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

NCT01783990 | Completed Results DMC

• 1 other identifier: HHSN268201200023C
• Study Type: observational
• Target: 150
• Locations: 1 country
• Sites: 14

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.

Conditions

Sickle Cell Anemia

Outcome Measures

Primary Outcomes (6)

• 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)

• 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)

Study Arms (2)

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.

Drug: Hydroxyurea

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.

Drug: Hydroxyurea

Interventions

Hydroxyurea DRUG

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

Active; Passive

Eligibility Criteria

• Age: 24 Months - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

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.

You may qualify if:

• 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

You may not qualify if:

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

Sponsors & Collaborators

• National Heart, Lung, and Blood Institute (NHLBI): lead
• National Institutes of Health (NIH): collaborator

Study Sites (14)

University of Alabama at Birmingham

Birmingham, Alabama, 35233, United States

Location

Children's National Medical Center Center for Cancer and Blood Disorders

Washington D.C., District of Columbia, 20010, United States

Location

Howard University College of Medicine

Washington D.C., District of Columbia, 20060, United States

Location

University of Miami School of Medicine

Miami, Florida, 33136, United States

Location

Emory University School of Medicine

Atlanta, Georgia, 30342, United States

Location

Johns Hopkins University School of Medicine

Baltimore, Maryland, 21205, United States

Location

Sinai Hospital of Baltimore Alfred I Coplan Pediatric Hematology Oncology Outpatient Center

Baltimore, Maryland, 21215, United States

Location

Children's Hospital of Michigan/Wayne State University

Detroit, Michigan, 48201, United States

Location

University of Mississippi Medical Center

Jackson, Mississippi, 39216, United States

Location

Downstate Medical Center

Brooklyn, New York, 11203, United States

Location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

Location

University of Texas Southwestern Medical Center at Dallas

Dallas, Texas, 75390, United States

Location

Biospecimen

Retention: SAMPLES WITH DNA

Stored blood and urine

MeSH Terms

Conditions

Anemia, Sickle Cell

Interventions

Hydroxyurea

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Urea; Amides; Organic Chemicals

Limitations and Caveats

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.

Results Point of Contact

• Title: Julie Miller
• Organization: New England Research Institutes, Inc

JMiller@healthcore.com

617-972-3197

Study Officials

• Susan Assmann, PhD

  New England Research Institutes, Watertown, MA

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 1, 2013
• First Posted: February 5, 2013
• Study Start: October 1, 2012
• Primary Completion: December 31, 2016
• Study Completion: December 31, 2016
• Last Updated: August 20, 2020
• Results First Posted: August 20, 2020

Record last verified: 2016-09

Locations

US (14)