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 for the treatment of HIV infection.
- AMD-070 is an investigational anti-HIV drug included in the entry inhibitor drug class.
- Entry inhibitors interfere with the first step in the HIV life cycle—binding and fusion to target cells. By preventing HIV from entering target immune cells, entry inhibitors stop HIV from replicating and reduce the amount of HIV in the blood.
- AMD-070 is a small molecule that specifically binds to the CXCR4 coreceptor located on the surface of certain immune cells, such as CD4 cells. When AMD-070 binds to the CXCR4 coreceptor, certain types of HIV (X4-tropic virus) cannot bind to, enter, or infect the immune cell.
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 AMD-070?
AMD-070 has been studied in a Phase II clinical trial.
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 in treatment-naive and treatment-experienced HIV-infected adults. (The treatment-experienced adults were required to be off anti-HIV therapy for at least 14 days prior to starting AMD-070.) Study participants did not receive additional anti-HIV drugs 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.
In these studies, AMD-070 was shown to have activity against X4-tropic HIV. In terms of safety, AMD-070 was reportedly well tolerated in one study, with the most common side effect being mild gastrointestinal symptoms; in the other study, the most common side effect was headache.
Because of liver-associated side effects in long-term animal studies, the development of AMD-070 was previously put on clinical hold.
More information on Phase I-II testing of AMD-070 is available from these sources:
- AMD-070 study results presented at the 14th Conference on Retroviruses and Opportunistic Infections (CROI), February 25–28, 2007:
- PubMed abstract:
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?
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: November 19, 2012
Last Updated: November 19, 2012