Trial · NCT00006400
Hydroxyurea to Prevent Organ Damage in Children With Sickle Cell Anemia
NCT00006400, Phase 3, Completed, National Heart, Lung, and Blood Institute (NHLBI); results first posted 2020-08-19
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Hydroxyurea to Prevent Organ Damage in Children With Sickle Cell Anemia. Trials, record NCT00006400. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00006400
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Hydroxyurea to Prevent Organ Damage in Children With Sickle Cell Anemia
- NCT number
- NCT00006400
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 193 participants, actual
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
- Conditions
- Hematologic Diseases, Anemia, Sickle Cell
- Interventions
- Hydroxyurea, Placebo
- First posted
- 2000-10-13
- Last updated
- 2020-08-19
- Results first posted
- 2020-08-19
Source ClinicalTrials.gov
Registered outcomes4
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Treatment Differences of the Change in Qualitative Splenic Function From Baseline | Before initiation of treatment and at 2 years | Primary Endpoint: Spleen function was assessed by uptake of 99mTc sulfur colloid on liver-spleen scan before initiation of treatment (baseline) and 2 years later (exit). The results of each of the two scans were categorized as normal, functional but abnormal, or not functional by a panel of nuclear medicine specialists blinded to treatment assignment. The proportion of patients whose paired scans demonstrated a decline in splenic function was compared in the hydroxyurea versus placebo groups. The change in splenic function from baseline to 2 years was defined as worse if it changed from normal to decreased or absent, or decreased to absent; and not worse if it changed from decreased to decreased, normal to normal, or decreased to normal. |
| Secondary | Change From Baseline in the Renal Function That Was Measured by Diethylenetriaminepentaacetic Acid (DTPA) Glomerular Filtration Rate (GFR) | Before initiation of treatment and at 2 years | DTPA GFR was originally a co-primary efficacy outcome for the study. Later in May 29, 2009, this measurement was discontinued because of statistical futility (an extremely small chance that the difference between treatment groups would be statistically significant for this outcome) and the small risk posed by the radiation exposure involved with performing the DTPA GFR test. Subjects who had missing data at baseline or 2 years measurement were excluded from the analysis (29 subjects from the hydroxurea, and 31 subjects from the placebo group excluded). |
| Secondary | Change From Baseline in the Renal Function That Was Measured by Glomerular Filtration Rate (GFR) (Calculated Using Schwartz Formula) | Before initiation of treatment and at 2 years | Schwartz formula used to calculate GFR is: 0.55× height (cm)/serum creatinine (mg/dL). Where height is in cm and serum creatinine is in mg/dL. Children with missing baseline or 2 years GFR were excluded from the analysis. |
| Secondary | Change From Baseline in the Renal Function That Was Measured by GFR (Calculated Using New Schwartz Formula) | Before initiation of treatment and at 2 years | GFR was calculated using new Schwartz formula: 39.1× \[height (cm)/serum creatinine (mg/dL)\]0.516 × \[1.8/cystatin C\]0.294 × \[30/blood urea nitrogen\]0.169 × \[1.099\]if male × \[height(m)/1.4\]0.188. Children with missing baseline or 2 years GFR were excluded from the analysis. |
Source ClinicalTrials.gov
Outcomes4
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Treatment Differences of the Change in Qualitative Splenic Function From Baseline | Before initiation of treatment and at 2 years | Subjects who had baseline and 2 years splenic function measurements, and had baseline measurement different from absent splenic function. A total of 26 hydroxyurea and 23 placebo subjects had missing data or had absent splenic function at baseline. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline in the Renal Function That Was Measured by Diethylenetriaminepentaacetic Acid (DTPA) Glomerular Filtration Rate (GFR) | Before initiation of treatment and at 2 years | Subjects who had baseline and 2 years measurements | mL/min/1.73m^2 | MEAN |
| SECONDARY | Change From Baseline in the Renal Function That Was Measured by Glomerular Filtration Rate (GFR) (Calculated Using Schwartz Formula) | Before initiation of treatment and at 2 years | All subjects who had baseline and 2 years GFR calculated using Schwartz formula (0.55× height (cm)/serum creatinine (mg/dL)). | mL/min/1.73m^2 | MEAN |
