Trial · NCT04269889
Treatment of Refractory Diamond-Blackfan Anemia With Eltrombopag
NCT04269889, Phase 1, Phase 2, Completed, National Heart, Lung, and Blood Institute (NHLBI); results first posted 2023-09-13
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Treatment of Refractory Diamond-Blackfan Anemia With Eltrombopag. Trials, record NCT04269889. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04269889
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Treatment of Refractory Diamond-Blackfan Anemia With Eltrombopag
- NCT number
- NCT04269889
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 15 participants, actual
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
- Conditions
- Anemia, Diamond-Blackfan
- Interventions
- Eltrombopag
- First posted
- 2020-02-17
- Last updated
- 2025-04-27
- Results first posted
- 2023-09-13
Source ClinicalTrials.gov
Registered outcomes12
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Participants That Responded to Eltrombopag | 6 months (24 weeks +/- 14 days) | Participants that responded to Eltrombopag as defined by: Response to treatment will be defined by one or more of the following: * Erythroid response for subjects with a pretreatment hemoglobin less than 9 G/dL will be defined as an increase in hemoglobin by \>1.5 G/dL from enrollment baseline, and/or * A reduction in the units of PRBC transfusions by at least 50% during the eight consecutive weeks prior to response assessment - compared with the pretreatment transfusion number in the previous 8 weeks. |
| Primary | Time (Weeks) to Response | 6 months (24 weeks +/- 14 days) | Time-to-response in weeks will also be measured according to the time from Eltrombopag initiation to the first time the patient met criteria for response. Response to treatment will be defined by one or more of the following: * Erythroid response for subjects with a pretreatment hemoglobin less than 9 G/dL will be defined as an increase in hemoglobin by \>1.5 G/dL from enrollment baseline, and/or * a reduction in the units of PRBC transfusions by at least 50% during the eight consecutive weeks prior to response assessment - compared with the pretreatment transfusion number in the previous 8 weeks. |
| Primary | Number of Adverse Events | 6 months (24 weeks +/- 14 days) | Number of Adverse Events Toxicity profile as measured by using the Common Terminology Criteria for Adverse Events (CTCAE version 5.0) for grade 2 and above. According to https://ctep.cancer.gov/, CTCAE is a descriptive terminology which can be utilized for Adverse Event (AE) reporting. A grading (severity) scale is provided for each AE term. CTCAE grades are defined as: Grade 2 Moderate; minimal, local or noninvasive intervention indicated; limiting age appropriate instrumental activities of daily living. Grade 3 Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self care activities of daily living. Grade 4 Life-threatening consequences; urgent intervention indicated. |
| Secondary | Number of Participants That Responded to Eltrombopag | 3 Months | Number of Participants That Responded to Eltrombopag. As determined by the response to treatment, which will be assessed based on one or more of the following criteria: Erythroid response for subjects with a pretreatment hemoglobin less than 9 G/dL will be defined as an increase in hemoglobin by \>1.5 G/dL from enrollment baseline, and/or A reduction in the units of PRBC transfusions by at least 50% during the eight consecutive weeks prior to response assessment - compared with the pretreatment transfusion number in the previous 8 weeks. |
| Secondary | Number of Participants With Robust Response to Eltrombopag | 3 Months, 6 Months, Up to 27 Months | Number of Participants with Robust Response to Eltrombopag regardless when it is achieved. Best response is defined as robust response or not robust response. Robust response to treatment will be defined both: * Stable hemoglobin (two consecutive measurements at least 2 weeks apart) greater than 10 g/dL, and * Transfusion independence for the previous 8 weeks |
