Use of Cidofovir for Recurrent Respiratory Papillomatosis

University of Wisconsin (UW)

Status and phase: Completed; Phase 4

Conditions: Recurrent Respiratory Papillomatosis

Treatments: Drug: Cidofovir; Drug: Placebo

Study type: Interventional

Funder types: Other

Identifiers

NCT00205374

1999-196

Details and patient eligibility

About

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.

Full 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?

Enrollment: 19 patients

Sex: All

Ages: 5+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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19 participants in 2 patient groups, including a placebo group

Cidofovir

Active Comparator group

Description:

Cidofovir (Vistide) is a commercially available agent approved by the FDA for the treatment of cytomegalovirus (CMV) retinitis in patients with acquired immunodeficiency syndrome (AIDS). The drug is not FDA approved for the treatment of RRP at this time. However, recent case reports have been encouraging with regard to the effectiveness of the agent in the treatment of RRP. The FDA has granted this study a "safe to proceed" designation with IND 58,481.

Treatment:

Drug: Cidofovir

Placebo

Placebo Comparator 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.

Treatment:

Drug: Placebo