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Drugs

Rintatolimod

Rintatolimod

Other Names: AMP, Ampligen, Atvogen, poly(I)-poly(C12U), poly(I:C12U), polyI:polyC12U Drug Class: Immune Modulators Molecular Formula: (C10 H13 N4 O8 P)x.(C9 H14 N3 O8 P.C9 H13 N2 O9 P)x Registry Number: 38640-92-5 (CAS) Chemical Name: 5'-Inosinic acid, homopolymer, complex with 5'-cytidylic acid polymer with 5'-uridylic acid (1:1)
Chemical Class: Oligonucleotides Organization: Hemispherx; Rega Institute For Medical Research Phase of Development: Rintatolimod is in Phase IIb development for HIV.

(Compound details obtained from ChemIDplus Advanced,1 NIAID Therapeutics Database,2 IAS 2003 Abstract 596,3 and Immunotherapy article4)

What is an investigational drug?

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.

To learn more about investigational drugs, read the AIDSinfo What is an Investigational HIV Drug? fact sheet.

What is rintatolimod?

What is rintatolimod?

Rintatolimod is an investigational drug that is categorized as an immune modulator.3,4 Immune modulators (also called immunomodulators) are substances that help to activate, boost, or restore normal immune function.

HIV infection can make a person’s immune system not work properly over the course of the viral infection. For example, certain immune system cells or proteins may not be produced in normal amounts and may not work properly.5-9

Rintatolimod helps to activate the immune system. Researchers have studied whether rintatolimod can delay re-emergence of HIV during treatment interruptions of antiretroviral therapy (ART). ART is the recommended treatment for HIV infection and involves using a combination of different antiretroviral (ARV) drugs to prevent HIV from replicating. A treatment interruption is a planned break from ART to evaluate how well an investigational drug can maintain control of a participant’s viral load during a clinical trial. (Viral load is the amount of HIV in a blood sample.)3,4,10

Also, rintatolimod has been studied for various non-HIV-related uses, including to treat chronic fatigue syndrome and cancer and to boost the body’s immune response to a flu vaccine.11-14

How are clinical trials of investigational drugs conducted?

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.15

  • 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.15


In most cases, an investigational drug must be proven effective and must show continued safety in a Phase III clinical trial to be considered for approval by FDA for sale in the United States. Some drugs go through FDA’s accelerated approval process and are approved before a Phase III clinical trial is complete. After a drug is approved by 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.15

Some clinical trials are categorized as “a” or “b,” such as “Phase Ia” or “Phase IIb.” These different subphases typically mean that a study is researching certain types of information or using a certain type of participant population.

In what phase of testing is rintatolimod?

In what phase of testing is rintatolimod?

Rintatolimod for HIV treatment has been studied in a Phase IIb clinical trial.3

What are some studies on rintatolimod?

What are some studies on rintatolimod?

Study Names: AMP 720; NCT0035893
Sponsor: Hemispherx Biopharma 
Phase: IIb
Location: United States
Participants:

  • Participants were adults with HIV who had taken HIV medicines before entering the study.
  • Participants’ HIV levels were under control, with a viral load level of less than 50 copies/mL while on ART.
  • Participants had a CD4 count of at least 400 cells/mm3. (A CD4 count is a laboratory test that measures the number of CD4 cells in a sample of blood and is an important indicator of immune function.)

Purpose: The purpose of this study was to evaluate whether rintatolimod could extend the time people could stay off HIV medicines during interruptions of ART (planned breaks from treatment) before their HIV began to rebound. (A rebound is when there are detectable levels of HIV in the blood after a period of undetectable levels.)3,10

*This study has been completed.

For more details on this study, see the Health Professional version.

Another Phase II study of rintatolimod (known as AMP 719; NCT00355581) was stopped early and not completed. In AMP 719, investigators were planning to study the safety and effectiveness of adding twice weekly rintatolimod to ART in people whose HIV medicines weren’t controlling their HIV infection.16

What side effects might rintatolimod cause?

What side effects might rintatolimod cause?

In the Phase IIb AMP 720 study (NCT0035893), which looked at using rintatolimod during interruptions of ART, side effects were generally mild and cleared up without treatment.3,16

Information on possible side effects of rintatolimod is not complete. As testing of rintatolimod continues, additional information on possible side effects will be gathered.

Where can I get more information about clinical trials studying rintatolimod?

Where can I get more information about clinical trials studying rintatolimod?

More information about rintatolimod-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.

How can I find more information about participating in a clinical trial?

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.15

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.

