What is an investigational drug?
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.
What is AMD-070?
AMD-070 is an investigational drug that is being studied for the treatment of HIV infection.
AMD-070 belongs to a class (group) of HIV drugs called entry and fusion inhibitors.2
Entry and fusion inhibitors block HIV from getting into and infecting certain cells of the immune system. This prevents HIV from multiplying and can reduce the amount of HIV in the body.
AMD-070 works by attaching to one of two proteins on the surface of the immune cells. These proteins are called the CCR5 and CXCR4 coreceptors. AMD-070 attaches to the CXCR4 coreceptor. When AMD-070 attaches to the CXCR4 coreceptor, certain strains of HIV—called X4-tropic virus—cannot attach to, enter, or infect the cell.3
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.4
- 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.4
In most cases, an investigational drug must be proven safe and effective in a Phase III clinical trial to be considered for 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.4
In what phase of testing is AMD-070?
AMD-070 has been studied in a Phase II clinical trial.2
What have recent studies shown about AMD-070?
Two early Phase I-II studies (known as XACT and ACTG A5210) looked at the efficacy of AMD-070 in participants infected with X4-tropic HIV (virus that uses CXCR4 as a coreceptor). In both studies, AMD-070 was taken twice daily by HIV-infected adults. Some of the participants had never taken HIV medicines before entering the study (treatment-naive) and others had taken HIV medicines previously (treatment-experienced). The treatment-experienced adults were required to be off HIV medicines for at least 14 days before starting AMD-070. Study participants did not receive additional HIV medicines as part of a background regimen. (A background regimen is a combination of drugs that are not being studied as the investigational drug[s] in the clinical trial, but are being given to help control a participant’s HIV infection.) A control arm was not used in these studies.5,6
In these studies, AMD-070 was shown to have activity against X4-tropic HIV. In terms of safety in the XACT Study, the most common side effects were gastrointestinal symptoms and headache, and there were no drug-related serious adverse events.5,6
Because of liver-associated side effects in long-term animal studies, the development of AMD-070 was previously put on clinical hold.7
What side effects might AMD-070 cause?
In the 10-day XACT Study discussed under the previous question, the following side effects were reported: mild gastrointestinal symptoms, including diarrhea and gas (flatulence); headache; and dizziness.5
Because AMD-070 is still being studied, information on possible side effects of the drug is not complete. As testing of AMD-070 continues, additional information on possible side effects will be gathered.
Where can I get more information about clinical trials studying AMD-070?
More information about AMD-070-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 AMD-070. How can I find more information about participating in a clinical trial?
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.4
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
. United States National Library of Medicine. ChemIDplus Advanced
. National Institute of Allergy and Infectious Diseases (NIAID). NIAID ChemDB, HIV Drugs in Development
. Wong R, Bodart V, Metz M, Labrecque J, Bridger G, Fricker S. Understanding the Interactions Between CXCR4 and AMD11070, a First-in-Class Small Molecule Antagonist of the HIV Coreceptor
. Poster presented at: 14th Conference on Retroviruses and Opportunistic Infections (CROI); February 25-28, 2007; Los Angeles, CA. Poster 495.
. National Institutes of Health (NIH). NIH Clinical Research Trials and You
. Moyle G, DeJesus E, Boffito M, et al. CXCR4 Antagonism: Proof of Activity with AMD11070
. Poster presented at: 14th Conference on Retroviruses and Opportunistic Infections (CROI); February 25-28, 2007; Los Angeles, CA. Poster 511.
. Saag M, Rosenkranz S, Becker S, et al. Proof of Concept of Antiretroviral Activity of AMD11070 (an Orally Administered CXCR4 Entry Inhibitor): Results of the First Dosing Cohort A Studied in ACTG Protocol A5210
. Paper presented at: 14th Conference on Retroviruses and Opportunistic Infections (CROI); February 25-28, 2007; Los Angeles, CA. Paper 512.
. Moyle G, DeJesus E, Boffito M, et al. Proof of activity with AMD11070, an orally bioavailable inhibitor of CXCR4-tropic HIV type 1
. Clin Infect Dis. 2009 Mar 15;48(6):798-805.
Last Reviewed: May 29, 2013
Last Updated: May 29, 2013