Executive Summary

NCI PHASE I PEDIATRIC TRIAL WITH GENENTECH's CD4 will begin in May or early June at the NIH Clinical Center, according to National Cancer Institute (NCI) Pediatric Branch Chief Philip Pizzo. The clinical trial, which is expected to have a total enrollment of 27 to 36 children, will also be conducted at the Walter Reed Army Medical Center, Children's Hospital in Washington, D.C. and the University of Maryland. The trial involves three separate parts, Pizzo said. First, a dosage level will be determined in symptomatic children aged three months to 13 years. CD4 will then be administered to newborns at birth to three months of age. Finally, in what Pizzo characterized as the "most important" part of the study, CD4 will be administered to pregnant HIV-infected mothers during labor and delivery to study the ability of the drug to block transmission of HIV. NCI is also planning a Phase II pediatric study comparing intravenous AZT with oral AZT. The trial will focus on changes in the central nervous system, Pizzo says. Also scheduled to begin in May or June, the study will enroll 42 patients and be conducted at the Clinical Center, Children's Hospital in Washington, D.C., Walter Reed, the University of Maryland and Johns Hopkins University. Other NCI pediatric AIDS trials being conducted at the Clinical Center include: a Phase I trial of dideoxyinosine (DDI) which began in January and involves 14 HIV-infected children who have not been treated or who are AZT-intolerant; a Phase I trial of dideoxycytidine (DDC) alternating with AZT in 16 children that began last March; and a Phase I trial of GM-CSF (granulocyte-macrophage colony stimulating factor) combined with AZT that began in January and has enrolled one child. The latter trial is expected to be completed in late summer. The National Institute of Allergy and Infectious Diseases, together with Burroughs Wellcome, is sponsoring a Phase I AZT trial with newborns born to HIV-infected mothers. National Institute of Child Health and Human Development scientist Anne Willoughby spoke about the NIAID pediatric AIDS program at an April 7 policy conference in Washington, DC on AIDS in infants and children. The conference was sponsored by several groups, including the American Academy of Pediatrics, the Children's Defense Fund, and the George Washington University Medical Center. The NIAID newborn AZT trial is being conducted at Johns Hopkins, Duke University and Stanford University. Sixteen out of 18 children have been enrolled in the trial, which will examine the safety and efficacy of intravenous AZT treatment followed by oral treatment in newborns.