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| Name | Class |
|---|---|
| National Institutes of Health (NIH) | NIH |
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The BABY HUG Treatment Study was designed to see if treatment with the drug hydroxyurea (also called HU) in children with sickle cell disease could prevent organ damage, especially in the spleen and kidneys. There was also a chance that treatment could prevent painful crises, lung disease, stroke, and blood infection.
The current observational trial, Follow-Up Study ((FUS) II includes enhanced neuropsychological, brain, cardiac, and pulmonary evaluations for this very well characterized cohort of subjects. Measures of spleen and renal function and markers of DNA damage will continue to be collected. Assessment of other target organs in sickle cell disease including pulmonary and cardiac function will be performed in addition to evaluation of developmental aspects of sickle cell disease (SCD) and potential HU toxicity.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Active | Complete blood counts (CBCs), reticulocytes, differential, lactate dehydrogenase (LDH), bilirubin and alanine transaminases (ALTs), cystatin C, blood urea nitrogen (BUN), Creatinine, fetal hemoglobin (HbF), pit counts, Howell Jolly Body (HJB), and urine microalbumin:creatinine ratio were collected at study entry, annually, and exit to Follow-Up Study II. Variable-diversity-joining (VDJ) and a stored blood sample were collected at study entry and study exit. Additional tests that include liver/spleen scan, abdominal sonogram, pulmonary function testing, magnetic resonance imaging (MRI) / magnetic resonance angiography (MRA), cardiac echocardiogram, or neuropsychology testing were collected once during the study when the child was 10 years old. |
| |
| Passive | Complete blood counts (CBCs), reticulocytes, differential, lactate dehydrogenase (LDH), bilirubin and alanine transaminases (ALTs), cystatin C, blood urea nitrogen (BUN), Creatinine, fetal hemoglobin (HbF), pit counts, Howell Jolly Body (HJB), variable-diversity-joining (VDJ), urine microalbumin:creatinine ratio and a stored blood sample were collected at study entry and exit to Follow-Up Study II. Additional tests that include liver/spleen scan, abdominal sonogram, pulmonary function testing, MRI/MRA, cardiac echocardiogram, or neuropsychology testing were collected as part of clinical care. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Hydroxyurea | Drug | Parents and child's doctor may plan to use or not to use hydroxyurea. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change in Qualitative Spleen Function From Randomized Control Trial Baseline Measurement - Compared Between Children Randomized to Hydroxyurea vs Placebo | The change in splenic function from the randomized control trial baseline measurement was one of the primary outcomes. The change in splenic function (worse vs not-worse) was compared between the randomized treatment groups (hydroxyurea vs placebo). The change in splenic function from baseline (before treatment initiation) to age 10 years (a visit when child turned 10 years old) 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, decreased to normal, absent to absent, or absent to decreased. | baseline and when child turned 10 years old |
| Change in Qualitative Spleen Function From Randomized Control Trial Baseline Measurement - Compared Between Children on Hydroxyurea vs Off Hydroxyurea at Study Visit | The change in splenic function from the randomized control trial baseline measurement was one of the primary outcomes. The change in splenic function (worse vs not-worse) was compared between children who were known to be on hydroxyurea vs. off hydroxyurea at the time of the visit. The change in splenic function from baseline (before treatment initiation) to age 10 years (a visit when child turned 10 years old) 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, decreased to normal, absent to absent, or absent to decreased. | baseline and when child turned 10 years old |
| Change in the Percentage of Pitted Cell From Randomized Control Trial Baseline Measurement - Compared Between Children Randomized to Hydroxyurea vs Placebo | The change in the percentage of pitted cell from randomized control trial baseline measurement was one of the primary outcomes. The change in the percentage of pitted cell was compared between the randomized treatment groups (hydroxyurea vs placebo). | Baseline and End of follow-up II study (up to 13 years from randomization date) |
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Inclusion Criteria:
Exclusion Criteria:
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All subjects enrolled in the BABY HUG Follow-Up I Study who participated for at least 24 months are eligible for the Follow-Up Study II.
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| Name | Affiliation | Role |
|---|---|---|
| Susan Assmann, PhD | New England Research Institutes, Watertown, MA | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Alabama at Birmingham | Birmingham | Alabama | 35233 | United States | ||
| Children's National Medical Center Center for Cancer and Blood Disorders |
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| ID | Title | Description |
|---|---|---|
| FG000 | Active | Complete blood counts (CBCs), reticulocytes, differential, lactate dehydrogenase (LDH), bilirubin and alanine transaminases (ALTs), cystatin C, blood urea nitrogen (BUN), Creatinine, fetal hemoglobin (HbF), pit counts, Howell Jolly Body (HJB), and urine microalbumin:creatinine ratio were collected at study entry, annually, and exit to Follow-Up Study II. Variable-diversity-joining (VDJ) and a stored blood sample were collected at study entry and study exit. Additional tests that include liver/spleen scan, abdominal sonogram, pulmonary function testing, magnetic resonance imaging (MRI) / magnetic resonance angiography (MRA), cardiac echocardiogram, or neuropsychology testing were collected once during the study when the child was 10 years old. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| ICF | No | No | Yes | Informed Consent Form | Oct 16, 2006 | Jan 23, 2020 |
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Stored blood and urine
| Change in the Percentage of Pitted Cell From Randomized Control Trial Baseline Measurement - Compared Between Children on Hydroxyurea vs Off Hydroxyurea at Study Visit | The change in the percentage of pitted cell from the randomized control trial baseline measurement was one of the primary outcomes. The change in the percentage of pitted cell was compared between children who were known to be on hydroxyurea vs. off hydroxyurea at the time of the visit. | Baseline and End of follow-up II study (up to 13 years from randomization date) |
| Change in Howell Jolly Body (HJB) From Randomized Control Trial Baseline Measurement - Compared Between Children Randomized to Hydroxyurea vs Placebo | The change in Howell Jolly Body from the randomized control trial baseline measurement was one of the primary outcomes. The change in Howell Jolly Body was compared between the randomized treatment groups (hydroxyurea vs placebo). | Baseline and End of follow-up II study (up to 13 years from randomization date) |
| Change in Howell Jolly Body (HJB) Count From Randomized Control Trial Baseline Measurement - Compared Between Children on Hydroxyurea vs Off Hydroxyurea | The change in Howell Jolly Body count from the randomized control trial baseline measurement was one of the primary outcomes. The change in Howell-Jolly Bodies was compared between children who were known to be on hydroxyurea vs. off hydroxyurea at the time of the visit. | Baseline and End of follow-up II study (up to 13 years from randomization date) |
| Washington D.C. |
| District of Columbia |
| 20010 |
| United States |
| Howard University College of Medicine | Washington D.C. | District of Columbia | 20060 | United States |
| University of Miami School of Medicine | Miami | Florida | 33136 | United States |
| Emory University School of Medicine | Atlanta | Georgia | 30342 | United States |
| Johns Hopkins University School of Medicine | Baltimore | Maryland | 21205 | United States |
| Sinai Hospital of Baltimore Alfred I Coplan Pediatric Hematology Oncology Outpatient Center | Baltimore | Maryland | 21215 | United States |
| Children's Hospital of Michigan/Wayne State University | Detroit | Michigan | 48201 | United States |
| University of Mississippi Medical Center | Jackson | Mississippi | 39216 | United States |
| Downstate Medical Center | Brooklyn | New York | 11203 | United States |
| Duke University Medical Center | Durham | North Carolina | 27710 | United States |
| Medical University of South Carolina | Charleston | South Carolina | 29425 | United States |
| St. Jude Children's Research Hospital | Memphis | Tennessee | 38105 | United States |
| University of Texas Southwestern Medical Center at Dallas | Dallas | Texas | 75390 | United States |
| FG001 | Passive | Complete blood counts (CBCs), reticulocytes, differential, lactate dehydrogenase (LDH), bilirubin and alanine transaminases (ALTs), cystatin C, blood urea nitrogen (BUN), Creatinine, fetal hemoglobin (HbF), pit counts, Howell Jolly Body (HJB), variable-diversity-joining (VDJ), urine microalbumin:creatinine ratio and a stored blood sample were collected at study entry and exit to Follow-Up Study II. Additional tests that include liver/spleen scan, abdominal sonogram, pulmonary function testing, MRI/MRA, cardiac echocardiogram, or neuropsychology testing were collected as part of clinical care. |
| COMPLETED |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | Active | Blood and urine specimens, and questionnaires related to the child's health status.
