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| ID | Type | Description | Link |
|---|---|---|---|
| N01HB07160 | U.S. NIH Grant/Contract | View source |
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Sickle cell anemia (SCA) is an inherited blood disorder that can cause organ damage. The BABY HUG study is evaluating the use of the medication hydroxyurea at preventing organ damage in children with SCA. The purpose of this follow-up study is to evaluate the long-term effects of hydroxyurea in children who have participated in the BABY HUG study.
SCA is an inherited blood disorder in which the body makes sickle-shaped red blood cells that contain abnormal hemoglobin. The sickled cells block blood flow in the vessels that lead to limbs and organs. This can cause pain, serious infections, and organ damage. The BABY HUG study (NCT00006400) is examining whether the medication hydroxyurea can prevent organ damage, especially in the spleen and kidneys, in children with SCA. This study is a follow-up study to the BABY HUG study and will enroll children who have participated in the BABY HUG study. The purpose of this study is to examine the long-term effects of using hydroxyurea as a treatment for SCA, including both the risks and benefits. Study researchers will also investigate the optimal age to begin treatment with hydroxyurea in children with SCA.
This study will enroll children between 2 and 7 years old who participated in the BABY HUG study. Hydroxyurea will not be provided to participants as part of this study, but participants may receive the medication from their own doctors. Parents of participants can choose for their child to participate in this study in one of two ways-by enrolling in either a passive follow-up group or an active follow-up group. For participants in the passive follow-up group, study researchers will review participants' medical records every 6 months, in addition to reviewing brain ultrasound tests and computed tomography (CT) or magnetic resonance imaging (MRI) scans, if completed. Participants will have a blood and urine collection at baseline and Year 4 (or at the end of the study, whichever comes first). Participants in the active follow-up group will take part in the same study procedures as participants in the passive follow-up group. In addition, at Year 2, participants in this group will undergo an additional blood and urine collection, a scanning procedure to obtain images of the liver and spleen, a kidney test, neuropsychological testing, and an ultrasound imaging test to evaluate liver and spleen size.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Active Follow-up | An active follow-up involved the performance of many of the laboratory tests and procedures done during BABY HUG clinical trials study. These included, but not limited to, serial laboratory parameters that were not part of routine clinical care such as Hgb F levels, pitted cell count, Howell-Jolly Body determination, a liver-spleen scan, diethylenetriaminepentaacetic acid (DTPA) glomerular filtration rate (GFR) measurement, creatinine clearance, Cystatin C, urine concentrating ability, transcranial Doppler, and neuropsychological testing. |
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| Passive Follow-up | A passive follow-up involved the abstraction of clinical data from the medical record. Results of physical examinations and laboratory tests performed as part of routine clinical care were recorded. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Hydroxyurea | Drug | Parents and child's doctor may plan to use or not to use hydroxyurea. |
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| 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 to 48 months 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. | 48 Months from the date of randomization |
| 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 to 48 months 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. | 48 Months from the date of randomization |
| 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). | 48 Months from the date of randomization |
| Change in the Percentage of Pitted Cell From Randomized Control Trial Baseline Measurement - Compared Between Children Randomized to Hydroxyurea vs Placebo |
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Inclusion Criteria:
Exclusion Criteria:
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Children from the initial BABY HUG study who agree to participate in this follow-up study.
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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 |
Parents and the participants' health care providers, who had not yet been informed of the BABY HUG randomized treatment, selected open label hydroxyurea (HU) treatment or standard care (SC).
Subjects that were eligible for enrollment included all children who have completed at least 18 months of the BABY HUG Treatment Study.
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| ID | Title | Description |
|---|---|---|
| FG000 | Active | An active follow-up involved the performance of many of the laboratory tests and procedures done during BABY HUG clinical trials study. These included, but not limited to, serial laboratory parameters that were not part of routine clinical care such as Hgb F levels, pitted cell count, Howell-Jolly Body determination, a liver-spleen scan, diethylenetriaminepentaacetic acid (DTPA) glomerular filtration rate (GFR) measurement, creatinine clearance, Cystatin C, urine concentrating ability, transcranial Doppler, and neuropsychological testing. |
| 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 |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan: BABY HUG Randomized Control Trial | Aug 9, 2009 |
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Stored blood and urine
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). |
| 72 Months from the date of randomization |
| 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. | 48 Months from the date of randomization |
| 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. | 72 Months from the date of randomization |
| Change in Howell-Jolly Bodies (HJB) From Randomized Control Trial Baseline Measurement - Compared Between Children Randomized to Hydroxyurea vs Placebo | The change in Howell-Jolly Bodies from randomized control trial baseline measurement was one of the primary outcomes. The change in Howell-Jolly Bodies was compared between the randomized treatment groups (hydroxyurea vs placebo). | 48 Months from the date of randomization |
| Change in Howell-Jolly Bodies (HJB) From Randomized Control Trial Baseline Measurement - Compared Between Children Randomized to Hydroxyurea vs Placebo | The change in Howell-Jolly Bodies from randomized control trial baseline measurement was one of the primary outcomes. The change in Howell-Jolly Bodies was compared between the randomized treatment groups (hydroxyurea vs placebo). | 72 Months from the date of randomization |
| Change in Howell-Jolly Bodies (HJB) From Randomized Control Trial Baseline Measurement - Compared Between Children on Hydroxyurea vs Off Hydroxyurea at Study Visit | The change in Howell-Jolly Bodies 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. | 48 Months from the date of randomization |
| Change in Howell-Jolly Bodies From Randomized Control Trial Baseline Measurement - Compared Between Children on Hydroxyurea vs Off Hydroxyurea at Study Visit | The change in Howell-Jolly Bodies (HJB) 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. | 72 Months from the date of randomization |
| 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 |
| 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 |
| Drexel University | Philadelphia | Pennsylvania | 19134 | 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 | A passive follow-up involved the abstraction of clinical data from the medical record. Results of physical examinations and laboratory tests performed as part of routine clinical care were recorded. |
| COMPLETED |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | Active | An active follow-up involved the performance of many of the laboratory tests and procedures done during BABY HUG clinical trials study. These included, but not limited to, serial laboratory parameters that were not part of routine clinical care such as Hgb F levels, pitted cell count, Howell-Jolly Body determination, a liver-spleen scan, DTPA GFR measurement, creatinine clearance, Cystatin C, urine concentrating ability, transcranial Doppler, and neuropsychological testing. Patients could be on or off hydroxyurea at the start of Follow-Up Study I, and could change treatment during the study. |
| BG001 | Passive | A passive follow-up involved the abstraction of clinical data from the medical record. Results of physical examinations and laboratory tests performed as part of routine clinical care were recorded. Patients could be on or off hydroxyurea at the start of Follow-Up Study I, 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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| 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 to 48 months 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 48 month and baseline spleen function measurement. | Posted | Count of Participants | Participants | 48 Months from the date of randomization |
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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 to 48 months 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 the 48 month visit, and had 48 month and baseline spleen function measurement. | Posted | Count of Participants | Participants | 48 Months from the date of randomization |
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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 48 month and baseline pitted cell measurements. | Posted | Median | Inter-Quartile Range | Percentage of pitted cell | 48 Months from the date of randomization |
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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 72 month and baseline pitted cell measurements. | Posted | Median | Inter-Quartile Range | Percentage of pitted cell | 72 Months from the date of randomization |
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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 48 month visit, and had 48 month and baseline pitted cell measurements. | Posted | Median | Inter-Quartile Range | Percentage of pitted cell | 48 Months from the date of randomization |
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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 72 month visit, and had 72 month and baseline pitted cell measurements. | Posted | Median | Inter-Quartile Range | Percentage of pitted cell | 72 Months from the date of randomization |
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| Primary | Change in Howell-Jolly Bodies (HJB) From Randomized Control Trial Baseline Measurement - Compared Between Children Randomized to Hydroxyurea vs Placebo | The change in Howell-Jolly Bodies from randomized control trial baseline measurement was one of the primary outcomes. The change in Howell-Jolly Bodies was compared between the randomized treatment groups (hydroxyurea vs placebo). | All subjects who had 48 month and baseline Howell-Jolly Bodies measurements. | Posted | Median | Inter-Quartile Range | Number of HJB per 10⁶ red blood cell | 48 Months from the date of randomization |
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| Primary | Change in Howell-Jolly Bodies (HJB) From Randomized Control Trial Baseline Measurement - Compared Between Children Randomized to Hydroxyurea vs Placebo | The change in Howell-Jolly Bodies from randomized control trial baseline measurement was one of the primary outcomes. The change in Howell-Jolly Bodies was compared between the randomized treatment groups (hydroxyurea vs placebo). | All subjects who had 72 month and baseline Howell-Jolly Bodies measurements. | Posted | Median | Inter-Quartile Range | Number of HJB per 10⁶ red blood cell | 72 Months from the date of randomization |
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| Primary | Change in Howell-Jolly Bodies (HJB) From Randomized Control Trial Baseline Measurement - Compared Between Children on Hydroxyurea vs Off Hydroxyurea at Study Visit | The change in Howell-Jolly Bodies 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 48 month visit, and had 48 month and baseline Howell-Jolly Bodies measurements. | Posted | Median | Inter-Quartile Range | Number of HJB per 10⁶ red blood cell | 48 Months from the date of randomization |
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| Primary | Change in Howell-Jolly Bodies From Randomized Control Trial Baseline Measurement - Compared Between Children on Hydroxyurea vs Off Hydroxyurea at Study Visit | The change in Howell-Jolly Bodies (HJB) 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 72 month visit, and had 72 month and baseline Howell-Jolly Bodies measurements. | Posted | Median | Inter-Quartile Range | Number of HJB per 10⁶ red blood cell | 72 Months from the date of randomization |
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The date of consent to follow-up study I (FUS-I) through end of FUS-I, up to 2 years
Serious adverse event (SAE) were Death, Life-threatening event, Prolonged hospitalization (greater than 7 days), Splenic sequestration crisis, Stroke / transient ischemic attack (TIA), Acute chest syndrome, or ICU admissions, and occurred during the first 5 days following performance of an active assessment study.
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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. | 0 | 84 | 1 | 84 | 0 | 84 |
| EG001 | Randomized to Placebo | Subjects randomized to Placebo in the randomized phase III clinical trial. | 0 | 79 | 1 | 79 | 0 | 79 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Acute chest syndrome | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
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Most children used hydroxyurea (HU) for at least part of the FUS-I 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 | |
|---|---|---|---|---|
| Susan Assmann, PhD | New England Research Institutes, Inc. | 617-972-3048 | sassmann@neriscience.com |
| Jan 23, 2020 |
| Prot_SAP_000.pdf |
| ICF | No | No | Yes | Informed Consent Form: BABY HUG Randomized Control Trial | Oct 16, 2006 | Jan 23, 2020 | ICF_001.pdf |
| ICF | No | No | Yes | Informed Consent Form: BABY HUG Follow-up Study I | Nov 10, 2006 | Feb 26, 2020 | ICF_002.pdf |
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan: BABY HUG Follow-up Study I | Jan 4, 2010 | Feb 26, 2020 | Prot_SAP_003.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 |
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| Male |
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