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Low subject accrual
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The goal of this study is to test the hypothesis that hydroxyurea is effective for the specific treatment of secondary pulmonary hypertension found on screening in children and young adults with sickle cell disease.
Increasing evidence suggests that pulmonary hypertension, defined by an elevated tricuspid regurgitant jet velocity (TRJV) on echocardiogram, is a major cause of morbidity and mortality in adults with sickle cell disease (SCD). However, both the prevalence and optimal treatment of pulmonary hypertension in children and young adults with SCD are unknown.
We hypothesize that short term therapy with hydroxyurea will decrease TRJV in children and young adults with pulmonary hypertension found on screening. Patients eligible for treatment will have had evidence of pulmonary hypertension on at least 2 screening echocardiograms. Baseline laboratory tests will be obtained and other causes of secondary pulmonary hypertension will be excluded prior to initiation of treatment. Patients will be treated with hydroxyurea according to a standard dose escalation schedule for a total of 12 months. A clinic visit will be required every 2 months and standard screening for toxicity will be performed monthly. There will be an interim analysis of the primary outcome at 6 months following therapy.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Hydroxyurea | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Hydroxyurea | Drug | 20 mg/kg/day and dose escalating every 2 months until maximum tolerated dose. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Tricuspid Regurgitant Jet Velocity | Primary outcome measure was tricuspid regurgitant jet velocity (TRJV) by echocardiogram after 6 and 12 months of hydroxyurea therapy. | 6 and 12 months after HU therapy begins |
| Measure | Description | Time Frame |
|---|---|---|
| Compliance | Secondary outcome measures included compliance; laboratory measures of therapy-related toxicity; laboratory biomarkers for hemolysis, oxidative stress and endothelial injury; and quality of life measures by Child Health Questionnaire (CHQ). | Throughout study |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Robert I Liem, MD | Ann & Robert H Lurie Children's Hospital of Chicago | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Ann & Robert H Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | United States |
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This study is currently closed to patient accrual at all sites due to poor enrollment, including Children's Memorial Hospital, Washington University, Johns Hopkins and the Medical College of Wisconsin. The FDA was notified in 2010, and the IND was removed for this study.
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| ID | Title | Description |
|---|---|---|
| FG000 | Hydroxyurea | Hydroxyurea : 20 mg/kg/day and dose escalating every 2 months until maximum tolerated dose. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Hydroxyurea | Hydroxyurea : 20 mg/kg/day and dose escalating every 2 months until maximum tolerated dose. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Tricuspid Regurgitant Jet Velocity | Primary outcome measure was tricuspid regurgitant jet velocity (TRJV) by echocardiogram after 6 and 12 months of hydroxyurea therapy. | Study was terminated and 0 participants were analyzed. | Posted | 6 and 12 months after HU therapy begins |
|
|
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Hydroxyurea | Hydroxyurea : 20 mg/kg/day and dose escalating every 2 months until maximum tolerated dose. |
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Early termination leading to no data analysis completed.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Robert I. Liem | Ann and Robert H. Lurie Children's Hospital of Chicago | 312-227-4842 | Rliem@luriechildrens.org |
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| ID | Term |
|---|---|
| D000755 | Anemia, Sickle Cell |
| D006976 | Hypertension, Pulmonary |
| ID | Term |
|---|---|
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
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| ID | Term |
|---|---|
| D006918 | Hydroxyurea |
| ID | Term |
|---|---|
| D014508 | Urea |
| D000577 | Amides |
| D009930 | Organic Chemicals |
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| Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| Secondary | Compliance | Secondary outcome measures included compliance; laboratory measures of therapy-related toxicity; laboratory biomarkers for hemolysis, oxidative stress and endothelial injury; and quality of life measures by Child Health Questionnaire (CHQ). | Not Posted | Throughout study | Participants |
| 0 |
| 6 |
| 0 |
| 6 |
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| D006425 |
| Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D006973 | Hypertension |
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |