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Cidofovir in Treating HIV-Infected Patients With High-Grade Squamous Intraepithelial Lesions of the Skin Near the Anus

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.
 
ClinicalTrials.gov Identifier: NCT00550589
Recruitment Status : Completed
First Posted : October 30, 2007
Results First Posted : June 22, 2011
Last Update Posted : December 21, 2015
Sponsor:
Collaborators:
National Cancer Institute (NCI)
The Emmes Company, LLC
Information provided by (Responsible Party):
AIDS Malignancy Consortium

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Anal Cancer
Precancerous Condition
Interventions Drug: cidofovir
Genetic: DNA methylation analysis
Genetic: gene expression analysis
Genetic: polymerase chain reaction
Procedure: biopsy
Procedure: histopathologic examination
Enrollment 33
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Cidofovir
Hide Arm/Group Description 1.0% topical cidofovir cream
Period Title: Overall Study
Started 33
Completed 33
Not Completed 0
Arm/Group Title Cidofovir
Hide Arm/Group Description 1.0% topical cidofovir cream
Overall Number of Baseline Participants 33
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 33 participants
<=18 years
0
   0.0%
Between 18 and 65 years
31
  93.9%
>=65 years
2
   6.1%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 33 participants
44  (8.9)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 33 participants
Female
9
  27.3%
Male
24
  72.7%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 33 participants
33
1.Primary Outcome
Title Proportion of Patients With Regression of Perianal High-grade Squamous Intraepithelial Lesions (HSIL)
Hide Description [Not Specified]
Time Frame 6 weeks after treatment discontinuation
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Cidofovir
Hide Arm/Group Description:
1.0% topical cidofovir cream
Overall Number of Participants Analyzed 33
Measure Type: Number
Unit of Measure: proportion of participants
0.515
2.Primary Outcome
Title Safety and Tolerability of Topical Cidofovir as Assessed by NCI CTCAE v3.0
Hide Description Number of study patients who had a serious adverse event
Time Frame Every 2 weeks on study, 6 weeks after treatment discontinuation
Hide Outcome Measure Data
Hide Analysis Population Description
All enrolled patients
Arm/Group Title Cidofovir
Hide Arm/Group Description:
1% cidofovir
Overall Number of Participants Analyzed 33
Measure Type: Number
Unit of Measure: participants
3
3.Secondary Outcome
Title Human Papilloma Virus (HPV) DNA Type in Perianal HSIL and Normal Perianal Tissue
Hide Description Number of patients with HPV16 at baseline in perianal HSIL and normal perianal tissue
Time Frame Baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Number of patients with tissue samples available at baseline
Arm/Group Title Cidofovir
Hide Arm/Group Description:
1.0% topical cidofovir cream
Overall Number of Participants Analyzed 30
Measure Type: Number
Unit of Measure: participants
16
4.Secondary Outcome
Title Correlation of Clinical Regression of Perianal HSIL With Clearance of HPV DNA
Hide Description Number of patients who cleared HPV among those who had a complete or partial response
Time Frame 6 weeks after treatment discontinuation
Hide Outcome Measure Data
Hide Analysis Population Description
Participants who had a partial or complete response and for whom pre and post-treatment HPV data were available
Arm/Group Title Cidofovir
Hide Arm/Group Description:

1.0% topical cidofovir cream

cidofovir: 1.0% topical cream self-applied once daily for 5 consecutive days, with no treatment for the remaining 9 days (a treatment cycle). Subjects will receive up to 6 cycles of treatment.

DNA methylation analysis: formalin fixed biopsy collected at baseline and 6 weeks after treatment discontinuation

gene expression analysis: punch biopsy collected at baseline, after cycle 1, and 6 weeks after treatment discontinuation

polymerase chain reaction: performed on punch biopsy specimens collected at baseline, after cycle 1, and 6 weeks after treatment discontinuation

biopsy: punch biopsy collected at baseline, after cycle 1, and 6 weeks after treatment discontinuation