| Secondary | Median Change in Platelet Count | Baseline, 3 months | Median Change in Platelet Count as measured by serial CBC assessments |
| Secondary | Median Change Absolute Neutrophil Count | Baseline, Month 3 | Median Change Absolute Neutrophil Count as measured by serial CBC assessments |
| Secondary | Number of Participants That Experienced Relapse | 6 months up to 27 months | Number of Participants that experienced Relapse during extension phase. Relapse is defined as no longer meeting response criteria. Response criteria is defined as: Response to treatment will be defined by one or more of the following: * Erythroid response for subjects with a pretreatment hemoglobin less than 9 G/dL will be defined as an increase in hemoglobin by \>1.5 G/dL from enrollment baseline, and/or * A reduction in the units of PRBC transfusions by at least 50% during the eight consecutive weeks prior to response assessment - compared with the pretreatment transfusion number in the previous 8 weeks. |
| Secondary | Number of Participants That Experienced Clonal Evolution | 6 Months and Up to 27 months | Number of participants that experienced clonal evolution according to cytogenetic, mutational or flow cytometric markers |
| Secondary | Number of Adverse Events During the Extension Phase | 6 months Up to 27 months | Number of Adverse Events Toxicity profile as measured by using the Common Terminology Criteria for Adverse Events (CTCAE version 5.0) for grade 2 and above. According to https://ctep.cancer.gov/, CTCAE is a descriptive terminology which can be utilized for Adverse Event (AE) reporting. A grading (severity) scale is provided for each AE term. CTCAE grades are defined as: Grade 2 Moderate; minimal, local or noninvasive intervention indicated; limiting age appropriate instrumental activities of daily living. Grade 3 Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self care activities of daily living. Grade 4 Life-threatening consequences; urgent intervention indicated. |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 registered outcomesOutcomes12
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Participants That Responded to Eltrombopag | 6 months (24 weeks +/- 14 days) | Participants | COUNT_OF_PARTICIPANTS | |
| PRIMARY | Time (Weeks) to Response | 6 months (24 weeks +/- 14 days) | Analysis includes only participants that responded to eltrombopag | weeks | NUMBER |
| PRIMARY | Number of Adverse Events | 6 months (24 weeks +/- 14 days) | Analysis includes number of adverse events with grade 2 events and above | Adverse Events | NUMBER |
| SECONDARY | Number of Participants That Responded to Eltrombopag | 3 Months | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Number of Participants With Robust Response to Eltrombopag | 3 Months, 6 Months, Up to 27 Months | Analysis post 6 months only includes those participants that met criteria for extension phase of the study | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Median Change in Platelet Count | Baseline, 3 months | Analysis includes those participants that completed baseline and month 3 visits | K/uL | MEDIAN |
| SECONDARY | Median Change Absolute Neutrophil Count | Baseline, Month 3 | Analysis includes those participants that completed baseline and month 3 visits | K/uL | MEDIAN |
| SECONDARY | Number of Participants That Experienced Relapse | 6 months up to 27 months | All participants that participated within the extended phase of the clinical trial | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants That Experienced Clonal Evolution | 6 Months and Up to 27 months | Analysis post 6 months only includes those participants that met criteria for extension phase of the study | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Adverse Events During the Extension Phase | 6 months Up to 27 months | All participants that participated within the extended phase of the clinical trial | Adverse Events | NUMBER |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 outcomesResults by group56
