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Hydroxyurea to Prevent Organ Damage in Children With Sickle Cell Anemia

Information source: National Heart, Lung, and Blood Institute (NHLBI)
ClinicalTrials.gov processed this data on August 23, 2015
Link to the current ClinicalTrials.gov record.

Condition(s) targeted: Hematologic Diseases; Anemia, Sickle Cell

Intervention: Hydroxyurea (Drug); Placebo (Drug)

Phase: Phase 3

Status: Completed

Sponsored by: National Heart, Lung, and Blood Institute (NHLBI)

Official(s) and/or principal investigator(s):
Sherron Jackson, MD, Principal Investigator, Affiliation: Medical University of South Carolina
James F. Casella, MD, Principal Investigator, Affiliation: Johns Hopkins University
Lori Luchtman-Jones, MD, Principal Investigator, Affiliation: Children's Research Institute
Rathi V. Iyer, MD, Principal Investigator, Affiliation: University of Mississippi Medical Center
Scott T. Miller, MD, Principal Investigator, Affiliation: SUNY Health Science Center, Brooklyn
Sohail R. Rana, MD, Principal Investigator, Affiliation: Howard University
Zora R. Rogers, MD, Principal Investigator, Affiliation: University of Texas SW Medical Center
Bruce W Thompson, Ph.D., Principal Investigator, Affiliation: Clinical Trials and Surveys Corp
Julio Barredo, MD, Principal Investigator, Affiliation: University of Miami Medical Center
Winfred C. Wang, MD, Study Chair, Affiliation: St. Jude Children's Research Hospital
Courtney Thornburg, MD, Principal Investigator, Affiliation: Duke University
Thomas Howard, MD, Principal Investigator, Affiliation: University of Alabama at Birmingham
Lori Luck, MD, Principal Investigator, Affiliation: Drexel University
R. Clark Brown, MD, PhD, Principal Investigator, Affiliation: Emory University
Sharada Sarnaik, MD, Principal Investigator, Affiliation: Wayne State University

Summary

The purpose of this study is to determine if hydroxyurea therapy is effective in the prevention of chronic end organ damage in pediatric patients with sickle cell anemia.

Clinical Details

Official title: Pediatric Hydroxyurea Phase III Clinical Trial (BABY HUG)

Study design: Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Prevention

Primary outcome: 50% reduction in rates of damage to the major organs with surrogate markers of organ function

Detailed description: BACKGROUND: In 1995, the Multicenter Study of Hydroxyurea (MSH) demonstrated that hydroxyurea is effective in decreasing the frequency of painful crises, hospitalizations for crises, acute chest syndrome, and blood transfusions by 50%. The recently completed phase II study of hydroxyurea in children (PED HUG) demonstrated that children have a response to hydroxyurea similar to that seen in adults in terms of increasing fetal hemoglobin levels and total hemoglobin, and decreasing complications associated with sickle cell anemia. In addition, this study demonstrated that the drug does not adversely affect growth and development between the ages of 5 and 15. A recently completed pilot study of hydroxyurea given to children between the ages of 6 months and 24 months demonstrated that the drug is tolerated well by small infant, and that the fetal hemoglobin switch can be forced to remain in the "on position" by hydroxyurea administration. A Special Emphasis Panel (SEP) met on April 12, 1996 to review the results of the MSH trial and the progress to date of the PED HUG study. The SEP recommended that NHLBI undertake the BABY HUG trial. DESIGN NARRATIVE: BABY HUG is a randomized, double-blind, placebo-controlled study to determine if hydroxyurea can prevent the onset of chronic end organ damage in young children with sickle cell anemia. Approximately 200 children with sickle cell disease will be recruited to receive either hydroxyurea or placebo. The children will be screened at study entry for signs of abnormal brain, kidney, pulmonary, and splenic function, and developmental milestones. They will then be randomly assigned to receive either hydroxyurea or placebo and followed yearly to assess chronic end organ damage of the major organ systems. The primary endpoint will be a 50% reduction in rates of damage to the major organs with surrogate markers of organ function during follow-up in Phase II of the trial.

Eligibility

Minimum age: 9 Months. Maximum age: 18 Months. Gender(s): Both.

Criteria:

Inclusion Criteria:

- Majority fetal and sickle (FS or SF) hemoglobin pattern confirmed centrally by

electrophoresis (screening may begin at 7 months of age) Exclusion Criteria:

- Chronic transfusion therapy

- Cancer

- Less than 5th percentile (10th percentile for the pilot study) height, weight, or

head circumference for age

- Severe developmental delay (e. g., cerebral palsy or other mental retardation, Grade

III/IV intraventricular hemorrhage)

- Stroke with neurological deficit

- Surgical splenectomy

- Participating in other clinical intervention trials

- Probable or known diagnosis of Hemoglobin S-Hereditary Persistence of Fetal

Hemoglobin

- Known hemoglobin S-beta plus thalassemia (hemoglobin A present)

- Any condition or chronic illness, which in the opinion of the principal investigator,

makes participation unadvised or unsafe

- Inability or unwillingness to complete baseline (pre-enrollment) studies, including

blood or urine specimen collection, liver-spleen scan, abdominal sonogram, neurological examination, neuropsychological testing, or transcranial Doppler ultrasound (interpretable study not required, but confirmed velocity greater than 200 cm/sec results in ineligibility)

- Previous or current treatment with hydroxyurea (HU) or another anti-sickling drug

- The following exclusion criteria are transient; patients can be re-evaluated for

eligibility: 1. Hemoglobin less than 6. 0 gm/dL 2. Reticulocyte count less than 80,000/cu mm if hemoglobin is less than 9 gm/dL 3. Neutrophil count less than 2,000/cu mm 4. Platelet count less than 130,000/cu mm 5. Blood transfusion in the 2 months prior to study entry unless HbA is less than 10% 6. ALT greater than twice the upper limit of normal 7. Ferritin less than 10 ng/ml 8. Serum creatinine greater than twice the upper limit of normal for age 9. Bayley standardized mental score below 70

Locations and Contacts

University of Alabama at Birmingham, Birmingham, Alabama 35233, United States

Children's National Medical Center, Washington, District of Columbia 20010, United States

Howard University, Washington, District of Columbia 20060, United States

University of Miami, Miami, Florida 33136, United States

Emory University School of Medicine, Atlanta, Georgia 30342, United States

Johns Hopkins University, Baltimore, Maryland 21287, United States

Children's Hospital of Michigan/Wayne State Univ., Detroit, Michigan 48201, United States

University of Mississippi Medical Center, Jackson, Mississippi 39216, United States

SUNY Health Science Center, Brooklyn, 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 SW Medical Center, Dallas, Texas 75390, United States

Additional Information

Related publications:

Heeney MM, Whorton MR, Howard TA, Johnson CA, Ware RE. Chemical and functional analysis of hydroxyurea oral solutions. J Pediatr Hematol Oncol. 2004 Mar;26(3):179-84.

Thompson BW, Miller ST, Rogers ZR, Rees RC, Ware RE, Waclawiw MA, Iyer RV, Casella JF, Luchtman-Jones L, Rana S, Thornburg CD, Kalpatthi RV, Barredo JC, Brown RC, Sarnaik S, Howard TH, Luck L, Wang WC. The pediatric hydroxyurea phase III clinical trial (BABY HUG): challenges of study design. Pediatr Blood Cancer. 2010 Feb;54(2):250-5. doi: 10.1002/pbc.22269.

Starting date: August 2000
Last updated: April 15, 2011

Page last updated: August 23, 2015

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