Trial · NCT01034592
Pilot Lenalidomide in Adult Diamond-Blackfan Anemia Patients w/ RBC Transfusion-Dependent Anemia
NCT01034592, Phase 1, Terminated, Jason Robert Gotlib; results first posted 2017-02-07
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Pilot Lenalidomide in Adult Diamond-Blackfan Anemia Patients w/ RBC Transfusion-Dependent Anemia. Trials, record NCT01034592. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01034592
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Pilot Lenalidomide in Adult Diamond-Blackfan Anemia Patients w/ RBC Transfusion-Dependent Anemia
- NCT number
- NCT01034592
- Status
- Terminated
- Phases
- Phase 1
- Enrollment
- 2 participants, actual
- Lead sponsor
- Jason Robert Gotlib
- Conditions
- Anemia, Leukemia, Acute Myeloid Leukemia (AML), Myelodysplastic Syndromes (MDS)
- Interventions
- Lenalidomide
- First posted
- 2009-12-17
- Last updated
- 2017-08-02
- Results first posted
- 2017-02-07
- Limitations and caveats
Only 2 subjects were enrolled on this protocol. Low accrual was related to various factors. Given the accrual status, no substantive conclusions about the efficacy or safety of lenalidomide in adults with Diamond-Blackfan Anemia could be reached.
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Red Blood Cell (RBC) Transfusion Independence | 6 months | Red blood cell (RBC) transfusion independence is reported as the number of subjects who achieve a continuous absence of the intravenous infusion of any RBC transfusion during any consecutive "rolling" 56 days during the treatment period. |
| Secondary | Red Blood Cell (RBC) Transfusions | 6 months | The effect on red blood cell (RBC) transfusions was assessed as the number of participants that achieved a greater than 50% decrease in RBC transfusion requirements. |
| Secondary | Hemoglobin Concentration | 6 months | The effect on hemoglobin concentration was assessed as the change from baseline, measured in g/dL. |
| Secondary | Neutrophil Response | 6 months | The effect on neutrophil levels was assessed as the change in neutrophil count from baseline. |
| Secondary | Platelet Response | 6 months | The effect on platelet levels as assessed as the change in platelet count from baseline. |
| Secondary | Duration of Response | 6 months | The response duration was measured from the last of the consecutive 56 days during which the subject was free of red blood cells (RBC) transfusions to the date of the first RBC transfusion after the 56-day RBC-transfusion-free period. |
| Secondary | Toxicity | 6 months | Toxicity was assessed as the number of adverse events related to lenalidomide. |
Source ClinicalTrials.gov
Outcomes7
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Red Blood Cell (RBC) Transfusion Independence | 6 months | All treated subjects were analyzed. | participants | NUMBER |
| SECONDARY | Red Blood Cell (RBC) Transfusions | 6 months | Both participants were assessed and contributed to the analysis. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Hemoglobin Concentration | 6 months | Both participants were assessed and contributed to the analysis. | g/dL | MEDIAN |
| SECONDARY | Neutrophil Response | 6 months | Both participants were assessed and contributed to the analysis. | 1000/uL | MEDIAN |
| SECONDARY | Platelet Response | 6 months | Both participants were assessed and contributed to the analysis. | 1000/uL | MEDIAN |
| SECONDARY | Duration of Response | 6 months | Both participants were assessed and contributed to the analysis, but never demonstrated any therapeutic response. | days | MEDIAN |
| SECONDARY | Toxicity | 6 months | Both participants were assessed and contributed to the analysis. | Related Adverse Events | NUMBER |
Source ClinicalTrials.gov
Results by group7
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Red Blood Cell (RBC) Transfusion Independence | Lenalidomide | 0 | participants | ||
| Red Blood Cell (RBC) Transfusions | Lenalidomide | 0 | Participants | ||
| Hemoglobin Concentration | Lenalidomide | 0.4 | Full Range 0.4 to 0.4 | g/dL | |
| Neutrophil Response | Lenalidomide | 0.50 | Full Range 0.10 to 0.91 | 1000/uL | |
| Platelet Response | Lenalidomide | 25.5 | Full Range -20.0 to 71.0 | 1000/uL | |
| Duration of Response | Lenalidomide | 0 | Full Range 0 to 0 | days | |
| Toxicity | Lenalidomide | 1 | Related Adverse Events |
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 | Lenalidomide | 2 | |
| Overall Study | COMPLETED | Lenalidomide | 2 | |
| Overall Study | NOT COMPLETED | Lenalidomide | 0 |
Source ClinicalTrials.gov
Adverse events6
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Fatigue | General disorders | Serious Adverse Events | 2 | 2 | 2 |
| other | Neutropenia | Blood and lymphatic system disorders | Serious Adverse Events | 1 | 2 | 1 |
| other | Ear Infection | Infections and infestations | Serious Adverse Events | 1 | 2 | 1 |
| other | Aspartate aminotransferase, increased | Investigations | Serious Adverse Events | 1 | 2 | 1 |
| other | Alanine aminotransferase, increased | Investigations | Serious Adverse Events | 1 | 2 | 1 |
| other | Dry cough | Respiratory, thoracic and mediastinal disorders | Serious Adverse Events | 1 | 2 | 1 |
Source ClinicalTrials.gov
Adverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Serious Adverse Events | ||
| Total, other adverse events | Serious Adverse Events | 2 | 2 |
| Total, serious adverse events | Serious Adverse Events | 0 | 2 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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/NCT01034592
GET https://conductscience.org/api/trials/v1/NCT01034592/registered