Long-Term Effects of Hydroxyurea in Children With Sickle Cell Anemia (The BABY HUG Follow-up Study)
Pediatric Hydroxyurea Phase III Clinical Trial (BABY HUG) Follow-up Study
2 other identifiers
observational
163
1 country
14
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for all trials
Started Sep 2008
Typical duration for all trials
14 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
September 1, 2008
CompletedFirst Submitted
Initial submission to the registry
April 27, 2009
CompletedFirst Posted
Study publicly available on registry
April 29, 2009
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 1, 2011
CompletedStudy Completion
Last participant's last visit for all outcomes
December 1, 2011
CompletedResults Posted
Study results publicly available
August 20, 2020
CompletedAugust 20, 2020
July 1, 2009
3.2 years
April 27, 2009
March 1, 2020
August 10, 2020
Conditions
Keywords
Outcome Measures
Primary Outcomes (10)
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
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
Study Arms (2)
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.
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.
Interventions
Parents and child's doctor may plan to use or not to use hydroxyurea.
Eligibility Criteria
Children from the initial BABY HUG study who agree to participate in this follow-up study.
You may qualify if:
- All children who completed at least 18 months of follow-up visits in the initial BABY HUG study
- Children from the initial BABY HUG study who are on a chronic transfusion program or who are recipients of a bone marrow transplant
You may not qualify if:
- Any child who was not enrolled in the initial BABY HUG study for at least 18 months
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (14)
University of Alabama at Birmingham
Birmingham, Alabama, 35233, United States
Children's National Medical Center
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
Biospecimen
Stored blood and urine
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Limitations and Caveats
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.
Results Point of Contact
- Title
- Susan Assmann, PhD
- Organization
- New England Research Institutes, Inc.
Study Officials
- PRINCIPAL INVESTIGATOR
Susan Assmann, PhD
New England Research Institutes, Watertown, MA
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- No
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- OTHER
- Sponsor Type
- NIH
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
April 27, 2009
First Posted
April 29, 2009
Study Start
September 1, 2008
Primary Completion
December 1, 2011
Study Completion
December 1, 2011
Last Updated
August 20, 2020
Results First Posted
August 20, 2020
Record last verified: 2009-07