Trial · NCT04304820
Early Initiation of Oral Therapy With Cyclosporine and Eltrombopag for Treatment Naive Severe Aplastic Anemia (SAA)
NCT04304820, Phase 2, Recruiting, National Heart, Lung, and Blood Institute (NHLBI); results first posted 2026-01-09
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Early Initiation of Oral Therapy With Cyclosporine and Eltrombopag for Treatment Naive Severe Aplastic Anemia (SAA). Trials, record NCT04304820. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04304820
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Early Initiation of Oral Therapy With Cyclosporine and Eltrombopag for Treatment Naive Severe Aplastic Anemia (SAA)
- NCT number
- NCT04304820
- Status
- Recruiting
- Phases
- Phase 2
- Enrollment
- 80 participants, estimated
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
- Conditions
- Severe Aplastic Anemia
- Interventions
- Eltrombopag, Cyclosporine, Horse-Anti-thymocyte-Globulin
- First posted
- 2020-03-11
- Last updated
- 2026-03-09
- Results first posted
- 2026-01-09
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants Meeting Safety and Feasibility Criteria During Study Treatment Period With Oral Treatment (Low Dose Cyclosporine and Eltrombopag) Until the Start of Standard of Care Treatment (Horse Anti-thymocyte-globulin) | Up to 12 Weeks from the initiation of oral treatment (Low Dose Cyclosporine and Eltrombopag) | For the primary endpoint of this study, the treatment is defined to be "feasible" to a participant if he/she meets the safety and feasibility criteria. Safety and feasibility criteria is a composite measure defined as TRSAE, mis- and altered diagnosis, and non-compliance with the regimen or failure to establish care at the National Institutes of Health Clinical Center. Study treatment period is defined as the duration from the initiation of oral treatment (low dose Cyclosporine and Eltrombopag) to the start of standard of care treatment (Day 1 of horse anti-thymocyte-globulin). |
| Secondary | Hematological Response | At 1, 2, 3, 6 and 12 months and yearly thereafter | |
| Secondary | Relapse | At 1, 2, 3, 6 and 12 months and yearly thereafter | |
| Secondary | Clonal Evolution to PNH, Clonal Chromosomal Population in Bone Marrow, Myelodysplasia by Morphology, or Acute Leukemia | At 1, 2, 3, 6 and 12 months and yearly thereafter | |
| Secondary | Overall Survival | At 5 Years (60 Months) | |
| Secondary | Hematological Response of Relapse Subjects That Re-start Treatment With Cyclosporine and/or Eltrombopag | At 1, 2, 3, 6 and 12 months and yearly thereafter | |
| Secondary | Freedom From h-ATG | At 1, 2, 3, 6 and 12 months and yearly thereafter |
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 | Number of Participants Meeting Safety and Feasibility Criteria During Study Treatment Period With Oral Treatment (Low Dose Cyclosporine and Eltrombopag) Until the Start of Standard of Care Treatment (Horse Anti-thymocyte-globulin) | Up to 12 Weeks from the initiation of oral treatment (Low Dose Cyclosporine and Eltrombopag) | analysis was intention-to-treat | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Hematological Response | At 1, 2, 3, 6 and 12 months and yearly thereafter | |||
| SECONDARY | Relapse | At 1, 2, 3, 6 and 12 months and yearly thereafter | |||
| SECONDARY | Clonal Evolution to PNH, Clonal Chromosomal Population in Bone Marrow, Myelodysplasia by Morphology, or Acute Leukemia | At 1, 2, 3, 6 and 12 months and yearly thereafter | |||
| SECONDARY | Overall Survival | At 5 Years (60 Months) | |||
| SECONDARY | Hematological Response of Relapse Subjects That Re-start Treatment With Cyclosporine and/or Eltrombopag | At 1, 2, 3, 6 and 12 months and yearly thereafter | |||
| SECONDARY | Freedom From h-ATG | At 1, 2, 3, 6 and 12 months and yearly thereafter |
Source ClinicalTrials.gov
Results by group1
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
Number of Participants Meeting Safety and Feasibility Criteria During Study Treatment Period With Oral Treatment (Low Dose Cyclosporine and Eltrombopag) Until the Start of Standard of Care Treatment (Horse Anti-thymocyte-globulin) | Participants With Severe Aplastic Anemia in Early Initiation of Cyclosporine and Eltrombopag | 37 | 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 | Participants With Severe Aplastic Anemia in Early Initiation of Cyclosporine and Eltrombopag | 39 | |
| Overall Study | COMPLETED | Participants With Severe Aplastic Anemia in Early Initiation of Cyclosporine and Eltrombopag | 28 | |
| Overall Study | NOT COMPLETED | Participants With Severe Aplastic Anemia in Early Initiation of Cyclosporine and Eltrombopag | 11 |
Source ClinicalTrials.gov
Adverse events305
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 | Anemia | Blood and lymphatic system disorders | Participants With Severe Aplastic Anemia in Early Initiation of Cyclosporine and Eltrombopag | 5 | 39 | 6 |
| other | Febrile neutropenia | Blood and lymphatic system disorders | Participants With Severe Aplastic Anemia in Early Initiation of Cyclosporine and Eltrombopag | 13 | 39 | 17 |
| other | Chest pain - cardiac | Cardiac disorders | Participants With Severe Aplastic Anemia in Early Initiation of Cyclosporine and Eltrombopag | 1 | 39 | 1 |
| other | Chest pressure (right) | Cardiac disorders | Participants With Severe Aplastic Anemia in Early Initiation of Cyclosporine and Eltrombopag | 1 | 39 | 1 |
| other | Chest Tighness | Cardiac disorders | Participants With Severe Aplastic Anemia in Early Initiation of Cyclosporine and Eltrombopag | 1 | 39 | 1 |
| other | Hypotension | Cardiac disorders | Participants With Severe Aplastic Anemia in Early Initiation of Cyclosporine and Eltrombopag | 1 | 39 | 1 |
| other | Non-cardiac chest pain | Cardiac disorders | Participants With Severe Aplastic Anemia in Early Initiation of Cyclosporine and Eltrombopag | 1 | 39 | 1 |
| other | Palpitations | Cardiac disorders | Participants With Severe Aplastic Anemia in Early Initiation of Cyclosporine and Eltrombopag | 2 | 39 | 2 |
| other | Paroxysmal atrial tachycardia | Cardiac disorders | Participants With Severe Aplastic Anemia in Early Initiation of Cyclosporine and Eltrombopag | 1 | 39 | 1 |
| other | Right ventricular dysfunction | Cardiac disorders | Participants With Severe Aplastic Anemia in Early Initiation of Cyclosporine and Eltrombopag | 1 | 39 | 1 |
1 to 10 of 305Source ClinicalTrials.gov
See all 305 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Participants With Severe Aplastic Anemia in Early Initiation of Cyclosporine and Eltrombopag | 1 | 39 |
| Total, other adverse events | Participants With Severe Aplastic Anemia in Early Initiation of Cyclosporine and Eltrombopag | 39 | 39 |
| Total, serious adverse events | Participants With Severe Aplastic Anemia in Early Initiation of Cyclosporine and Eltrombopag | 24 | 39 |
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/NCT04304820
GET https://conductscience.org/api/trials/v1/NCT04304820/events?page=2