Use of Cidofovir Injection in the Treatment of Recurrent Respiratory Papillomatosis."
The focus of the present study is to evaluate the usefulness of cidofovir injection in
diminishing the frequency and magnitude of papilloma recurrences in adult and pediatric RRP
patients. Briefly, patients will be randomized into either a treatment (cidofovir injection)
or a placebo group. The following measures will be made at each of 6 data collection time
points, over the course of one year: (1) tumor load, based upon a published staging system
for papilloma, (2) degree of respiratory obstruction for phonation, as assessed by phonation
threshold pressures, and (3) general health, on validated health inventories (SF12 and Voice
Handicap Index for adults; Peds QL for children) and via measures of height weight and days
absent from school or daycare, where applicable, for children. A repeated measures analysis
will allow examination of time by treatment interactions to determine if the cidofovir
injection group has fewer, or less severe, recurrences than the placebo group.
Specifically, we will answer the following questions in this investigation:
1. Does cidofovir injection reduce the frequency of RRP recurrences?
2. Does cidofovir injection reduce the magnitude of RRP, as assessed with a proposed
staging system for RRP (Derkay et al., 1998) and measures of phonatory threshold
3. Does cidofovir injection improve general health, as assessed by height, weight and
days absent from school in pediatric patients and health inventories (general health
and voice-related) in children and adults?
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Treatment
Reduction in papilloma severity
Treatment of RRP with cidofovir injection will be considered efficacious if drug group patients experience clinically or statistically significant reductions in papilloma severity and inter-surgery time intervals (relative to pre-treatment assessments), when compared with placebo group patients.
J. Scott McMurray, MD
University of Wisconsin Medical School
United States: Food and Drug Administration