Patients could be on or off hydroxyurea at the start of Follow-Up Study II, and could change treatment during the study. |
| BG001 | Passive | Information from usual clinical care of sickle cell disease. We will collect information from routine tests ordered by the child's clinical sickle cell doctors. Patients could be on or off hydroxyurea at the start of Follow-Up Study II, and could change treatment during the study. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
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| Age, Continuous | Mean | Standard Deviation | Years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Region of Enrollment | Number | participants |
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| 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 | Change in Qualitative Spleen Function From Randomized Control Trial Baseline Measurement - Compared Between Children Randomized to Hydroxyurea vs Placebo | The change in splenic function from the randomized control trial baseline measurement was one of the primary outcomes. The change in splenic function (worse vs not-worse) was compared between the randomized treatment groups (hydroxyurea vs placebo). The change in splenic function from baseline (before treatment initiation) to age 10 years (a visit when child turned 10 years old) 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, decreased to normal, absent to absent, or absent to decreased. | All subjects who had age 10 years and baseline spleen function measurement. | Posted | Count of Participants | Participants | baseline and when child turned 10 years old |
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| Primary | Change in Qualitative Spleen Function From Randomized Control Trial Baseline Measurement - Compared Between Children on Hydroxyurea vs Off Hydroxyurea at Study Visit | The change in splenic function from the randomized control trial baseline measurement was one of the primary outcomes. The change in splenic function (worse vs not-worse) was compared between children who were known to be on hydroxyurea vs. off hydroxyurea at the time of the visit. The change in splenic function from baseline (before treatment initiation) to age 10 years (a visit when child turned 10 years old) 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, decreased to normal, absent to absent, or absent to decreased. | All subjects who were known to be on hydroxyurea vs. off hydroxyurea at age 10 years, and had age 10 years and baseline spleen function measurement. | Posted | Count of Participants | Participants | baseline and when child turned 10 years old |
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| Primary | Change in the Percentage of Pitted Cell From Randomized Control Trial Baseline Measurement - Compared Between Children Randomized to Hydroxyurea vs Placebo | The change in the percentage of pitted cell from randomized control trial baseline measurement was one of the primary outcomes. The change in the percentage of pitted cell was compared between the randomized treatment groups (hydroxyurea vs placebo). | All subjects who had end of study and baseline pitted cell measurement. | Posted | Median | Inter-Quartile Range | Percentage of pitted cell | Baseline and End of follow-up II study (up to 13 years from randomization date) |
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| Primary | Change in the Percentage of Pitted Cell From Randomized Control Trial Baseline Measurement - Compared Between Children on Hydroxyurea vs Off Hydroxyurea at Study Visit | The change in the percentage of pitted cell from the randomized control trial baseline measurement was one of the primary outcomes. The change in the percentage of pitted cell was compared between children who were known to be on hydroxyurea vs. off hydroxyurea at the time of the visit. | All subjects who were known to be on hydroxyurea vs. off hydroxyurea at the end of study visit, and had end of study and baseline pitted cell count measurement. | Posted | Median | Inter-Quartile Range | Percentage of pitted cell | Baseline and End of follow-up II study (up to 13 years from randomization date) |
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| Primary | Change in Howell Jolly Body (HJB) From Randomized Control Trial Baseline Measurement - Compared Between Children Randomized to Hydroxyurea vs Placebo | The change in Howell Jolly Body from the randomized control trial baseline measurement was one of the primary outcomes. The change in Howell Jolly Body was compared between the randomized treatment groups (hydroxyurea vs placebo). | All subjects who had end of follow-up study II and baseline howell jolly body count measurement. | Posted | Median | Inter-Quartile Range | Number of HJB per 10⁶ red blood cell | Baseline and End of follow-up II study (up to 13 years from randomization date) |
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| Primary | Change in Howell Jolly Body (HJB) Count From Randomized Control Trial Baseline Measurement - Compared Between Children on Hydroxyurea vs Off Hydroxyurea | The change in Howell Jolly Body count from the randomized control trial baseline measurement was one of the primary outcomes. The change in Howell-Jolly Bodies was compared between children who were known to be on hydroxyurea vs. off hydroxyurea at the time of the visit. | All subjects who were known to be on hydroxyurea vs. off hydroxyurea at the end of study visit, and had end of study and baseline Howell Jolly Body count measurement. | Posted | Median | Inter-Quartile Range | Number of HJB per 10⁶ red blood cell | Baseline and End of follow-up II study (up to 13 years from randomization date) |
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The date of consent to follow-up study II (FUS-II) through end of FUS-II, up to 4 years
Serious adverse events (SAEs) were Death, Life-threatening event, Prolonged hospitalization (>7 days), Splenic sequestration crisis, Stroke, transient ischemic attack (TIA), Acute chest syndrome, or intensive care unit (ICU) admissions which occurred during the first 5 days following performance of an active assessment study.
