An investigational drug is one that is under study and is not approved by the U.S. Food and Drug Administration (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.
Tucaresol is an investigational drug that is categorized as an immune modulator.2 Immune modulators (also called immunomodulators) are substances that help to activate, boost, or restore normal immune function. Tucaresol is being studied for its ability to help restore or boost the immune systems of HIV-infected individuals. The drug may help to repair damage that HIV has done to the immune system and protect the long-term health of HIV-infected individuals.3-6
Clinical trials are conducted in phases. Each phase has a different purpose and helps researchers answer different questions.7
Tucaresol is being studied in Phase II clinical trials.2
A Phase I/II study compared the immune modulating effects of tucaresol in four groups of HIV-infected participants.
In the Phase I/II study discussed under the previous question, the most common side effects that occurred in the treatment-experienced participants who were taking ART and tucaresol were low-grade fever and other mild constitutional symptoms. (Constitutional symptoms are symptoms affecting the whole body, such as fever and weight loss.)3
Information on possible side effects of the drug is not complete. If testing of tucaresol continues, additional information on possible side effects will be gathered.
More information about tucaresol-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.
Participating in a clinical trial can provide benefits. For example, a volunteer participant can benefit from new research treatments before they are widely available. Participants also receive regular and careful medical attention from a research team that includes doctors and other health professionals. However, clinical trials may also involve risks of varying degrees, such as unpleasant, serious, or even life-threatening side effects from the treatment being studied.7
Your health care provider can help you decide whether participating in a clinical trial is right for you. For more information, visit NIH Clinical Research Trials and You.
1. United States National Library of Medicine. ChemIDplus Advanced. Last accessed on April 23, 2014.
2. National Institute of Allergy and Infectious Diseases (NIAID). NIAID ChemDB, HIV Drugs in Development. Last accessed on April 23, 2014.
3. Gori A, Trabattoni D, Bandera A, et al. Immunomodulation induced by tucaresol in HIV infection: results of a 16 week pilot Phase I/II trial. Antivir Ther. 2004 Aug;9(4):603-14. Last accessed on April 23, 2014.
4. Corbeau P, Reynes J. Immune reconstitution under antiretroviral therapy: the new challenge in HIV-1 infection. Blood. 2011 May 26;117(21):5582-90. Last accessed on April 23, 2014.
5. Lévy Y. Immunotherapy in HIV infection; current and future challenges. Retrovirology. 2010; 7(Suppl 1): I13. Last accessed on April 23, 2014.
6. Napolitano LA. Approaches to immune reconstitution in HIV infection. Top HIV Med. 2003 Sep-Oct;11(5):160-3. Last accessed on April 23, 2014.
7. National Institutes of Health (NIH). NIH Clinical Research Trials and You. Last accessed on April 23, 2014.
8. Bandera A, Gori A, Trabattoni D, et al. Positive Immunomodulatory Effects of Tucaresol in HIV-infected Patients: Results from a Phase I/II Trial after 40 Weeks of Follow-up. Abstract presented at: 10th Conference on Retroviruses and Opportunistic Infections (CROI); February 10-14, 2003; Boston, MA. Abstract 654. Last accessed on April 23, 2014.
9. Gazzola L, Marchetti G, Bandera A, et al. Dynamics of T Cells Homeostasis Induced by Tucaresol. Abstract presented at: 11th Conference on Retroviruses and Opportunistic Infections (CROI); February 8-11, 2004; San Francisco, CA. Abstract 523. Last accessed on April 23, 2014.
Last Reviewed: April 23, 2014
Last Updated: January 21, 2015