Clinical trial NCT00205374: Use of Cidofovir for Recurrent Respiratory Papillomatosis
Official title: Use of Cidofovir Injection in the Treatment of Recurrent Respiratory Papillomatosis."
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Recurrent Respiratory Papillomatosis
Lead sponsor: University of Wisconsin, Madison
Sponsor class: OTHER
First posted: 2005-09-20
Enrollment: 19
Sex: ALL
Minimum age (years): 5
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: Recurrent respiratory papillomatosis (RRP) is the most common benign neoplasm of the larynx in the pediatric population. The impact of the disease on patients and families can be tremendous due to the need for frequent treatment. It would be highly beneficial to develop effective medical therapies as adjunctive measures to surgical ablation with the goal of reducing the frequency of reoccurrence.

Detailed description: 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; PedsQL (Trademarked) 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 pressure?
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?

Eligibility criteria: Inclusion Criteria:

* 4 surgeries for RRP in last 12 months

Exclusion Criteria:

* Renal insufficiency
* Nephrotoxic drugs in the last 7 days
* Sulfa allergies
* Currently treated with systemic or topical HPV chemotherapeutic agents
* Females of childbearing potential with a positive pregnancy test
* Women who are breast feeding

Primary outcomes:
measure: Change in Papilloma Severity
timeFrame: Baseline, 2 months, and 12 months
Secondary outcomes:
measure: 12-month Change in Voice Handicap Index (VHI) Score
timeFrame: 2 months, and 12 months
Other outcomes:
Locations:
Study officials:
affiliation: University of Wisconsin Medical School
name: J. Scott McMurray, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: McMurray JS, Connor N, Ford CN. Cidofovir efficacy in recurrent respiratory papillomatosis: a randomized, double-blind, placebo-controlled study. Ann Otol Rhinol Laryngol. 2008 Jul;117(7):477-83. doi: 10.1177/000348940811700702.