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

This study is enrolling participants by invitation only.
Sponsor:
Collaborator:
Information provided by (Responsible Party):
National Heart, Lung, and Blood Institute (NHLBI)
ClinicalTrials.gov Identifier:
NCT01783990
First received: February 1, 2013
Last updated: February 4, 2013
Last verified: January 2013
  Purpose

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.


Condition Intervention
Sickle Cell Anemia
Drug: Hydroxyurea

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Retrospective
Official Title: Pediatric Hydroxyurea Phase III Clinical Trial (BABY HUG) Follow-up Observational Study II Protocol

Resource links provided by NLM:


Further study details as provided by National Heart, Lung, and Blood Institute (NHLBI):

Primary Outcome Measures:
  • The primary objective of Follow-Up Study II is to monitor the continued safety and potential efficacy of HU treatment. [ Time Frame: Every 6 months ] [ Designated as safety issue: Yes ]
    Safety of HU will be assessed by ongoing clinical monitoring of growth and development, age-appropriate neuropsychological evaluation, serial hematologic and chemistry parameters, and the frequency of expected and unexpected clinical events related to sickle cell disease. Disease and treatment-related effects on the spleen will be measured by a liver-spleen scan, pitted cell counts and HJB enumeration. Kidney function will be measured by GFR estimation from the Schwartz formula or cystatin C measurements and the urine osmolality and urine microalbumin:creatinine ratio. The BH FU II investigators will also seek to study how early treatment with HU changes the child's disease trajectory on open-label HU.


Biospecimen Retention:   Samples With DNA

Stored blood and urine


Estimated Enrollment: 158
Study Start Date: October 2012
Estimated Study Completion Date: December 2016
Estimated Primary Completion Date: December 2016 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
Active

Blood and urine specimens, and questionnaires related to the child's health status.

  • Liver Spleen Scan
  • Abdominal Ultrasound
  • Brain MRI/MRA
  • Cardiac Echocardiogram/Pulmonary Function Testing
  • Transcranial Doppler
  • Neuropsychology Testing (Vineland, WISC-IV, Connor CPT II and Peds QOL)
Drug: Hydroxyurea
Active and Passive groups both are on HU.
Other Name: HU
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.
Drug: Hydroxyurea
Active and Passive groups both are on HU.
Other Name: HU

Detailed Description:

The current observational trial, Follow-Up Study II (designated 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 SCD and potential HU toxicity.

  Eligibility

Ages Eligible for Study:   24 Months to 18 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   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.

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01783990

Locations
United States, Alabama
University of Alabama at Birmingham
Birmingham, Alabama, United States, 35233
United States, District of Columbia
Howard University College of Medicine
Washington, District of Columbia, United States, 20060
Children's National Medical Center Center for Cancer and Blood Disorders
Washington, District of Columbia, United States, 20010
United States, Florida
University of Miami School of Medicine
Miami, Florida, United States, 33136
United States, Georgia
Emory University School of Medicine
Atlanta, Georgia, United States, 30342
United States, Maryland
Johns Hopkins University School of Medicine
Baltimore, Maryland, United States, 21205
Sinai Hospital of Baltimore Alfred I Coplan Pediatric Hematology Oncology Outpatient Center
Baltimore, Maryland, United States, 21215
United States, Michigan
Children's Hospital of Michigan/Wayne State University
Detroit, Michigan, United States, 48201
United States, Mississippi
University of Mississippi Medical Center
Jackson, Mississippi, United States, 39216
United States, New York
Downstate Medical Center
Brooklyn, New York, United States, 11203
United States, North Carolina
Duke University Medical Center
Durham, North Carolina, United States, 27710
United States, South Carolina
Medical University of South Carolina
Charleston, South Carolina, United States, 29425
United States, Tennessee
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105
United States, Texas
University of Texas Southwestern Medical Center at Dallas
Dallas, Texas, United States, 75390
Sponsors and Collaborators
Investigators
Principal Investigator: Bruce Thompson, Ph.D. Clinical Trials and Surveys
  More Information

No publications provided

Responsible Party: National Heart, Lung, and Blood Institute (NHLBI)
ClinicalTrials.gov Identifier: NCT01783990     History of Changes
Other Study ID Numbers: HHSN268201200023C
Study First Received: February 1, 2013
Last Updated: February 4, 2013
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Anemia
Anemia, Sickle Cell
Hematologic Diseases
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hemoglobinopathies
Genetic Diseases, Inborn
Hydroxyurea
Antineoplastic Agents
Therapeutic Uses
Pharmacologic Actions
Antisickling Agents
Hematologic Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Nucleic Acid Synthesis Inhibitors

ClinicalTrials.gov processed this record on July 20, 2014