Major events met the definition of SAE but didn't occur within 5 days following the performance of active assessment. These are included below as if they were SAEs.
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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 | Randomized to Hydroxyurea | Subjects randomized to Hydroxyurea in the randomized phase III clinical trial. 150 subjects (130 in active and 20 in passive follow-up group) consented to follow-up II study. Of the 150 subjects, 77 subjects were randomized to the hydroxyurea treatment group during the randomized phase III clinical trial. | 0 | 77 | 24 | 77 | 0 | 77 |
| EG001 | Randomized to Placebo | Subjects randomized to Placebo in the randomized phase III clinical trial. 150 subjects (130 in active and 20 in passive follow-up group) consented to follow-up II study. Of the 150 subjects, 73 subjects were randomized to the placebo treatment group during the randomized phase III clinical trial. | 0 | 73 | 31 | 73 | 0 | 73 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Acute chest syndrome | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
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| Vaso-occlusive crisis | Blood and lymphatic system disorders | Systematic Assessment |
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| VOC. Acute hematogenous Osteomyelitis | Infections and infestations | Systematic Assessment |
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| Stroke, TIA | Nervous system disorders | Systematic Assessment |
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| Binocular diplopia and new onset left esotropia | Eye disorders | Systematic Assessment |
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| Pain Crisis | Blood and lymphatic system disorders | Systematic Assessment |
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| Pain | Musculoskeletal and connective tissue disorders | Systematic Assessment |
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| Pain | Gastrointestinal disorders | Systematic Assessment |
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| Splenic sequestration crisis | Blood and lymphatic system disorders | Systematic Assessment |
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| Pneumonia | Infections and infestations | Systematic Assessment |
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| Cholelithiasis | Hepatobiliary disorders | Systematic Assessment |
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| Bilateral infiltrates on chest x-ray. | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
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| Sickle cell anemia with crisis | Blood and lymphatic system disorders | Systematic Assessment |
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| Acute hepatic crisis with component of hepatic sequestration. | Hepatobiliary disorders | Systematic Assessment |
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| Delayed hemolytic transfusion reaction | Injury, poisoning and procedural complications | Systematic Assessment |
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| Hypoxia | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
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| Lymphangioma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | Systematic Assessment |
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| Osteomyelitis radial | Infections and infestations | Systematic Assessment |
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| Tonsilitis | Infections and infestations | Systematic Assessment |
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| Fever rhinovirus | Infections and infestations | Systematic Assessment |
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| Fever, cough, runny nose, sore throat and difficulty breathing. | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
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| Aplastic crisis | Blood and lymphatic system disorders | Systematic Assessment |
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Most children used hydroxyurea (HU) for at least part of the FUS-II observation period. This may dilute any effect of randomized group.
Most children were on HU at the specified time points, which reduces power to compare on/off HU.
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Julie Miller | New England Research Institutes, Inc | 617-972-3197 | JMiller@healthcore.com |
| ICF_000.pdf |
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Aug 9, 2009 | Jan 23, 2020 | Prot_SAP_001.pdf |
| ID | Term |
|---|---|
| D000755 | Anemia, Sickle Cell |
| ID | Term |
|---|---|
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| ID | Term |
|---|---|
| D006918 | Hydroxyurea |
| ID | Term |
|---|---|
| D014508 | Urea |
| D000577 | Amides |
| D009930 | Organic Chemicals |
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| >=65 years |
|
| Male |
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| Not Hispanic or Latino |
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| Unknown or Not Reported |
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| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
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| White |
|
| More than one race |
|
| Unknown or Not Reported |
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| Units | Counts |
|---|---|
| Participants |
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