We are updating the design of this site. Learn more.
Show more
ClinicalTrials.gov
ClinicalTrials.gov Menu

Pediatric Hydroxyurea in Sickle Cell Anemia (PED HUG)

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00000602
First Posted: October 28, 1999
Last Update Posted: September 16, 2016
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by:
National Heart, Lung, and Blood Institute (NHLBI)
  Purpose
To determine whether hydroxyurea prevents the onset of chronic end organ damage in young children with sickle cell anemia.

Condition Intervention Phase
Anemia, Sickle Cell Hematologic Diseases Hemoglobinopathies Drug: hydroxyurea Phase 2

Study Type: Interventional
Study Design: Primary Purpose: Treatment

Resource links provided by NLM:


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

Study Start Date: April 1994
Study Completion Date: March 1997
Detailed Description:

BACKGROUND:

Sickle cell anemia is a complex syndrome with multiple organ system disturbances brought about by the interplay of genetic, humoral, vascular and environmental factors. The clinical course can be one of abrupt and insidious exacerbations and remissions, often migratory and repetitive. These events may result in impairment of function, permanently damaged organs, and ultimately death. Although there is wide variability in the clinical expression of sickle cell disease, this complex set of clinical manifestations is experienced by most patients. In addition, there is no evidence that the primary disease process is different in children when compared with adults with regard to painful episodes. However, children have a higher incidence of respiratory viral infections, and are susceptible to pneumococcal septicemia. With the successful completion of the Multicenter Study of Hydroxyurea (MSH) Trial in adults, attention has now been focused on the use of this agent in children.

The Cooperative Study of Sickle Cell Disease (CSSCD) has demonstrated that sickle cell anemia patients with increased painful episode rates die at a younger age. In addition, increased levels of fetal hemoglobin are associated with improved survival, and is probably a reliable childhood forecaster of adult life expectancy. The beneficial effect produced by hydroxyurea is thought to occur because it increases fetal hemoglobin levels. Therefore, if chronic end organ damage can be prevented in early childhood by hydroxyurea administration, and if the crisis rate can be decreased by hydroxyurea use early in life, sickle cell anemia patients may experience increased longevity and an improved quality of life.

DESIGN NARRATIVE:

The Phase I-Phase II study, HUG-KIDS, examined the safety of hydroxyurea. Children with sickle cell anemia, age 5 to 15 years, were eligible for this multicenter Phase I/II trial. Hydroxyurea was started at 15 mg/kg/d and escalated to 30 mg/kg/d unless the patient experienced laboratory toxicity. Patients were monitored by 2-week visits to assess compliance, toxicity, clinical adverse events, growth parameters, and laboratory efficacy associated with hydroxyurea treatment. Eighty-four children were enrolled between December 1994 and March 1996. Sixty-eight children reached maximum tolerated dose (MTD) and 52 were treated at MTD for 1 year. The study was conducted at four Comprehensive Sickle Cell Centers by the following investigators: Thomas R. Kinney at Duke University Medical Center, Durham, North Carolina; Kwaku Ohene-Frempong at Children's Hospital of Philadelphia; Orah S. Platt at Children's Hospital in Boston; and Elliot Vichinsky at Children's Hospital in Oakland, California. The complete study lasted three years.

The study completion date listed in this record was obtained from the "End Date" entered in the Protocol Registration and Results System (PRS) record.

  Eligibility

Information from the National Library of Medicine

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, Learn About Clinical Studies.


Ages Eligible for Study:   1 Year to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria
Children with sickle cell disease and between the ages of five and eighteen years.
  Contacts and Locations
No Contacts or Locations Provided
  More Information

Publications:
ClinicalTrials.gov Identifier: NCT00000602     History of Changes
Other Study ID Numbers: 315
P60HL015157 ( U.S. NIH Grant/Contract )
P60HL028391 ( U.S. NIH Grant/Contract )
P60HL020985 ( U.S. NIH Grant/Contract )
First Submitted: October 27, 1999
First Posted: October 28, 1999
Last Update Posted: September 16, 2016
Last Verified: October 2005

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