Clinical trial NCT00000945: A Study to Evaluate the Use of Cidofovir (an Experimental Drug) for the Treatment of Progressive Multifocal Leukoencephalopathy (PML) in AIDS Patients
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)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: HIV Infections
Leukoencephalopathy, Progressive Multifocal
Keywords: Leukoencephalopathy, Progressive Multifocal
Probenecid
HIV-1
Central Nervous System Diseases
Acquired Immunodeficiency Syndrome
DNA, Viral
cidofovir
Anti-HIV Agents
Neurologic Examination
Renal Agents
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID)
Sponsor class: NIH
First posted: 2001-08-31
Enrollment: 24
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Primary purpose: TREATMENT

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.

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.

Eligibility criteria: 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.

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: Univ of Alabama at Birmingham
state: Alabama
city: San Francisco
country: United States
facility: San Francisco AIDS Clinic / San Francisco Gen Hosp
state: California
city: San Francisco
country: United States
facility: San Francisco Gen Hosp
state: California
city: Denver
country: United States
facility: Univ of Colorado Health Sciences Ctr
state: Colorado
city: Washington
country: United States
facility: Howard Univ
state: District of Columbia
city: Chicago
country: United States
facility: Northwestern Univ Med School
state: Illinois
city: Chicago
country: United States
facility: Cook County Hosp
state: Illinois
city: Chicago
country: United States
facility: Rush Presbyterian - Saint Luke's Med Ctr
state: Illinois
city: Chicago
country: United States
facility: Louis A Weiss Memorial Hosp
state: Illinois
city: Baltimore
country: United States
facility: Johns Hopkins Hosp
state: Maryland
city: Buffalo
country: United States
facility: SUNY / Erie County Med Ctr at Buffalo
state: New York
city: New York
country: United States
facility: Beth Israel Med Ctr
state: New York
city: New York
country: United States
facility: Bellevue Hosp / New York Univ Med Ctr
state: New York
city: New York
country: United States
facility: Mount Sinai Med Ctr
state: New York
city: Rochester
country: United States
facility: Univ of Rochester Medical Center
state: New York
city: Chapel Hill
country: United States
facility: Univ of North Carolina
state: North Carolina
city: Cincinnati
country: United States
facility: Univ of Kentucky Lexington
state: Ohio
city: West Columbia
country: United States
facility: Julio Arroyo
state: South Carolina
city: Seattle
country: United States
facility: Univ of Washington
state: Washington
Study officials:
name: Marra CM
role: STUDY_CHAIR
name: Barker DE
role: STUDY_CHAIR
References:
citation: Marra CM, Rajicic N, Barker DE, Cohen BA, Clifford D, Donovan Post MJ, Ruiz A, Bowen BC, Huang ML, Queen-Baker J, Andersen J, Kelly S, Shriver S; Adult AIDS Clinical Trials Group 363 Team. A pilot study of cidofovir for progressive multifocal leukoencephalopathy in AIDS. AIDS. 2002 Sep 6;16(13):1791-7. doi: 10.1097/00002030-200209060-00012.