Trial · NCT01187017
A Pilot Study of Fludarabine Plus Cyclophosphamide in Refractory Severe Aplastic Anemia
NCT01187017, Phase 1, Phase 2, Completed, National Heart, Lung, and Blood Institute (NHLBI); results first posted 2016-03-02
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. A Pilot Study of Fludarabine Plus Cyclophosphamide in Refractory Severe Aplastic Anemia. Trials, record NCT01187017. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01187017
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Pilot Study of Fludarabine Plus Cyclophosphamide in Refractory Severe Aplastic Anemia
- NCT number
- NCT01187017
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 1 participants, actual
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
- Conditions
- Aplastic Anemia, Neutropenia, Pancytopenia, Severe Aplastic Anemia
- Interventions
- Cyclophosphamide, Fludarabine
- First posted
- 2010-08-23
- Last updated
- 2021-07-02
- Results first posted
- 2016-03-02
Source ClinicalTrials.gov
Registered outcomes5
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Response Rate at 6 Months | 6 months | The primary objective is to assess hematologic response of Refractory Severe aplastic anemia (SAA) subjects to who have received fludarabine and cyclophosphamide. Subjects blood counts will be evaluated at 6 months to assess a hematologic response. The hematologic response will be defined as complete, partial or no response. |
| Secondary | Number of Participants With Hematologic Response | 3 months | Number of participants with hematologic response at 3 and 12 months and yearly. The hematologic response will be defined as complete, partial or no response. |
| Secondary | Number of Patients Who Experienced Disease Relapse | 6 months | Number of patients who relapsed who experienced disease relapse following Fludarabine Plus Cyclophosphamide in Refractory Severe Aplastic Anemia |
| Secondary | Number of Participants With Clonal Evolution | 6 months | Number of participants with Severe Aplastic Anemia who received Fludarabine Plus Cyclophosphamide that experienced Clonal Evolution to Paroxysmal Nocturnal Hemoglobinuria (PNH), Myelodysplasia or Acute Leukemia. |
| Secondary | Participant Survival Following Fludarabine/ Cyclophosphamide in Participants With Severe Aplastic Anemia | 6 months | Participant survival at 6 months following Fludarabine/ Cyclophosphamide in participants with Severe Aplastic Anemia |
Source ClinicalTrials.gov
Outcomes5
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Response Rate at 6 Months | 6 months | There was 1 participant enrolled in this study. | participants | NUMBER |
| SECONDARY | Number of Participants With Hematologic Response | 3 months | 1 participant was enrolled in this study. Patient was taken off study for lack of efficacy at month 6. No data is available to report past month 6. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Patients Who Experienced Disease Relapse | 6 months | 1 participant was enrolled in this study. Patient was taken off study for lack of efficacy at month 6. No data is available to report past month 6. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Clonal Evolution | 6 months | 1 participant was enrolled in this study. Patient was taken off study for lack of efficacy at month 6. No data is available to report past month 6. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Participant Survival Following Fludarabine/ Cyclophosphamide in Participants With Severe Aplastic Anemia | 6 months | 1 participant was enrolled in this study. Patient was taken off study for lack of efficacy at month 6. No data is available to report past month 6. | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group9
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Response Rate at 6 Months | Fludarabine/Cyclophosphamide in Participants With Severe Aplastic Anemia | No Response | 1 | participants | |
| Response Rate at 6 Months | Fludarabine/Cyclophosphamide in Participants With Severe Aplastic Anemia | Partial Response | 0 | participants | |
| Response Rate at 6 Months | Fludarabine/Cyclophosphamide in Participants With Severe Aplastic Anemia | Complete Response | 0 | participants | |
| Number of Participants With Hematologic Response | Fludarabine/Cyclophosphamide in Participants With Severe Aplastic Anemia at 3 Months | Complete Response | 0 | Participants | |
| Number of Participants With Hematologic Response | Fludarabine/Cyclophosphamide in Participants With Severe Aplastic Anemia at 3 Months | Partial Response | 0 | Participants | |
| Number of Participants With Hematologic Response | Fludarabine/Cyclophosphamide in Participants With Severe Aplastic Anemia at 3 Months | No Response | 1 | Participants | |
| Number of Patients Who Experienced Disease Relapse | Fludarabine/ Cyclophosphamide in Participants With Severe Aplastic Anemia | 0 | Participants | ||
| Number of Participants With Clonal Evolution | Fludarabine/ Cyclophosphamide in Participants With Severe Aplastic Anemia | 0 | Participants | ||
| Participant Survival Following Fludarabine/ Cyclophosphamide in Participants With Severe Aplastic Anemia | Fludarabine/ Cyclophosphamide in Participants With Severe Aplastic Anemia | 1 | 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 | Fludarabine/Cyclophosphamide in Participants With Severe Aplastic Anemia | 1 | |
| Overall Study | COMPLETED | Fludarabine/Cyclophosphamide in Participants With Severe Aplastic Anemia | 1 | |
| Overall Study | NOT COMPLETED | Fludarabine/Cyclophosphamide in Participants With Severe Aplastic Anemia | 0 |
Source ClinicalTrials.gov
Adverse events2
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 | infection | Infections and infestations | Fludarabine/Cyclophosphamide in Participants With Severe Aplastic Anemia | 1 | 1 | 1 |
| serious | neutropenic fever | Infections and infestations | Fludarabine/Cyclophosphamide in Participants With Severe Aplastic Anemia | 1 | 1 | 2 |
Source ClinicalTrials.gov
Adverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Fludarabine/Cyclophosphamide in Participants With Severe Aplastic Anemia | 0 | 1 |
| Total, other adverse events | Fludarabine/Cyclophosphamide in Participants With Severe Aplastic Anemia | 0 | 1 |
| Total, serious adverse events | Fludarabine/Cyclophosphamide in Participants With Severe Aplastic Anemia | 1 | 1 |
Source ClinicalTrials.gov
Papers3
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/NCT01187017
GET https://conductscience.org/api/trials/v1/NCT01187017/registered