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A Study to Evaluate the Use of Cidofovir (an Experimental Drug) for the Treatment of Progressive Multifocal Leukoencephalopathy (PML) in AIDS Patients

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00000945
Recruitment Status : Completed
First Posted : August 31, 2001
Last Update Posted : February 17, 2012
Information provided by (Responsible Party):
National Institute of Allergy and Infectious Diseases (NIAID)

Brief Summary:

The purpose of this study is to evaluate the safety, tolerance, and overall effectiveness of cidovir to treat PML in AIDS patients.

PML is an opportunistic infection (HIV-associated, due to weak immune system) caused by a virus that attacks the brain. Cidovir has been used effectively to treat cytomegalovirus (CMV) of the eye. Cidovir could be an effective treatment for PML as well.

Condition or disease Intervention/treatment Phase
HIV Infections Leukoencephalopathy, Progressive Multifocal Drug: Cidofovir Drug: Probenecid Not Applicable

Detailed Description:

PML is a demyelinating disease of the brain's white matter, occurring when the JC virus infects the brain of patients infected with HIV-1. Cidofovir is known to be an effective treatment for cytomegalovirus of the eye and, in laboratory and animal testing, has also been shown to be effective against several other viruses. However, cidofovir is considered investigational as a treatment for PML.

In this multicenter, open-label study 24 patients receive cidofovir iv over 1 hr on days 0, 7, then every 2 wk for a total of 13 doses.

Oral probenecid is given 3h prior to and 2h and 8h following cidofovir administration. Nucleoside and non-nucleoside reverse transcriptors are withheld on days of probenecid administration. Protease inhibitors are continued during probenecid administration.

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Study Type : Interventional  (Clinical Trial)
Enrollment : 24 participants
Primary Purpose: Treatment
Official Title: A Pilot Study of the Effect of Cidofovir for the Treatment of Progressive Multifocal Leukoencephalopathy (PML) in Subjects With Acquired Immunodeficiency Syndrome (AIDS)
Actual Study Completion Date : March 2001

Resource links provided by the National Library of Medicine

MedlinePlus related topics: HIV/AIDS

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria

You may be eligible for this study if you:

  • Are HIV-positive.
  • Have had symptoms of PML for no more than 90 days before study entry, or have had abnormal neurological exams related to PML.
  • Have negative tests for bacterial or fungal infections.
  • Agree to practice abstinence or use effective methods of birth control during the study.
  • Are at least 18 years old.
  • Have a life expectancy of at least 6 months.

Exclusion Criteria

You will not be eligible for this study if you:

  • Have a history of uveitis.
  • Are allergic to sulfa drugs or probenecid.
  • Have had active opportunistic infections other than Kaposi's sarcoma within 30 days before study entry.
  • Have sickle cell anemia or trait.
  • Are pregnant or breast-feeding.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00000945

Show Show 19 study locations
Sponsors and Collaborators
National Institute of Allergy and Infectious Diseases (NIAID)
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Study Chair: Marra CM
Study Chair: Barker DE
Additional Information:
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Responsible Party: National Institute of Allergy and Infectious Diseases (NIAID) Identifier: NCT00000945    
Other Study ID Numbers: ACTG 363
11327 ( Registry Identifier: DAIDS ES )
First Posted: August 31, 2001    Key Record Dates
Last Update Posted: February 17, 2012
Last Verified: February 2012
Keywords provided by National Institute of Allergy and Infectious Diseases (NIAID):
Leukoencephalopathy, Progressive Multifocal
Central Nervous System Diseases
Acquired Immunodeficiency Syndrome
DNA, Viral
Anti-HIV Agents
Neurologic Examination
Renal Agents
Additional relevant MeSH terms:
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Acquired Immunodeficiency Syndrome
Leukoencephalopathy, Progressive Multifocal
HIV Infections
Lentivirus Infections
Retroviridae Infections
RNA Virus Infections
Virus Diseases
Sexually Transmitted Diseases, Viral
Sexually Transmitted Diseases
Immunologic Deficiency Syndromes
Immune System Diseases
Slow Virus Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Encephalitis, Viral
Central Nervous System Viral Diseases
Polyomavirus Infections
DNA Virus Infections
Infectious Encephalitis
Central Nervous System Infections
Demyelinating Diseases
Uricosuric Agents
Gout Suppressants
Antirheumatic Agents
Renal Agents