References

References

  1. United States National Library of Medicine. ChemIDplus Advanced. Available at: https://chem.sis.nlm.nih.gov/chemidplus/rn/38640-92-5. Last accessed on July 28, 2017.
  2. National Institute of Allergy and Infectious Diseases (NIAID). NIAID ChemDB, HIV Drugs in Development. Available at: https://chemdb.niaid.nih.gov/DrugDevelopmentHIV.aspx. Last accessed on July 28, 2017.
  3. Blick G, Greiger-Zanlungo P, Strayer DR, Mitchell WM, Carter WA. Interim results of AMP720: a Phase IIb prospective, randomized, controlled study evaluating the immunomodulatory role of ampligen (poly I:poly C12U) against HIV during STI. International AIDS Society (IAS) Conference on HIV Pathogenesis and Treatment; July 13-16, 2003; Paris, France. Abstract 596. Available at: http://www.abstract-archive.org/Abstract/Share/37573. Last accessed on July 28, 2017.
  4. Nicodemus CF, Berek JS. TLR3 agonists as immunotherapeutic agents. Immunotherapy. 2010 Mar;2(2):137-40. Available at: http://www.futuremedicine.com/doi/full/10.2217/imt.10.8. Last accessed on July 28, 2017.
  5. Barouch DH, Deeks SG. Immunologic strategies for HIV-1 remission and eradication. Science. 2014 Jul 11; 345(6193): 169–174. Available at: http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4096716/. Last accessed on July 28, 2017.
  6. Lederman MM, Rodriguez B, Sieg S. Immunopathogenesis of HIV infection. In: Coffey S and Volberding P, eds. HIV InSite Knowledge Base. University of California San Francisco; 2004. Available at: http://hivinsite.ucsf.edu/InSite?page=kb-00&doc=kb-02-01-04. Last accessed on July 28, 2017.
  7. Kedzierska K, Crowe SM. Cytokines and HIV-1: interactions and clinical implications. Antivir Chem Chemother. 2001 May; 12(3): 133-50. Available at: http://www.ncbi.nlm.nih.gov/pubmed/12959322. Last accessed on July 28, 2017.
  8. Siliciano RF, Greene WC. HIV latency. Cold Spring Harb Perspect Med. 2011 Sep; 1(1): a007096. Available at: http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3234450/. Last accessed on July 28, 2017.
  9. Savarino A, Shytaj IL. Chloroquine and beyond: exploring anti-rheumatic drugs to reduce immune hyperactivation in HIV/AIDS. Retrovirology. 2015; 12: 51. Available at: http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4472405/. Last accessed on July 28, 2017.
  10. Hemispherx Biopharma. The Role of Ampligen in Strategic Therapeutic Intervention (STI) of Highly Active Anti-Retroviral Therapy (HAART): A Multi-Center, Randomized, Controlled Study of Ampligen Potentiation of the HAART-Free Interval. In: ClinicalTrials.gov. Bethesda (MD): National Library of Medicine (US). Registered on May 6, 2002. NLM Identifier: NCT00035893. Available at: https://www.clinicaltrials.gov/ct2/show/NCT00035893. Last accessed on July 28, 2017.
  11. Strayer DR, Carter WA, Stouch BC, et al. A Double-Blind, Placebo-Controlled, Randomized, Clinical Trial of the TLR-3 Agonist Rintatolimod in Severe Cases of Chronic Fatigue Syndrome. PLoS One.2012;7(3):e31334. Available at: http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3303772/. Last accessed on July 28, 2017.
  12. University of Washington. Phase I-II Study of HER2 Vaccination With Poly(I) • Poly(C12U) (Ampligen®) as an Adjuvant in Optimally Treated Breast Cancer Patients. In: ClinicalTrials.gov. Bethesda (MD): National Library of Medicine (US). Registered on May 16, 2011. NLM Identifier: NCT01355393. Available at: https://www.clinicaltrials.gov/ct2/show/NCT01355393. Last accessed on July 28, 2017.
  13. Pawel Kalinski. Randomized Phase I/II Evaluation of Neoadjuvant Administration of a Chemokine-Modulatory Regimen in Patients With Recurrent Resectable Colorectal Cancer. In: ClinicalTrials.gov. Bethesda (MD): National Library of Medicine (US). Registered on February 29, 2012. NLM Identifier: NCT01545141. Available at: https://www.clinicaltrials.gov/ct2/show/NCT01545141. Last accessed on July 28, 2017.
  14. Hemispherx Biopharma. A Phase I/II, Two-Staged, Single-Center, Randomized, Double-Blind, Antibody Titer Study to Assess Immunogenicity and Safety of FluMist® Intranasal Influenza Vaccine Administered With and Without a TLR-3 Agonist, Ampligen®. In: ClinicalTrials.gov. Bethesda (MD): National Library of Medicine (US). Registered on May 2, 2012. NLM Identifier: NCT01591473. Available at: https://www.clinicaltrials.gov/ct2/show/NCT01591473. Last accessed on July 28, 2017.
  15. National Institutes of Health (NIH). NIH Clinical Research Trials and You. Available at: https://www.nih.gov/health-information/nih-clinical-research-trials-you. Last accessed on July 28, 2017.
  16. Hemispherx Biopharma. A Multi-Center, Randomized, Controlled Study of the Biological Actions of Ampligen as an Adjunct to HAART in HIV Disease. In: ClinicalTrials.gov. Bethesda (MD): National Library of Medicine (US). Registered on May 3, 2002. NLM Identifier: NCT00035581. Available at: https://www.clinicaltrials.gov/ct2/show/NCT00035581. Last accessed on July 28, 2017.
 

Last Reviewed: July 28, 2017