Pediatric Hydroxyurea Phase III Clinical Trial (BABY HUG) Follow-up Observational Study II Protocol

Pediatric Hydroxyurea Phase III Clinical Trial (BABY HUG) Follow-up Observational Study II Protocol

Sponsors

Lead Sponsor: National Heart, Lung, and Blood Institute (NHLBI)

Collaborator: National Institutes of Health (NIH)

Source National Heart, Lung, and Blood Institute (NHLBI)
Brief Summary

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.

Detailed Description

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.

Overall Status Completed
Start Date October 2012
Completion Date December 31, 2016
Primary Completion Date December 31, 2016
Study Type Observational
Primary Outcome
Measure Time Frame
Change in Qualitative Spleen Function From Randomized Control Trial Baseline Measurement - Compared Between Children Randomized to Hydroxyurea vs Placebo 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 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 Baseline and End of follow-up II study (up to 13 years from randomization date)
Change in the Percentage of Pitted Cell From Randomized Control Trial Baseline Measurement - Compared Between Children on Hydroxyurea vs Off Hydroxyurea at Study 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 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 Baseline and End of follow-up II study (up to 13 years from randomization date)
Enrollment 150
Condition
Intervention

Intervention Type: Drug

Intervention Name: Hydroxyurea

Description: Parents and child's doctor may plan to use or not to use hydroxyurea.

Eligibility

Sampling Method: Non-Probability Sample

Criteria:

Inclusion Criteria:

- 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

Exclusion Criteria:

- Subjects that have received a Stem Cell Transplant are not eligible for enrollment

Gender: All

Minimum Age: 24 Months

Maximum Age: 18 Years

Healthy Volunteers: No

Overall Official
Last Name Role Affiliation
Susan Assmann, PhD Principal Investigator New England Research Institutes, Watertown, MA
Location
Facility:
University of Alabama at Birmingham | Birmingham, Alabama, 35233, United States
Children's National Medical Center Center for Cancer and Blood Disorders | Washington, District of Columbia, 20010, United States
Howard University College of Medicine | Washington, 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
Location Countries

United States

Verification Date

September 2016

Responsible Party

Type: Sponsor

Has Expanded Access No
Condition Browse
Arm Group

Label: Active

Description: 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.

Label: Passive

Description: 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.

Acronym BABY HUG
Study Design Info

Observational Model: Cohort

Time Perspective: Prospective

Source: ClinicalTrials.gov