Trial · NCT01623167
Eltrombopag With Standard Immunosuppression for Severe Aplastic Anemia
NCT01623167, Phase 1, Phase 2, Active not recruiting, National Heart, Lung, and Blood Institute (NHLBI); results first posted 2020-06-05
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Eltrombopag With Standard Immunosuppression for Severe Aplastic Anemia. Trials, record NCT01623167. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01623167
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Eltrombopag With Standard Immunosuppression for Severe Aplastic Anemia
- NCT number
- NCT01623167
- Status
- Active not recruiting
- Phases
- Phase 1, Phase 2
- Enrollment
- 207 participants, actual
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
- Conditions
- Severe Aplastic Anemia
- Interventions
- Cohort 1: hATG, CsA, EPAG Day 14 to Month 6, Cohort 2: hATG, CsA, EPAG Day 14 to Month 3, Cohort 3: hATG, CsA (dose reduced), EPAG day 1 to month 6, Extension Cohort
- First posted
- 2012-06-19
- Last updated
- 2026-04-13
- Results first posted
- 2020-06-05
Source ClinicalTrials.gov
Registered outcomes2
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Rate of Complete Hematologic Response | 6 months | Rate of complete hematologic response at six months for cohorts 1, 2 and 3. |
| Secondary | Rate of Response at 3 and 12 Months Then Yearly; Rate of Relapse; Rate of Clonal Evolution to PNH, MDS and AML; Rate of Survival; Rate of Response for Relapse Subjects That Re-start Treatment and Effects of CsA Dose Starting at Month 6 to Month 24. | 3 months to 5 years | Secondary endpoints will also be evaluated for the study to include: (a) hematological response at 3 and 12 months and yearly thereafter; (b) relapse (c) clonal evolution to PNH, clonal chromosomal population in bone marrow, myelodysplasia by morphology, or acute leukemia; (d) survival; (e) health-related quality of life; (f) hematological response of relapse subjects that re-start treatment; and (g) affects of a 2.0mg/kg/day CsA dose starting month 6 for 18 months until month 24 on the rate of relapse of subjects deemed responders at month 6. |
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 | Rate of Complete Hematologic Response | 6 months | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Rate of Response at 3 and 12 Months Then Yearly; Rate of Relapse; Rate of Clonal Evolution to PNH, MDS and AML; Rate of Survival; Rate of Response for Relapse Subjects That Re-start Treatment and Effects of CsA Dose Starting at Month 6 to Month 24. | 3 months to 5 years |
Source ClinicalTrials.gov
Results by group12
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Rate of Complete Hematologic Response | hATG, CsA, EPAG Day 14 to Month 6 | No Response | 3 | Participants | |
| Rate of Complete Hematologic Response | hATG, CsA, EPAG Day 14 to Month 3 | No Response | 2 | Participants | |
| Rate of Complete Hematologic Response | hATG, CsA (Dose Reduced), EPAG Day 1 to Month 6 | No Response | 1 | Participants | |
| Rate of Complete Hematologic Response | hATG, CsA, EPAG Day 14 to Month 6 | Partial Response | 14 | Participants | |
| Rate of Complete Hematologic Response | hATG, CsA, EPAG Day 14 to Month 3 | Partial Response | 19 | Participants | |
| Rate of Complete Hematologic Response | hATG, CsA (Dose Reduced), EPAG Day 1 to Month 6 | Partial Response | 11 | Participants | |
| Rate of Complete Hematologic Response | hATG, CsA, EPAG Day 14 to Month 6 | Complete Response | 10 | Participants | |
