Leucine | No hematologic response | 36 | Participants | |
| Response to Leucine in Transfusion Dependent Patients With Diamond Blackfan Anemia | Leucine | Progression | 0 | Participants | |
| Severe Adverse Events Attributable to Leucine | Leucine | No Adverse events | 41 | Participants | |
| Severe Adverse Events Attributable to Leucine | Leucine | Withdrew early | 2 | Participants |
Source ClinicalTrials.gov
Participant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Leucine | 55 | |
| Overall Study | COMPLETED | Leucine | 43 | |
| Overall Study | NOT COMPLETED | Leucine | 12 |
Source ClinicalTrials.gov
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Leucine | 0 | 43 |
| Total, other adverse events | Leucine | 0 | 43 |
| Total, serious adverse events | Leucine | 0 | 43 |
Source ClinicalTrials.gov
Papers50
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT01362595
GET https://conductscience.org/api/trials/v1/NCT01362595/papers?page=2