Trial · NCT01464164
Safety and Efficacy Study of Sotatercept in Adults With Transfusion Dependent Diamond Blackfan Anemia
NCT01464164, Phase 1, Phase 2, Terminated, Northwell Health; results first posted 2021-12-21
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Safety and Efficacy Study of Sotatercept in Adults With Transfusion Dependent Diamond Blackfan Anemia. Trials, record NCT01464164. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01464164
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Safety and Efficacy Study of Sotatercept in Adults With Transfusion Dependent Diamond Blackfan Anemia
- NCT number
- NCT01464164
- Status
- Terminated
- Phases
- Phase 1, Phase 2
- Enrollment
- 19 participants, actual
- Lead sponsor
- Northwell Health
- Conditions
- Diamond Blackfan Anemia
- Interventions
- Sotatercept, Sotatercept with prednisone boost
- First posted
- 2011-11-03
- Last updated
- 2023-03-17
- Results first posted
- 2021-12-21
- Limitations and caveats
Early termination of 3 subjects lead to small numbers of subjects analyzed, as does the early closure of the study.~Time to response was unable to be evaluated as no subjects attained response. Response duration was also not evaluated as no subjects responded.
Source ClinicalTrials.gov
Registered outcomes2
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Complete Response and Partial Response | 9 months | Complete response is transfusion independence with hemoglobin \>9 gm/dl; partial response is transfusion dependence with hemoglobin \< 9gm/dl with an increase in reticulocyte count over baseline |
| Secondary | Severe Adverse Events Attributable to Study Drug | 9 months | Assess severity of adverse events and relationship to sotatercept according to the currently active minor version of the NCI Common Terminology for Adverse Events version 4.0 |
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 | Number of Participants With Complete Response and Partial Response | 9 months | Only subjects who completed the trial are analyzed. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Severe Adverse Events Attributable to Study Drug | 9 months | Only subjects who completed the trial are analyzed. | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group14
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Complete Response and Partial Response | Sotatercept Cohort 1 | 0 | Participants | ||
| Number of Participants With Complete Response and Partial Response | Sotatercept Cohort 2 | 0 | Participants | ||
| Number of Participants With Complete Response and Partial Response | Sotatercept Cohort 3 | 0 | Participants | ||
| Number of Participants With Complete Response and Partial Response | Sotatercept With Prednisone Boost Cohort 4a | 0 | Participants | ||
| Number of Participants With Complete Response and Partial Response | Sotatercept Cohort 4b | 0 | Participants | ||
| Number of Participants With Complete Response and Partial Response | Sotatercept With Prednisone Boost Cohort 5a | 0 | Participants | ||
| Number of Participants With Complete Response and Partial Response | Sotatercept Cohort 5b | 0 | Participants | ||
| Severe Adverse Events Attributable to Study Drug | Sotatercept Cohort 1 | 0 | Participants | ||
| Severe Adverse Events Attributable to Study Drug | Sotatercept Cohort 2 | 0 | Participants | ||
| Severe Adverse Events Attributable to Study Drug | Sotatercept Cohort 3 | 0 | Participants |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 results by groupParticipant flow21
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Sotatercept Cohort 1 | 3 | |
| Overall Study | STARTED | Sotatercept Cohort 2 | 4 | |
| Overall Study | STARTED | Sotatercept Cohort 3 | 4 | |
| Overall Study | STARTED | Sotatercept With Prednisone Boost Cohort 4a | 2 | |
| Overall Study | STARTED | Sotatercept Cohort 4b | 3 | |
| Overall Study | STARTED | Sotatercept With Prednisone Boost Cohort 5a | 0 | |
| Overall Study | STARTED | Sotatercept Cohort 5b | 3 | |
| Overall Study | COMPLETED | Sotatercept Cohort 1 | 3 | |
| Overall Study | COMPLETED | Sotatercept Cohort 2 | 2 | |
| Overall Study | COMPLETED | Sotatercept Cohort 3 | 2 |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 participant flowAdverse events21
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 |
|---|---|---|---|---|---|---|
| serious | Hospitalization | Infections and infestations | Sotatercept Cohort 1 | 0 | 3 | 0 |
| serious | Hospitalization | Infections and infestations | Sotatercept Cohort 2 | 1 | 2 | 1 |
| serious | Hospitalization | Infections and infestations | Sotatercept Cohort 3 | 0 | 2 | 0 |
| serious | Hospitalization | Infections and infestations | Sotatercept With Prednisone Boost Cohort 4a | 0 | 2 | 0 |
| serious | Hospitalization | Infections and infestations | Sotatercept Cohort 4b | 0 | 3 | 0 |
| serious | Hospitalization | Infections and infestations | Sotatercept With Prednisone Boost Cohort 5a | 0 | 0 | 0 |
| serious | Hospitalization | Infections and infestations | Sotatercept Cohort 5b | 0 | 1 | 0 |
| serious | Hospitalization | Endocrine disorders | Sotatercept Cohort 1 | 0 | 3 | 0 |
| serious | Hospitalization | Endocrine disorders | Sotatercept Cohort 2 | 1 | 2 | 1 |
| serious | Hospitalization | Endocrine disorders | Sotatercept Cohort 3 | 0 | 2 | 0 |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 adverse eventsAdverse event totals21
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Sotatercept Cohort 1 | 0 | 3 |
| Total, all-cause mortality | Sotatercept Cohort 2 | 0 | 2 |
| Total, all-cause mortality | Sotatercept Cohort 3 | 0 | 2 |
| Total, all-cause mortality | Sotatercept With Prednisone Boost Cohort 4a | 0 | 2 |
| Total, all-cause mortality | Sotatercept Cohort 4b | 0 | 3 |
| Total, all-cause mortality | Sotatercept With Prednisone Boost Cohort 5a | 0 | 0 |
| Total, all-cause mortality | Sotatercept Cohort 5b | 0 | 1 |
| Total, other adverse events | Sotatercept Cohort 1 | 0 | 3 |
| Total, other adverse events | Sotatercept Cohort 2 | 0 | 2 |
| Total, other adverse events | Sotatercept Cohort 3 | 0 | 2 |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 adverse event totalsPapers30
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/NCT01464164
GET https://conductscience.org/api/trials/v1/NCT01464164/flow?page=2