| Rate of Complete Hematologic Response | hATG, CsA, EPAG Day 14 to Month 3 | Complete Response | 8 | Participants | |
| Rate of Complete Hematologic Response | hATG, CsA (Dose Reduced), EPAG Day 1 to Month 6 | Complete Response | 18 | Participants | |
| Rate of Complete Hematologic Response | hATG, CsA, EPAG Day 14 to Month 6 | Off study/Not Evaluable | 3 | Participants |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 results by groupParticipant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | hATG, CsA, EPAG Day 14 to Month 6 | 30 | |
| Overall Study | STARTED | hATG, CsA, EPAG Day 14 to Month 3 | 31 | |
| Overall Study | STARTED | hATG, CsA (Dose Reduced), EPAG Day 1 to Month 6 | 31 | |
| Overall Study | COMPLETED | hATG, CsA, EPAG Day 14 to Month 6 | 13 | |
| Overall Study | COMPLETED | hATG, CsA, EPAG Day 14 to Month 3 | 17 | |
| Overall Study | COMPLETED | hATG, CsA (Dose Reduced), EPAG Day 1 to Month 6 | 18 | |
| Overall Study | NOT COMPLETED | hATG, CsA, EPAG Day 14 to Month 6 | 17 | |
| Overall Study | NOT COMPLETED | hATG, CsA, EPAG Day 14 to Month 3 | 14 | |
| Overall Study | NOT COMPLETED | hATG, CsA (Dose Reduced), EPAG Day 1 to Month 6 | 13 |
Source ClinicalTrials.gov
Adverse events489
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 | Abdominal pain | Gastrointestinal disorders | hATG, CsA, EPAG Day 14 to Month 6 | 2 | 30 | 2 |
| other | Abdominal pain | Gastrointestinal disorders | hATG, CsA, EPAG Day 14 to Month 3 | 1 | 31 | 1 |
| other | Abdominal pain | Gastrointestinal disorders | hATG, CsA (Dose Reduced), EPAG Day 1 to Month 6 | 1 | 31 | 1 |
| other | Acute kidney injury | Renal and urinary disorders | hATG, CsA, EPAG Day 14 to Month 6 | 0 | 30 | 0 |
| other | Acute kidney injury | Renal and urinary disorders | hATG, CsA, EPAG Day 14 to Month 3 | 1 | 31 | 1 |
| other | Acute kidney injury | Renal and urinary disorders | hATG, CsA (Dose Reduced), EPAG Day 1 to Month 6 | 0 | 31 | 0 |
| other | Alanine aminotransferase increased | Investigations | hATG, CsA, EPAG Day 14 to Month 6 | 3 | 30 | 3 |
| other | Alanine aminotransferase increased | Investigations | hATG, CsA, EPAG Day 14 to Month 3 | 8 | 31 | 17 |
| other | Alanine aminotransferase increased | Investigations | hATG, CsA (Dose Reduced), EPAG Day 1 to Month 6 | 11 | 31 | 17 |
| other | Alanine aminotransferase increased (intermittent) | Investigations | hATG, CsA, EPAG Day 14 to Month 6 | 0 | 30 | 0 |
1 to 10 of 489Source ClinicalTrials.gov
See all 489 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | hATG, CsA, EPAG Day 14 to Month 6 | 1 | 30 |
| Total, all-cause mortality | hATG, CsA, EPAG Day 14 to Month 3 | 0 | 31 |
| Total, all-cause mortality | hATG, CsA (Dose Reduced), EPAG Day 1 to Month 6 | 0 | 31 |
| Total, other adverse events | hATG, CsA, EPAG Day 14 to Month 6 | 30 | 30 |
| Total, other adverse events | hATG, CsA, EPAG Day 14 to Month 3 | 31 | 31 |
| Total, other adverse events | hATG, CsA (Dose Reduced), EPAG Day 1 to Month 6 | 31 | 31 |
| Total, serious adverse events | hATG, CsA, EPAG Day 14 to Month 6 | 17 | 30 |
| Total, serious adverse events | hATG, CsA, EPAG Day 14 to Month 3 | 15 | 31 |
| Total, serious adverse events | hATG, CsA (Dose Reduced), EPAG Day 1 to Month 6 | 14 | 31 |
Source ClinicalTrials.gov
Papers12
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/NCT01623167
GET https://conductscience.org/api/trials/v1/NCT01623167/events?page=2