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Jackson, Mississippi 39216

  • Anemia, Sickle Cell

Purpose:

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.


Study summary:

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.


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


Study is Available At:


Original ID:

89


NCT ID:

NCT00006400


Secondary ID:

N01 HB07150


Study Acronym:


Brief Title:

Hydroxyurea to Prevent Organ Damage in Children With Sickle Cell Anemia


Official Title:

Pediatric Hydroxyurea Phase III Clinical Trial (BABY HUG)


Overall Status:

Completed


Study Phase:

Phase 3


Genders:

Both


Minimum Age:

9 Months


Maximum Age:

18 Months


Quick Facts

Healthy Volunteers
Oversight Has DMC
Study Is FDA Regulated
Study Is Section 801
Has Expanded Access

Study Source:

National Heart, Lung, and Blood Institute (NHLBI)


Oversight Authority:

United States: Federal Government


Reasons Why Stopped:


Study Type:

Interventional


Study Design:

Allocation: Randomized, Endpoint Classification:


Number of Arms:

2


Number of Groups:

0


Total Enrollment:

191


Enrollment Type:

Actual


Overall Contact Information

Official Name:Sherron Jackson, MD
Principal Investigator
Medical University of South Carolina

Study Dates

Start Date:August 2000
Completion Date:September 2009
Completion Type:Actual
Primary Completion Date:September 2009
Primary Completion Type:Actual
Verification Date:April 2011
Last Changed Date:April 15, 2011
First Received Date:October 12, 2000

Study Outcomes

Outcome Type:Primary Outcome
Measure:50% reduction in rates of damage to the major organs with surrogate markers of organ function
Time Frame:Measured during follow-up evaluations
Safety Issues:True

Study Interventions

Intervention Type:Drug
Name:Hydroxyurea
Description:Participants will receive hydroxyurea.
Arm Name:1
Intervention Type:Drug
Name:Placebo
Description:Participants will receive placebo.
Arm Name:2

Study Arms

Study Arm Type:Active Comparator
Arm Name:1
Description:Participants will receive hydroxyurea.
Study Arm Type:Placebo Comparator
Arm Name:2
Description:Participants will receive placebo.

Study Agencies

Agency Class:NIH
Agency Type:Lead Sponsor
Agency Name:National Heart, Lung, and Blood Institute (NHLBI)

Sample and Retention Information

There are no available Sample and Retention Information

Study References

Reference Type:Reference
Citation: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.
PMID:15125610
Reference Type:Reference
Citation: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.
PMID:19731330
Reference Type:Results Reference
Citation:Wang WC, Pavlakis SG, Helton KJ, McKinstry RC, Casella JF, Adams RJ, Rees RC; BABY HUG Investigators. MRI abnormalities of the brain in one-year-old children with sickle cell anemia. Pediatr Blood Cancer. 2008 Nov;51(5):643-6.
PMID:18478575
Reference Type:Results Reference
Citation:Miller ST, Wang WC, Iyer R, Rana S, Lane P, Ware RE, Li D, Rees RC; BABY-HUG Investigators. Urine concentrating ability in infants with sickle cell disease: baseline data from the phase III trial of hydroxyurea (BABY HUG). Pediatr Blood Cancer. 2010 Feb;54(2):265-8.
PMID:19621454
Reference Type:Results Reference
Citation:Pavlakis SG, Rees RC, Huang X, Brown RC, Casella JF, Iyer RV, Kalpatthi R, Luden J, Miller ST, Rogers ZR, Thornburg CD, Wang WC, Adams RJ; BABY HUG Investigators. Transcranial doppler ultrasonography (TCD) in infants with sickle cell anemia: baseline data from the BABY HUG trial. Pediatr Blood Cancer. 2010 Feb;54(2):256-9.
PMID:19813252
Reference Type:Results Reference
Citation:Thornburg CD, Rogers ZR, Jeng MR, Rana SR, Iyer RV, Faughnan L, Hassen L, Marshall J, McDonald RP, Wang WC, Huang X, Rees RC; BABY HUG Investigators. Adherence to study medication and visits: data from the BABY HUG trial. Pediatr Blood Cancer. 2010 Feb;54(2):260-4.
PMID:19856395
Reference Type:Results Reference
Citation:Ware RE, Rees RC, Sarnaik SA, Iyer RV, Alvarez OA, Casella JF, Shulkin BL, Shalaby-Rana E, Strife CF, Miller JH, Lane PA, Wang WC, Miller ST; BABY HUG Investigators. Renal function in infants with sickle cell anemia: baseline data from the BABY HUG trial. J Pediatr. 2010 Jan;156(1):66-70.e1.
PMID:19880138

Data Source: ClinicalTrials.gov

Date Processed: March 30, 2020

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