What is an investigational drug?
An investigational drug is one that is under study and is not approved by FDA for sale in the United States. Medical research studies are conducted to evaluate the safety and effectiveness of an investigational drug. These research studies are also called clinical trials. Once an investigational drug has been proven safe and effective in clinical trials, FDA may approve the drug for sale in the United States.
What is elvitegravir?
Elvitegravir, used as a stand alone agent, is an investigational drug for the treatment of HIV infection.
- Elvitegravir is an investigational anti-HIV drug included in the integrase inhibitor drug class.
- Integrase inhibitors prevent HIV from replicating by blocking integrase, an HIV enzyme. By preventing HIV from replicating, integrase inhibitors reduce the amount of HIV in the blood.
- Elvitegravir requires boosting with an additional drug, such as ritonavir (brand name: Norvir) or the investigational drug cobicistat.
- Elvitegravir is a component of the fixed-dose combination (FDC) anti-HIV drug elvitegravir/cobicistat/emtricitabine/tenofovir disoproxil fumarate (brand name: Stribild), approved by the U.S. Food and Drug Administration (FDA).
How are clinical trials of investigational drugs conducted?
Clinical trials are conducted in “phases.” Each phase has a different purpose and helps researchers answer different questions.
- Phase I trials: Researchers test an investigational drug in a small group of people (20–80) for the first time. The purpose is to evaluate its safety and identify side effects.
- Phase II trials: The investigational drug is administered to a larger group of people (100–300) to determine its effectiveness and to further evaluate its safety.
- Phase III trials: The investigational drug is administered to large groups of people (1,000–3,000) to confirm its effectiveness, monitor side effects, compare it with standard or equivalent treatments, and collect information that will allow the investigational drug to be used safely.
An investigational drug must be proven safe and effective in a Phase III clinical trial to be considered for traditional approval by the FDA for sale in the United States. Some drugs go through the FDA’s accelerated approval process and are approved before a Phase III clinical trial is complete. After a drug is approved by the FDA and made available to the public, researchers track its safety in Phase IV trials to seek more information about the drug’s risks, benefits, and optimal use.
In what phase of testing is elvitegravir?
Elvitegravir is currently being studied in a Phase III clinical trial.
What have recent studies shown about elvitegravir?
In a Phase III study, ritonavir-boosted elvitegravir taken once daily was compared with raltegravir (brand name: Isentress), an FDA-approved integrase inhibitor, taken twice daily in treatment-experienced HIV-infected participants. Study participants also received additional anti-HIV drugs as part of their optimized background therapy. (An optimized background regimen is a combination of drugs, chosen on the basis of a person’s resistance test results and treatment history, that are not being studied as the investigational drug[s] in the clinical trial, but are given to help control a participant’s HIV infection.) The background regimen therapy included a boosted protease inhibitor plus another anti-HIV drug.
In HIV treatment-experienced adults, ritonavir-boosted elvitegravir proved as effective as raltegravir. In terms of safety, elvitegravir was comparable to raltegravir. However, diarrhea was reported more frequently in patients taking elvitegravir, while elevated liver enzymes occurred more frequently in patients receiving raltegravir.
More information on Phase III testing of elvitegravir is available from these sources:
- Elvitegravir study results presented at the 19th International AIDS Conference, July 22-27, 2012:
- PubMed abstracts:
- Molina JM, LaMarca A, Andrade-Villanueva J, et al. Efficacy and safety of once daily elvitegravir versus twice daily raltegravir in treatment-experienced patients with HIV-1 receiving a ritonavir-boosted protease inhibitor: randomised, double-blind, phase 3, non-inferiority study. Lancet Infect Dis. 2012 Jan;12(1):27-35. Epub 2011 Oct 18.
- Wills T, Vega V. Elvitegravir: a once-daily inhibitor of HIV-1 integrase. Expert Opin Investig Drugs. 2012;21(3):395-401. Epub 2012 Feb 10.
- Lee JS, Calmy A, Andrieux-Meyer I, et al. Review of the safety, efficacy, and pharmacokinetics of elvitegravir with an emphasis on resource-limited settings. HIV AIDS (Auckl). 2012;4:5-15. Epub 2012 Jan 12.
Where can I get more information about clinical trials studying elvitegravir?
More information about elvitegravir-related research studies
is available from the AIDSinfo
database of ClinicalTrials.gov
study summaries. Click on the title of any trial in the list to see the ClinicalTrials.gov
trial summary and more information about the study.
I am interested in participating in a clinical trial of elvitegravir. How can I find more information about participating in a clinical trial?
Clinical trials involve benefits and risks. Before deciding to participate in a clinical trial, talk to your health care provider. For more information on participating in clinical trials, visit NIH Clinical Research Trials and You
Last Reviewed: October 31, 2012
Last Updated: October 31, 2012