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 PRO-140?
PRO-140 is an investigational drug for the treatment of HIV infection.
- PRO-140 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.
- PRO-140 is a monoclonal antibody that specifically binds to the CCR5 coreceptor located on the surface of certain immune cells, such as CD4 cells. When PRO-140 binds to the CCR5 coreceptor, certain types of HIV (R5-tropic virus) cannot bind to, enter, or infect the immune cell.
- PRO-140 binds to the CCR5 coreceptor in a way that is unique from the way that small-molecule CCR5 inhibitors, such as maraviroc (brand name: Selzentry), do. These binding differences may allow for a distinct resistance profile.
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 PRO-140?
PRO-140 is currently being studied in Phase II clinical trials.
What have recent studies shown about PRO-140?
In an early Phase II study, PRO-140, given by subcutaneous (also called SC) infusion (over a 5-minute period) at different strengths and dosing frequencies (every week or every other week), was compared with placebo in treatment-naive and treatment-experienced HIV-infected participants. Treatment-experienced participants were required to be off anti-HIV therapy for at least 12 weeks prior to starting the trial. Study participants included only those individuals who were shown to have R5-tropic virus.
In this study, PRO-140 at all dosages studied was shown to have significant antiviral activity. In terms of safety, PRO-140 was generally well tolerated. No drug-related serious adverse events occurred.
An additional Phase II trial of long-acting subcutaneous PRO-140 is underway in HIV-infected injection drug users with viral rebound and poor adherence to prior antiretroviral therapy.
More information on Phase II testing of PRO-140 is available from these sources:
Where can I get more information about clinical trials studying PRO-140?
More information about PRO-140-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 PRO-140. 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 22, 2012
Last Updated: October 22, 2012