histopathologic examination: Evaluated at baseline and 6 weeks after treatment discontinuation
Overall Number of Participants Analyzed 12
Measure Type: Number
Unit of Measure: participants
2
5.Secondary Outcome
Title Identification of HPV-DNA Types Present in the Anus
Hide Description Number of patients with HPV16 type present in the anus from anal swab or cytobrush at baseline
Time Frame Baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Number of patients with anal swabs or cytobrush results at baseline
Arm/Group Title Cidofovir
Hide Arm/Group Description:
1.0% topical cidofovir cream
Overall Number of Participants Analyzed 32
Measure Type: Number
Unit of Measure: participants
14
6.Secondary Outcome
Title Identification of Abnormally Methylated Genes in Perianal Dysplasia
Hide Description Identification of abnormally methylated genes in perianal dysplasia
Time Frame Baseline, after cycle 1, and 6 weeks after treatment discontinuation
Hide Outcome Measure Data
Hide Analysis Population Description
The lab analysis of genes was not performed
Arm/Group Title Cidofovir
Hide Arm/Group Description:
1.0% topical cidofovir cream
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
7.Secondary Outcome
Title Distribution of Abnormally Methylated Genes Among HSIL, Low-grade Squamous Intraepithelial Lesions, and Normal Perianal Skin
Hide Description [Not Specified]
Time Frame Baseline, after cycle 1, and 6 weeks after treatment discontinuation
Hide Outcome Measure Data
Hide Analysis Population Description
The gene analysis was not done
Arm/Group Title Cidofovir
Hide Arm/Group Description:
1.0% topical cidofovir cream
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
8.Secondary Outcome
Title Changes in Gene Expression in Perianal HSIL After Exposure to Cidofovir as Assessed by RNA Microarray Analysis
Hide Description [Not Specified]
Time Frame Baseline, after cycle 1, and 6 weeks after treatment discontinuation
Hide Outcome Measure Data
Hide Analysis Population Description
The gene expression analysis was not done
Arm/Group Title Cidofovir
Hide Arm/Group Description:
1.0% topical cidofovir cream
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Cidofovir
Hide Arm/Group Description 1.0% topical cidofovir cream
All-Cause Mortality
Cidofovir
Affected / at Risk (%)
Total   --/--    
Hide Serious Adverse Events
Cidofovir
Affected / at Risk (%) # Events
Total   3/33 (9.09%)    
Infections and infestations   
Cellulitis  1/33 (3.03%)  1
Pneumonia  1/33 (3.03%)  1
Skin and subcutaneous tissue disorders   
Dermatology/skin  2/33 (6.06%)  2
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Cidofovir
Affected / at Risk (%) # Events
Total   32/33 (96.97%)    
Blood and lymphatic system disorders   
Hemoglobin  3/33 (9.09%)  3
Hemorrhage, Anus  5/33 (15.15%)  7
Hemorrhage, other  4/33 (12.12%)  5
Neutropenia  3/33 (9.09%)  6
Anemia  3/33 (9.09%)  3
Gastrointestinal disorders   
Constipation  3/33 (9.09%)  3
Constitutional Symptoms  4/33 (12.12%)  4
Diarrhea  6/33 (18.18%)  8
Hemorrhage, Rectum  2/33 (6.06%)  2
Nausea  4/33 (12.12%)  11
Pain, anus  9/33 (27.27%)  15
Vomiting  5/33 (15.15%)  5
General disorders   
Fatigue  3/33 (9.09%)  4
Pain, other  2/33 (6.06%)  3
Headache  6/33 (18.18%)  7
Pain, perineum  14/33 (42.42%)  49
Infections and infestations   
Infection, other  3/33 (9.09%)  3
Infection, lung  2/33 (6.06%)  2
Infection, upper airway  2/33 (6.06%)  2
Infection, vagina  2/33 (6.06%)  2
Infection, skin  6/33 (18.18%)  9
Upper respiratory infection  2/33 (6.06%)  2
Vaginal infection  3/33 (9.09%)  4
Metabolism and nutrition disorders   
AST,SGOT Elevation  2/33 (6.06%)  2
Metabolic, other  6/33 (18.18%)  7
Musculoskeletal and connective tissue disorders   
Pain, back  2/33 (6.06%)  2
Renal and urinary disorders   
Hemoglobinuria  2/33 (6.06%)  4
Proteinuria  7/33 (21.21%)  7
Respiratory, thoracic and mediastinal disorders   
Cough  4/33 (12.12%)  4
Pulmonary, other  2/33 (6.06%)  2
Respiratory, thoracic, other  2/33 (6.06%)  2
Skin and subcutaneous tissue disorders   
Dermatology/skin  13/33 (39.39%)  21
Pruritus  11/33 (33.33%)  21
Decubitus ulcer  2/33 (6.06%)  3
Ulceration  12/33 (36.36%)  25
Skin ulceration  17/33 (51.52%)  30
[Not Specified]
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Jeannette Lee
Organization: Statistical Center of AIDS Malignancy Consortium
Phone: (501) 526-6712
EMail: jylee@uams.edu
Publications of Results:
Stier EA, Goldstone SE, Einstein MH, Jay N, Berry JM, Wilkin T, Lee JY, Darragh TM, Da Costa M, Panther L, Aboulafia D, Palefsky JM. Safety and efficacy of topical cidofovir to treat high-grade perianal and vulvar intraepithelial neoplasia in HIV-positive men and women. AIDS. 2013 Feb 20;27(4):545-51. doi: 10.1097/QAD.0b013e32835a9b16.
Layout table for additonal information
Responsible Party: AIDS Malignancy Consortium
ClinicalTrials.gov Identifier: NCT00550589    
Other Study ID Numbers: AMC-046
U01CA121947 ( U.S. NIH Grant/Contract )
CDR0000570720 ( Other Identifier: NCI )
First Submitted: October 26, 2007
First Posted: October 30, 2007
Results First Submitted: May 24, 2011
Results First Posted: June 22, 2011
Last Update Posted: December 21, 2015