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Use of Cidofovir for Recurrent Respiratory Papillomatosis

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ClinicalTrials.gov Identifier: NCT00205374
Recruitment Status : Completed
First Posted : September 20, 2005
Results First Posted : July 18, 2017
Last Update Posted : February 26, 2019
Sponsor:
Information provided by (Responsible Party):
University of Wisconsin, Madison

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Double (Participant, Investigator);   Primary Purpose: Treatment
Condition Recurrent Respiratory Papillomatosis
Interventions Drug: Cidofovir
Drug: Placebo
Enrollment 19
Recruitment Details The participants in this randomized, double-blind, placebo-controlled clinical trial were 19 consecutive adult or pediatric patients who presented to the University of Wisconsin Otolaryngology-Head and Neck Surgery Clinic with a diagnosis of RRP who met inclusio/exclusion criteria and gave informed consent to participate.
Pre-assignment Details Before inclusion in this study, and 48 hours before each cidofovir injection, laboratory tests confirmed that the (female) participants were not pregnant and that laboratory findings were not exclusionary for study participation.
Arm/Group Title Cidofovir Placebo
Hide Arm/Group Description With regard to cidofovir concentration, the FDA has allowed us to inject a concentration of 5 mg/ml into both children and adults. The injection will add less than 2 additional minutes to the surgery time and discharge time will not be affected. Because the volumes of cidofovir injected into the airway will be reasonably small (typically less than 2 mL), the total systemic dose of cidofovir administered per visit will be far below the FDA-approved systemic limit of 5 mg/kg for HIV-related CMV retinitis. No more than 6 treatments were performed per patient within the 12-month time interval of the study. The placebo treatment was injection of saline solution that had a color and viscosity identical to those of the active drug. Up to 6 injections per participant were given during the study.
Period Title: Overall Study
Started 10 9
Completed 10 9
Not Completed 0 0
Arm/Group Title Cidofovir Placebo Total
Hide Arm/Group Description On the baseline study day, patients will be randomized into either a treatment group (cidofovir injection) or a placebo group. A restricted randomization procedure, in groups of 4, will be used to encourage uniformity in sample sizes between groups. On the baseline study day, patients will be randomized into either a treatment group (cidofovir injection) or a placebo group. A restricted randomization procedure, in groups of 4, will be used to encourage uniformity in sample sizes between groups. Total of all reporting groups
Overall Number of Baseline Participants 10 9 19
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants 9 participants 19 participants
<=18 years
2
  20.0%
3
  33.3%
5
  26.3%
Between 18 and 65 years
8
  80.0%
6
  66.7%
14
  73.7%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 10 participants 9 participants 19 participants
33  (18) 29  (12) 31  (15)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants 9 participants 19 participants
Female
2
  20.0%
1
  11.1%
3
  15.8%
Male
8
  80.0%
8
  88.9%
16
  84.2%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 10 participants 9 participants 19 participants
10 9 19
1.Primary Outcome
Title Change in Papilloma Severity
Hide Description

Derkay Severity Score. Minimum Score is Zero. Maximum score is 86. Treatment of RRP with cidofovir injection will be considered efficacious if drug group patients experience clinically or statistically significant changes in papilloma severity and inter-surgery time intervals (relative to pre-treatment assessments), when compared with placebo group patients.

The scale rates - Voice (normal 0, abnormal 1, aphonic 2) Stridor (absent 0, present with activity 1, present at rest 2) Urgency of the intervention (scheduled 0, elective 1, urgent 2, emergent 3) Respiratory distress (none 0, mild 1, mod 2, severe 3, extreme 4).

For multiple anatomical sites (18 or more) in the upper airway, left and right sides, lesions are rated as 0=none, 1=surface lesion, 2=raised lesion, and 3=bulky lesion).

A higher rating value indicates more advanced disease and a worse outcome.

Time Frame Baseline, 2 months, and 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Cidofovir Placebo
Hide Arm/Group Description:
On the baseline study day, patients will be randomized into either a treatment group (cidofovir injection) or a placebo group. A restricted randomization procedure, in groups of 4, will be used to encourage uniformity in sample sizes between groups.
On the baseline study day, patients will be randomized into either a treatment group (cidofovir injection) or a placebo group. A restricted randomization procedure, in groups of 4, will be used to encourage uniformity in sample sizes between groups.
Overall Number of Participants Analyzed 10 9
Mean (Full Range)
Unit of Measure: scores on a scale
Baseline
13.2
(5 to 33)
12
(5 to 23)
2 months
3.7
(0 to 11)
3.6
(1 to 11)
12 months
2.7
(0 to 13)
5.1
(0 to 16)
2.Secondary Outcome
Title 12-month Change in Voice Handicap Index (VHI) Score
Hide Description

Voice Handicap Index. This scale rates a patient's perception of voice related handicap in the domains of functional, physical, and emotional. There are 10 questions for each domain. Each question is rated by the subject on a 5-point scale, never =0, almost never =1, sometimes =2, almost always =3, always =4). A lower total score or domain score indicates improved, or less, voice handicap. Thus, the maximum total score is 4 X 30 = 120. For each domain, the maximum score is 4 X 10 = 40. Scores of 0 are the lowest possible score. In the paper we say that the Maximum score is 100, but obviously is it actually 120. Complete scale = 0-120.

Lower score indicates improved perceived voice-realted quality of life.

Time Frame 2 months, and 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
[raw data no longer exist for this study, there a no data to report for a Baseline measure]
Arm/Group Title Cidofovir Placebo
Hide Arm/Group Description:
On the baseline study day, patients will be randomized into either a treatment group (cidofovir injection) or a placebo group. A restricted randomization procedure, in groups of 4, will be used to encourage uniformity in sample sizes between groups.
On the baseline study day, patients will be randomized into either a treatment group (cidofovir injection) or a placebo group. A restricted randomization procedure, in groups of 4, will be used to encourage uniformity in sample sizes between groups.
Overall Number of Participants Analyzed 10 9
Mean (Full Range)
Unit of Measure: scores on a scale
2 months
22
(0 to 59)
32
(8 to 61)
12 months
31
(2 to 63)
42
(0 to 93)
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Cidofovir Placebo
Hide Arm/Group Description On the baseline study day, patients will be randomized into either a treatment group (cidofovir injection) or a placebo group. A restricted randomization procedure, in groups of 4, will be used to encourage uniformity in sample sizes between groups. On the baseline study day, patients will be randomized into either a treatment group (cidofovir injection) or a placebo group. A restricted randomization procedure, in groups of 4, will be used to encourage uniformity in sample sizes between groups.
All-Cause Mortality
Cidofovir Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/-- 
Hide Serious Adverse Events
Cidofovir Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   0/10 (0.00%)   0/9 (0.00%) 
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 1%
Cidofovir Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   0/10 (0.00%)   0/9 (0.00%) 
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: J. Scott McMurray, MD
Organization: University of Wisconsin-Madison
Phone: 608-262-7181
EMail: mcmurray@surgery.wisc.edu
Layout table for additonal information
Responsible Party: University of Wisconsin, Madison
ClinicalTrials.gov Identifier: NCT00205374    
Other Study ID Numbers: 1999-196
First Submitted: September 13, 2005
First Posted: September 20, 2005
Results First Submitted: February 22, 2013
Results First Posted: July 18, 2017
Last Update Posted: February 26, 2019