Hydroxyurea to Prevent Organ Damage in Children With Sickle Cell Anemia - Tabular View

This Tabular View shows the required WHO registration data elements as marked by ^†

Tracking Information

First Received Date ^†

October 12, 2000

Last Updated Date

October 14, 2008

Start Date ^†

August 2000

Current Primary Outcome Measures ^†

50% reduction in rates of damage to the major organs with surrogate markers of organ function [ Time Frame: Measured during follow-up evaluations ] [ Designated as safety issue: Yes ]

Original Primary Outcome Measures ^†

Same as current

Change History

Complete list of historical versions of study NCT00006400 on ClinicalTrials.gov Archive Site

Current Secondary Outcome Measures ^†

Original Secondary Outcome Measures ^†

Descriptive Information

Brief Title ^†

Hydroxyurea to Prevent Organ Damage in Children With Sickle Cell Anemia

Official Title ^†

Pediatric Hydroxyurea in Sickle Cell Anemia (BABY HUG)

Brief 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.

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.

Study Phase

Phase III

Study Type ^†

Interventional

Study Design ^†

Prevention, Randomized, Double Blind (Subject, Investigator), Placebo Control, Parallel Assignment, Efficacy Study

Condition ^†

Hematologic Diseases
Anemia, Sickle Cell

Intervention ^†

Drug: Hydroxyurea
Drug: Placebo

Study Arms / Comparison Groups

Active Comparator: Participants will receive hydroxyurea.
Placebo Comparator: Participants will receive placebo.

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.

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status ^†

Active, not recruiting

Enrollment ^†

200

Estimated Completion Date

September 2009

Estimated Primary Completion Date

September 2009 (final data collection date for primary outcome measure)

Eligibility 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

Gender

Both

Ages

9 Months to 18 Months

Accepts Healthy Volunteers

Contacts ^††

Location Countries ^†

United States

Expanded Access Status

Administrative Information

NCT ID ^†

NCT00006400

Responsible Party

Sohail R. Rana, MD, Howard University

Secondary IDs ^††

N01 HB07150, N01 HB07151, N01 HB07152, N01 HB07153, N01 HB07154, N01 HB07155, N01 HB07156, N01 HB07157, N01 HB07158, N01 HB07159, N01 HB07160

Study Sponsor ^†

National Heart, Lung, and Blood Institute (NHLBI)

Collaborators ^††

Investigators ^†

Study Chair:	Julio Barredo, MD	Medical University of South Carolina
Study Chair:	James F. Casella, MD	Johns Hopkins University
Study Chair:	Caterina Minnetti, MD	Children's Research Institute
Study Chair:	Rathi V. Iyer, MD	University of Mississippi Medical Center
Study Chair:	Scott T. Miller, MD	SUNY Health Science Center, Brooklyn
Study Chair:	Sohail R. Rana, MD	Howard University
Study Chair:	Zora R. Rogers, MD	University of Texas SW Medical Center
Study Chair:	Bruce Thompson	Clinical Trials and Surveys Corporation
Study Chair:	Stuart Toledano, MD	University of Miami
Study Chair:	Winfred C. Wang, MD	St. Jude Children's Research Hospital
Study Chair:	Sherri A. Zimmerman, MD	Duke University

Information Provided By

National Heart, Lung, and Blood Institute (NHLBI)

Verification Date

October 2008

^† Required WHO trial registration data element.
^†† WHO trial registration data element that is required only if it exists.