The Efficacy of Trimethoprim in Wound Healing of Patients With Epidermolysis Bullosa

Condition(s) targeted: Epidermolysis Bullosa

Intervention: Trimethoprim (Drug); Trimethoprim (Drug)

Phase: Phase 2

Status: Completed

Sponsored by: The Hospital for Sick Children

Official(s) and/or principal investigator(s):
Elena Pope, MD, Principal Investigator, Affiliation: The Hospital for Sick Children, Toronto Canada

The purpose of this study is to assess the efficacy of trimethoprim in promoting wound healing and decreasing blister formation in patients with Epidermolysis Bullosa.

Official title: The Efficacy of Trimethoprim in Wound Healing of Patients With Epidermolysis Bullosa: A Randomized, Double Blinded, Placebo Controlled, Cross-over Pilot Study

Study design: Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Crossover Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Treatment

Primary outcome: Percentage change of area of the wound from visit to visit, estimated from the longest length and width of up to three target chronic wounds

Secondary outcome:

Total number of blisters at each visit

Total number of opened areas at each visit

Qualitative wound score

Parent/patient/physician perception of improvement, assessed with a visual analog scale at each visit

Quality of life, assessed by the Children's Dermatology Life Quality Index (CDLQI) and the Cardiff Wound Impact Schedule

Number of infections that require systemic antibiotics

Detailed description: Epidermolysis Bullosa (EB) comprises a series of hereditary disorders characterized by fragility of the skin and mucous membranes and the tendency of the skin to blister in response to minor friction or trauma. The care of patients with EB is a complex task that has to be carried out by a multi-professional team. In the absence of a cure, the goal of therapy is the prevention and healing of chronic wounds. In patients with EB, chronic inflammation interferes with proper wound healing. One treatment option is the use of anti-inflammatory antimicrobial agents, such as trimethoprim, to hasten wound healing and decrease blister formation. This treatment may lead to decreased pain and improvement of the quality of life for these patients.

Minimum age: N/A. Maximum age: 20 Years. Gender(s): Both.

Criteria:

Inclusion Criteria:

- Patients younger than 20 years of age

- Diagnosis of Recessive Dystrophic Epidermis Bullosa (RDEB)or Junctional Epidermis

Bullosa (JEB)

- Signed consent/assent form

Exclusion Criteria:

- Previous known allergy or intolerance to trimethoprim

The Hospital for Sick Children, Toronto, Ontario M5G 1X8, Canada

Starting date: September 2006
Last updated: August 1, 2013