Trial · NCT05451940
Hydroxyurea and EPO in Sickle Cell Disease
NCT05451940, Phase 1, Phase 2, Completed, Julia Xu; results first posted 2026-01-07
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Hydroxyurea and EPO in Sickle Cell Disease. Trials, record NCT05451940. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05451940
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Hydroxyurea and EPO in Sickle Cell Disease
- NCT number
- NCT05451940
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 17 participants, actual
- Lead sponsor
- Julia Xu
- Conditions
- Anemia, Sickle Cell, Sickle Cell Disease
- Interventions
- Hydroxyurea, Epoetin Alfa-BioSimilar
- First posted
- 2022-07-11
- Last updated
- 2026-01-07
- Results first posted
- 2026-01-07
- Limitations and caveats
The sponsor provided the study drug (EPO) for the duration of the treatment period (12 weeks). However, the sponsor was not able to provide study drug for a longer study, and participants who wanted to continue on EPO treatment during the observation period had to pay out of pocket or get insurance coverage. Thus, most participants had to discontinue EPO after the treatment period. The long-term safety of EPO treatment in patients with sickle cell disease cannot be evaluated from this study.
Source ClinicalTrials.gov
Registered outcomes13
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants With Hemoglobin (Hb) Response | Baseline to 12 weeks | Hb response is defined as a Hb increase of ≥ 1.0 g/dL at 12 weeks compared to baseline |
| Secondary | Change in Number of Blood Transfusions Per Year | Baseline to 12 weeks | Annualized number of units of simple red blood cell transfusions received in the 12 months before treatment initiation, compared to during the treatment period |
| Other | Changes in Tricuspid Valve Regurgitant Jet Velocity as Assessed by Echocardiography | Baseline to 12 weeks; Baseline to 24 weeks | Changes in tricuspid valve regurgitant jet velocity |
| Other | Changes in Cardiac Index as Assessed by Echocardiography | Baseline to 12 weeks; Baseline to 24 weeks | Changes in cardiac index |
| Other | Changes in Left Ventricular End-diastolic Volume as Assessed by Echocardiography | Baseline to 12 weeks; Baseline to 24 weeks | Changes in left ventricular end-diastolic volume |
| Other | Changes in Exercise Capacity as Assessed by 6-minute Walk Test With Modified Borg Dyspnea Scale | Baseline to 12 weeks; Baseline to 24 weeks | Changes in 6-minute walk distance and Modified Borg Dyspnea scale (severity of dyspnea during the 6-minute walk test will be measured on a 10-point scale with 0=nothing at all and 10= maximum severity of breathlessness) |
| Other | Changes in Hemoglobin (Hb) | Baseline to 12 weeks | Mean change in Hb at 12 weeks compared to baseline |
| Other | Changes in Absolute Reticulocyte Count | Baseline to 12 weeks; Baseline to 24 weeks | Changes in absolute reticulocyte count |
| Other | Changes in Lactate Dehydrogenase | Baseline to 12 weeks; Baseline to 24 weeks | Changes in lactate dehydrogenase |
| Other | Changes in Renal Function | Baseline to 12 weeks; Baseline to 24 weeks | Changes in serum creatinine (and associated eGFR) |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 registered outcomesOutcomes13
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Participants With Hemoglobin (Hb) Response | Baseline to 12 weeks | All enrolled participants who have completed baseline and end of treatment testing, regardless of duration of treatment with EPO, will be included in the analysis. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change in Number of Blood Transfusions Per Year | Baseline to 12 weeks | All enrolled participants who have completed baseline and end-of-treatment (12-week) testing, regardless of duration of treatment with EPO, will be included in the analysis. | Transfusions per year | MEAN |
| OTHER_PRE_SPECIFIED | Changes in Tricuspid Valve Regurgitant Jet Velocity as Assessed by Echocardiography | Baseline to 12 weeks; Baseline to 24 weeks | |||
| OTHER_PRE_SPECIFIED | Changes in Cardiac Index as Assessed by Echocardiography | Baseline to 12 weeks; Baseline to 24 weeks | |||
| OTHER_PRE_SPECIFIED | Changes in Left Ventricular End-diastolic Volume as Assessed by Echocardiography | Baseline to 12 weeks; Baseline to 24 weeks | |||
| OTHER_PRE_SPECIFIED | Changes in Exercise Capacity as Assessed by 6-minute Walk Test With Modified Borg Dyspnea Scale | Baseline to 12 weeks; Baseline to 24 weeks | |||
| OTHER_PRE_SPECIFIED | Changes in Hemoglobin (Hb) | Baseline to 12 weeks | All enrolled participants who have completed baseline and end-of-treatment (12-week) testing, regardless of duration of treatment with EPO, will be included in the analysis. | g/dL | MEAN |
| OTHER_PRE_SPECIFIED | Changes in Absolute Reticulocyte Count | Baseline to 12 weeks; Baseline to 24 weeks | |||
| OTHER_PRE_SPECIFIED | Changes in Lactate Dehydrogenase | Baseline to 12 weeks; Baseline to 24 weeks | |||
| OTHER_PRE_SPECIFIED | Changes in Renal Function | Baseline to 12 weeks; Baseline to 24 weeks |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 outcomesResults by group3
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Participants With Hemoglobin (Hb) Response | Erythropoietin | 15 | Participants | ||
| Change in Number of Blood Transfusions Per Year | Erythropoietin | -1.1 | Standard Deviation 3.7 | Transfusions per year | |
| Changes in Hemoglobin (Hb) | Erythropoietin | 3.0 | Standard Deviation 1.6 | g/dL |
Source ClinicalTrials.gov
Participant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Treatment Period | STARTED | Erythropoietin | 17 | |
| Treatment Period | COMPLETED | Erythropoietin | 16 | |
| Treatment Period | NOT COMPLETED | Erythropoietin | 1 | |
| Observation Period | STARTED | Erythropoietin | 16 | |
| Observation Period | COMPLETED | Erythropoietin | 16 | |
| Observation Period | NOT COMPLETED | Erythropoietin | 0 |
Source ClinicalTrials.gov
Adverse events23
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 | Anorexia | Gastrointestinal disorders | Erythropoietin | 1 | 17 | 1 |
| other | Cardiac troponin I increased | Cardiac disorders | Erythropoietin | 1 | 17 | 1 |
| other | Cholelithiasis | Hepatobiliary disorders | Erythropoietin | 1 | 17 | 1 |
| other | Conjunctivitis infective | Eye disorders | Erythropoietin | 1 | 17 | 1 |
| other | Diarrhea | Gastrointestinal disorders | Erythropoietin | 1 | 17 | 1 |
| other | Dizziness | Nervous system disorders | Erythropoietin | 2 | 17 | 2 |
| other | Dysgeusia | Nervous system disorders | Erythropoietin | 1 | 17 | 1 |
| other | Edema limbs | Blood and lymphatic system disorders | Erythropoietin | 1 | 17 | 1 |
| other | Headache | Nervous system disorders | Erythropoietin | 1 | 17 | 1 |
| other | Lung infection | Infections and infestations | Erythropoietin | 1 | 17 | 1 |
1 to 10 of 23Source ClinicalTrials.gov
See all 23 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Erythropoietin | 0 | 17 |
| Total, other adverse events | Erythropoietin | 15 | 17 |
| Total, serious adverse events | Erythropoietin | 2 | 17 |
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/NCT05451940
GET https://conductscience.org/api/trials/v1/NCT05451940/events?page=2