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Serial Controlled N-of-1 Trials of Topical Vitamin E as Prophylaxis for Chemotherapy-Induced Oral Mucositis in Pediatric Patients

Phase 2/Phase 3
6 Years
18 Years
Not Enrolling

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Trial Information

Serial Controlled N-of-1 Trials of Topical Vitamin E as Prophylaxis for Chemotherapy-Induced Oral Mucositis in Pediatric Patients

Oral mucositis is a common consequence of chemotherapy and is an important sequela of cancer
therapy because it is painful and affects quality of life, may lead to hospitalization for
hydration or pain control, and provides a portal of entry for oral microflora. In addition,
oral mucositis has become a major dose-limiting toxicity and consequently, may limit
delivery of anti-cancer therapy.

Despite the frequency of mucositis, there are no feasible therapies proven to be successful
in preventing mucositis in children. Vitamin E is a fat-soluble essential vitamin that may
protect against doxorubicin-induced oral mucositis through its anti-oxidant properties.

In this study, we will examine the efficacy of topical vitamin E as prophylaxis against
chemotherapy-induced mucositis with a novel methodology appropriate for the study of rare
conditions, namely combining N-of-1 trials using Bayesian meta-analysis.

The primary outcome is an objective mucositis score measured on days 7, 10, 14 and 17.
Secondary outcomes included daily pain and swallowing visual analogue scale scores, and
World Health Organization mucositis scores collected on days 5 to 20.

Comparisons: Objective and subjective mucositis scores will be compared in cycles associated
with topical vitamin E versus cycles associated with placebo administration. We will use
repeated measures analysis within a Bayesian framework in order to conduct this comparison.

Inclusion Criteria:

- diagnosis of cancer and treated at the Hospital for Sick Children, Toronto, Canada

- planned chemotherapy includes at least two identical courses of
doxorubicin-containing chemotherapy in which the dose of doxorubicin was at least 60
mg/m2 per course

- at least 6 years of age and less than 18 years

- lives in the Greater Toronto area

Exclusion Criteria:

- allergy to vitamin E or placebo ingredients

- child is unable to comply with topical vitamin E application

- cild is receiving head or neck irradiation

Type of Study:


Study Design:

Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Crossover Assignment, Masking: Double-Blind, Primary Purpose: Prevention

Outcome Measure:

Objective mucositis score at 7, 10, 14, and 17 days post initiation of chemotherapy

Principal Investigator

Brian M Feldman, MD, MSc

Investigator Role:

Principal Investigator

Investigator Affiliation:

The Hospital for Sick Children


Canada: Ethics Review Committee

Study ID:




Start Date:

July 2002

Completion Date:

February 2005

Related Keywords:

  • Cancer
  • Vitamin E
  • Cancer
  • pediatrics
  • supportive care
  • oral mucositis
  • Bayesian analysis
  • N-of-1 trial
  • prophylaxis
  • Stomatitis
  • Mucositis