| SECONDARY | Change From Baseline in the Renal Function That Was Measured by GFR (Calculated Using New Schwartz Formula) | Before initiation of treatment and at 2 years | All subjects who had baseline and 2 years GFR calculated using the new Schwartz formula (39.1× \[height (cm)/serum creatinine (mg/dL)\]0.516 × \[1.8/cystatin C\]0.294 × \[30/blood urea nitrogen\]0.169 × \[1.099\]if male × \[height(m)/1.4\]0.188). | mL/min/1.73m^2 | MEAN |
Source ClinicalTrials.gov
Results by group10
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Treatment Differences of the Change in Qualitative Splenic Function From Baseline | Hydroxyurea | Worse | 19 | Participants | |
| Treatment Differences of the Change in Qualitative Splenic Function From Baseline | Placebo | Worse | 28 | Participants | |
| Treatment Differences of the Change in Qualitative Splenic Function From Baseline | Hydroxyurea | Not worse | 51 | Participants | |
| Treatment Differences of the Change in Qualitative Splenic Function From Baseline | Placebo | Not worse | 46 | Participants | |
| Change From Baseline in the Renal Function That Was Measured by Diethylenetriaminepentaacetic Acid (DTPA) Glomerular Filtration Rate (GFR) | Hydroxyurea | 22.56 | Standard Deviation 54.67 | mL/min/1.73m^2 | |
| Change From Baseline in the Renal Function That Was Measured by Diethylenetriaminepentaacetic Acid (DTPA) Glomerular Filtration Rate (GFR) | Placebo | 20.74 | Standard Deviation 51.07 | mL/min/1.73m^2 | |
| Change From Baseline in the Renal Function That Was Measured by Glomerular Filtration Rate (GFR) (Calculated Using Schwartz Formula) | Hydroxyurea | 28.65 | Standard Deviation 76.46 | mL/min/1.73m^2 | |
| Change From Baseline in the Renal Function That Was Measured by Glomerular Filtration Rate (GFR) (Calculated Using Schwartz Formula) | Placebo | 33.36 | Standard Deviation 95.85 | mL/min/1.73m^2 | |
| Change From Baseline in the Renal Function That Was Measured by GFR (Calculated Using New Schwartz Formula) | Hydroxyurea | 10.57 | Standard Deviation 20.78 | mL/min/1.73m^2 | |
| Change From Baseline in the Renal Function That Was Measured by GFR (Calculated Using New Schwartz Formula) | Placebo | 14.33 | Standard Deviation 24.01 | mL/min/1.73m^2 |
Source ClinicalTrials.gov
Participant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Hydroxyurea | 96 | |
| Overall Study | STARTED | Placebo | 97 | |
| Overall Study | COMPLETED | Hydroxyurea | 83 | |
| Overall Study | COMPLETED | Placebo | 84 | |
| Overall Study | NOT COMPLETED | Hydroxyurea | 13 | |
| Overall Study | NOT COMPLETED | Placebo | 13 |
Source ClinicalTrials.gov
Adverse events116
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 | FEVER >101.5 F (38.4 C) | General disorders | Hydroxyurea | 75 | 96 | 231 |
| other | FEVER >101.5 F (38.4 C) | General disorders | Placebo | 82 | 97 | 272 |
| other | UPPER RESPIRATORY INFECTION | Infections and infestations | Hydroxyurea | 83 | 96 | 298 |
| other | UPPER RESPIRATORY INFECTION | Infections and infestations | Placebo | 77 | 97 | 326 |
| other | UPPER RESPIRATORY INFECTION | Respiratory, thoracic and mediastinal disorders | Hydroxyurea | 8 | 96 | 12 |
| other | UPPER RESPIRATORY INFECTION | Respiratory, thoracic and mediastinal disorders | Placebo | 14 | 97 | 16 |
| other | Pain | General disorders | Hydroxyurea | 50 | 96 | 131 |
| other | Pain | General disorders | Placebo | 62 | 97 | 268 |
| other | Pain | Musculoskeletal and connective tissue disorders | Hydroxyurea | 15 | 96 | 20 |
| other | Pain | Musculoskeletal and connective tissue disorders | Placebo | 27 | 97 | 49 |
1 to 10 of 116Source ClinicalTrials.gov
See all 116 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Hydroxyurea | 0 | 96 |
| Total, all-cause mortality | Placebo | 0 | 97 |
| Total, other adverse events | Hydroxyurea | 95 | 96 |
| Total, other adverse events | Placebo | 95 | 97 |
| Total, serious adverse events | Hydroxyurea | 19 | 96 |
| Total, serious adverse events | Placebo | 35 | 97 |
Source ClinicalTrials.gov
Papers16
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 comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Hydroxyurea vs Placebo: deaths from any cause | Trial comparisons |
| Hydroxyurea vs Placebo: serious adverse events | Trial comparisons |
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/NCT00006400
GET https://conductscience.org/api/trials/v1/NCT00006400/events?page=2