Pilot randomised double blinded comparative trial to evaluate the efficacy of topical tacrolimus versus placebo in the management of itch in patients with recessive dystrophic epidermolysis bullosa
Trial Snapshot
- Phase
- Not Applicable
- Status
- Not yet recruiting
- Sponsor
- Enrollment
- 20
- Locations
- 1
- Primary Endpoint
- To evaluate the improvement of pruritus as determined by numeric rating scale and Leuven score for pruritus in patients with recessive dystrophic epidermolysis bullosa with the use of topical tacrolimus 0.03% for 3 months as compared to placebo.
Study Overview
Brief Summary
Epidermolysis bullosa (EB) is a group of inherited skin disorders caused by mutations in genes that affect the dermal-epidermal junction, leading to fragile skin, blistering, scarring, and contractures. Itching (pruritus) is a particularly disabling symptom, especially in children with recessive dystrophic EB (RDEB), worsening the condition by causing more blisters and erosions. This study aims to evaluate the effectiveness of topical tacrolimus in managing pruritus in RDEB patients, with the goal of improving patient outcomes and quality of life.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Masking
- Participant and Outcome Assessor Blinded
Eligibility Criteria
- Ages
- 6.00 Year(s) to 60.00 Year(s) (—)
- Sex
- All
Inclusion Criteria
- •Patients with a clinical diagnosis of recessive dystrophic epidermolysis bullosa, aged over six years, and experiencing moderate to severe pruritus with a Numeric Rating Scale score greater than 3.
Exclusion Criteria
- •Patients of recessive dystrophic epidermolysis bullosa not complaining of pruritus.
- •Not willing to participate in study.
Outcomes
Primary Outcomes
To evaluate the improvement of pruritus as determined by numeric rating scale and Leuven score for pruritus in patients with recessive dystrophic epidermolysis bullosa with the use of topical tacrolimus 0.03% for 3 months as compared to placebo.
Time Frame: Baseline, 4 weeks, 8 weeks and 12 weeks
To assess the improvement in disease severity and quality of life as determined by EBDASI and EBQOL in patients with recessive dystrophic epidermolysis bullosa with the use of topical tacrolimus 0.03% for 3 months as compared to placebo
Time Frame: Baseline, 4 weeks, 8 weeks and 12 weeks
Secondary Outcomes
- Determination of severity of neuropathy as determined by nerve conduction and quantitative sensory testing with its correlation with severity of pruritus in patients with recessive dystrophic epidermolysis bullosa.(Quantitative assessment of pruritus related cytokines including IL-31, IL-33, IL-22, IL-4, IL-13, IL-17 and TSLP at baseline and at 3 months in patients with recessive dystrophic epidermolysis bullosa)
Investigators
Dr. Neetu Bhari
AIIMS New Delhi