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Participants That Responded to Eltrombopag | Refractory Diamond-Blackfan Anemia in Eltrombopag | Response to Treatment | 1 | Participants | |
| Participants That Responded to Eltrombopag | Refractory Diamond-Blackfan Anemia in Eltrombopag | No Response to Treatment | 14 | Participants | |
| Time (Weeks) to Response | Refractory Diamond-Blackfan Anemia in Eltrombopag | 15 | weeks | ||
| Number of Adverse Events | Refractory Diamond-Blackfan Anemia in Eltrombopag With Grade 2 Adverse Events | Headache | 0 | Adverse Events | |
| Number of Adverse Events | Refractory Diamond-Blackfan Anemia in Eltrombopag With Grade 3 Adverse Events | Headache | 2 | Adverse Events | |
| Number of Adverse Events | Refractory Diamond-Blackfan Anemia in Eltrombopag With Grade 4 Adverse Events | Headache | 0 | Adverse Events | |
| Number of Adverse Events | Refractory Diamond-Blackfan Anemia in Eltrombopag With Grade 2 Adverse Events | Arthralgia | 1 | Adverse Events | |
| Number of Adverse Events | Refractory Diamond-Blackfan Anemia in Eltrombopag With Grade 3 Adverse Events | Arthralgia | 1 | Adverse Events | |
| Number of Adverse Events | Refractory Diamond-Blackfan Anemia in Eltrombopag With Grade 4 Adverse Events | Arthralgia | 0 | Adverse Events | |
| Number of Adverse Events | Refractory Diamond-Blackfan Anemia in Eltrombopag With Grade 2 Adverse Events | Back pain | 1 | Adverse Events |
1 to 10 of 56Source ClinicalTrials.gov
See all 56 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Initial Phase (Day 0 to Month 6) | STARTED | Refractory Diamond-Blackfan Anemia in Eltrombopag | 15 | |
| Initial Phase (Day 0 to Month 6) | COMPLETED | Refractory Diamond-Blackfan Anemia in Eltrombopag | 1 | |
| Initial Phase (Day 0 to Month 6) | NOT COMPLETED | Refractory Diamond-Blackfan Anemia in Eltrombopag | 14 | |
| Extended Phase (6 Months to Year 3) | STARTED | Refractory Diamond-Blackfan Anemia in Eltrombopag | 1 | |
| Extended Phase (6 Months to Year 3) | COMPLETED | Refractory Diamond-Blackfan Anemia in Eltrombopag | 1 | |
| Extended Phase (6 Months to Year 3) | NOT COMPLETED | Refractory Diamond-Blackfan Anemia in Eltrombopag | 0 |
Source ClinicalTrials.gov
Adverse events16
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 | Dysphagia | Gastrointestinal disorders | Refractory Diamond-Blackfan Anemia in Eltrombopag | 1 | 15 | 1 |
| other | Fever | General disorders | Refractory Diamond-Blackfan Anemia in Eltrombopag | 1 | 15 | 1 |
| other | Acute inception without sepsis/Acute viral syndrome | Infections and infestations | Refractory Diamond-Blackfan Anemia in Eltrombopag | 1 | 15 | 1 |
| other | Arthralgia | Musculoskeletal and connective tissue disorders | Refractory Diamond-Blackfan Anemia in Eltrombopag | 1 | 15 | 1 |
| other | Back pain | Musculoskeletal and connective tissue disorders | Refractory Diamond-Blackfan Anemia in Eltrombopag | 1 | 15 | 1 |
| other | Chills | Musculoskeletal and connective tissue disorders | Refractory Diamond-Blackfan Anemia in Eltrombopag | 1 | 15 | 1 |
| other | Myalgia | Musculoskeletal and connective tissue disorders | Refractory Diamond-Blackfan Anemia in Eltrombopag | 1 | 15 | 1 |
| other | Headache | Nervous system disorders | Refractory Diamond-Blackfan Anemia in Eltrombopag | 1 | 15 | 1 |
| serious | Nausea | Gastrointestinal disorders | Refractory Diamond-Blackfan Anemia in Eltrombopag | 1 | 15 | 1 |
| serious | Chills | General disorders | Refractory Diamond-Blackfan Anemia in Eltrombopag | 1 | 15 | 1 |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Refractory Diamond-Blackfan Anemia in Eltrombopag | 0 | 15 |
| Total, other adverse events | Refractory Diamond-Blackfan Anemia in Eltrombopag | 2 | 15 |
| Total, serious adverse events | Refractory Diamond-Blackfan Anemia in Eltrombopag | 2 | 15 |
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/NCT04269889
GET https://conductscience.org/api/trials/v1/NCT04269889/results?page=2