Use of Cidofovir for Recurrent Respiratory Papillomatosis

NCT00205374 | Completed Phase 4 Results DMC

• 1 other identifier: 1999-196
• Study Type: interventional
• Target: 19
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Recurrent Respiratory Papillomatosis

Outcome Measures

Primary Outcomes (1)

• Change in Papilloma Severity

  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.

  Baseline, 2 months, and 12 months

Secondary Outcomes (1)

• 12-month Change in Voice Handicap Index (VHI) Score

  2 months, and 12 months

Study Arms (2)

Cidofovir

ACTIVE COMPARATOR

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.

Drug: Cidofovir

Placebo

PLACEBO COMPARATOR

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.

Drug: Placebo

Interventions

Cidofovir DRUG

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.

Also known as: Vistide

Cidofovir

Placebo DRUG

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.

Also known as: Sugar pill

Placebo

Eligibility Criteria

• Age: 5 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• surgeries for RRP in last 12 months

You may not qualify if:

• 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

Sponsors & Collaborators

• University of Wisconsin, Madison: lead

Related Publications (1)

• 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.

  PMID: 18700421 RESULT

MeSH Terms

Conditions

Recurrent respiratory papillomatosis

Interventions

Cidofovir; Sugars

Intervention Hierarchy (Ancestors)

Organophosphonates; Organophosphorus Compounds; Organic Chemicals; Cytosine; Pyrimidinones; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Carbohydrates

Results Point of Contact

• Title: J. Scott McMurray, MD
• Organization: University of Wisconsin-Madison

mcmurray@surgery.wisc.edu

608-262-7181

Study Officials

• J. Scott McMurray, MD

  University of Wisconsin Medical School

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 13, 2005
• First Posted: September 20, 2005
• Study Start: November 1, 1999
• Primary Completion: December 1, 2009
• Study Completion: December 1, 2009
• Last Updated: February 26, 2019
• Results First Posted: July 18, 2017

Record last verified: 2019-02

Data Sharing

• IPD Sharing: Will not share