Clinical Trials for AIDS Therapies

Date: February 1, 1997
Source: National Institutes of Health (NIH)
Author: National Institute of Allergy and Infectious Diseases (NIAID)

The National Institute of Allergy and Infectious Diseases (NIAID) has the lead responsibility within the National Institutes of Health for the discovery and development of interventions to treat or prevent HIV infection and its complications in adults and children. In keeping with this mission, the Institute's research effort encompasses primary HIV disease, opportunistic infections, oncology and neurology. There is also a comprehensive HIV research program that focuses on identifying and improving interventions for the prevention of HIV infection and its sequelae in infants.

NIAID's extensive portfolio of clinical research on new HIV therapeutics includes studies conducted through several multicenter clinical trials networks and within its intramural facilities in the Clinical Center in Bethesda. Although each network has distinct capabilities, the research is very much intertwined and, taken together, represents the largest AIDS therapy research initiative in the world. These networks serve a large patient base and provide extensive clinical expertise and sophisticated laboratory capabilities, thereby giving NIAID an extraordinary ability to assess state-of-the-art therapies and further our understanding of HIV pathogenesis.

The extramural clinical trials networks supported by NIAID's Division of AIDS include: the Adult AIDS Clinical Trials Group (AACTG), the Pediatric AIDS Clinical Trials Group (PACTG), the Terry Beirn Community Programs for Clinical Research on AIDS (CPCRA) and the Strategic Program for Innovative Research on AIDS (SPIRAT). The Adult and Pediatric ACTGs are multicenter cooperative clinical trials groups involved in all phases of clinical research, from early safety studies to large-scale efficacy trials; they evaluate new drugs and drug combinations for the treatment of HIV infection in adults and children, respectively. The PACTG also evaluates regimens for the prevention of maternal-fetal HIV transmission. The CPCRA is based mainly in primary care settings and principally conducts large (clinical endpoint) studies. The SPIRAT integrates both clinical and preclinical research focused on innovative therapeutic approaches, such as immune system restoration, DNA-based therapeutic vaccines and gene therapy.

Scientists in NIAID's Division of Intramural Research focus primarily on intensive clinical/laboratory investigations involving novel agents or modalities.

The clinical research conducted by investigators in these networks has yielded valuable information that has provided the basis for much of the current state-of-the-art treatment for HIV disease. For example, NIAID-supported clinical trials have:

  • demonstrated and defined the efficacy of zidovudine (AZT), zalcitibine (ddC), didanosine (ddI) and stavudine (d4T) as monotherapies and in combination regimens for a wide variety of clinical conditions.
  • demonstrated that combination regimens employing two or more antiretroviral agents are more effective than AZT monotherapy, particularly early in disease.
  • defined the standard of care for acute treatment and prophylaxis of Pneumocystis carinii pneumonia, treatment and maintenance for cryptococcal meningitis (fluconazole), treatment and maintenance therapy for disseminated histoplasmosis (itraconazole), treatment of disseminated Mycobacterium avium complex (clarithromycin), management of cytomegalovirus retinitis and acyclovir-resistant herpes simplex (foscarnet).
  • assessed and validated assays for use as surrogate markers of drug effectiveness including p24 antigen, quantitative peripheral blood mononuclear cells (PBMC), microculture and DNA/RNA (PCR).
  • developed state-of-the-art study design methodology for HIV disease (e.g., endpoints) to permit rapid clinical and laboratory evaluation of antiretroviral compounds.

NIAID's pediatric clinical research effort has significantly advanced the treatment and prevention of HIV infection in infants and children. Through clinical trials, NIAID has:

  • demonstrated that AZT therapy reduced dramatically the likelihood of childhood HIV infection resulting from transmission from infected mothers during pregnancy or delivery.
  • demonstrated that ddI or ddI plus AZT are superior to AZT monotherapy for children with symptomatic HIV infection or AIDS.
  • demonstrated the effectiveness of IVIG for preventing serious bacterial infections in children with HIV.
  • developed guidelines for the prevention of PCP in HIV-infected children.

Over the next several years, NIAID research will continue to define the standard of care for HIV-infected people of all ages. Particular emphasis will be placed on the use of combination regimens with protease inhibitors.

For More Information About Clinical Trials

For further information about any NIAID-supported clinical trials, call 1-800-TRIALS-A (1-800-243-7012 TTY/TDD) weekdays from 9 a.m. to 7 p.m. Eastern Time, or visit the web site at or For further information about clinical trials conducted by the NIAID DIR AIDS program, call 1-800-243-7644 weekdays from 9 a.m. to 5 p.m. Eastern Time.

NIAID, a component of the National Institutes of Health (NIH), supports research on AIDS, tuberculosis and other infectious diseases, as well as allergies and immunology. NIH is an agency of the U.S. Department of Health and Human Services.

Prepared by:
Office of Communications National Institute of Allergy and Infectious Diseases National Institutes of Health Bethesda, MD 20892
U.S. Department of Health and Human Services