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Efficacy and tolerability of the investigational topical cream SD-101 (6% allantoin) in patients with epidermolysis bullosa: a phase 3, randomized, double-blind, vehicle-controlled trial (ESSENCE study).

ABSTRACT: BACKGROUND:Epidermolysis bullosa (EB) is a rare genetic disorder that manifests as blistering and/or skin erosion. There is no approved treatment for EB; current standard of care consists of wound and pain management. SD-101 6% is a topical cream containing 6% allantoin that was developed for treating skin lesions in patients with EB. The aim of this phase 3, multicenter, randomized, double-blind, vehicle-controlled study was to assess the efficacy and safety of SD-101 6% cream versus vehicle (0% allantoin) on lesions in patients with EB. METHODS:Eligible patients were??1?month old, had a diagnosis of EB (simplex, recessive dystrophic, or intermediate junctional) and a target wound 10-50?cm2 in size that was present for ?21?days. Patients were randomly assigned to SD-101 6% cream or vehicle, which was applied topically once a day to the entire body for 3?months. Primary efficacy endpoints were time to complete target wound closure within 3?months and the proportion of patients who experienced complete target wound closure within 3?months. Post hoc subgroup analyses were conducted by patient age and in those with body surface area index of total body wound burden ?5% at baseline. RESULTS:In total, 169 patients were enrolled and randomly assigned to SD-101 6% (n?=?82) or vehicle (n?=?87). Baseline demographics and disease characteristics were similar between treatment groups. There were no statistically significant differences between treatment groups in time to target wound closure (hazard ratio, 1.004; 95% confidence interval [CI] 0.651, 1.549; P?=?0.985) or proportion of patients with complete target wound closure within 3?months (odds ratio [95% CI], 0.733 [0.365, 1.474]; nominal P?=?0.390). A positive trend toward faster wound closure with SD-101 6% versus vehicle was observed in patients aged 2 to <12?years and those with total body wound burden ?5% at baseline. SD-101 6% cream was well tolerated. CONCLUSIONS:SD-101 6% cream for treatment of EB-associated lesions was not more effective than vehicle in shortening the time to complete target wound closure or achieving complete target wound closure within 3?months. TRIAL REGISTRATION:ClinicalTrials.gov, NCT02384460; Date of trial registration, February 13, 2015; First participant enrolled, March 11, 2015.

SUBMITTER: Paller AS

PROVIDER: S-EPMC7310548 | biostudies-literature | 2020 Jun

REPOSITORIES: biostudies-literature

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Efficacy and tolerability of the investigational topical cream SD-101 (6% allantoin) in patients with epidermolysis bullosa: a phase 3, randomized, double-blind, vehicle-controlled trial (ESSENCE study).

Paller Amy S AS Browning John J Nikolic Milos M Bodemer Christine C Murrell Dedee F DF Lenon Willistine W Krusinska Eva E Reha Allen A Lagast Hjalmar H Barth Jay A JA

Orphanet journal of rare diseases 20200623 1

<h4>Background</h4>Epidermolysis bullosa (EB) is a rare genetic disorder that manifests as blistering and/or skin erosion. There is no approved treatment for EB; current standard of care consists of wound and pain management. SD-101 6% is a topical cream containing 6% allantoin that was developed for treating skin lesions in patients with EB. The aim of this phase 3, multicenter, randomized, double-blind, vehicle-controlled study was to assess the efficacy and safety of SD-101 6% cream versus ve ...[more]

PMID: 32576219

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Project description:BackgroundChronic wounds are a fundamental issue for patients with epidermolysis bullosa (EB). Herein, we assess the natural history of wound closure in patients with EB who were randomly assigned to the vehicle-control arm of the multicenter, randomized, double-blind, phase 3 ESSENCE (NCT02384460) trial.MethodsESSENCE was designed to assess the efficacy and safety of a topical cream formulation of 6% allantoin (SD-101 6%) vs vehicle (SD-101 0%) in patients ≥1 month old who had a diagnosis of EB (simplex, recessive dystrophic, or intermediate junctional) and a target wound 10-50 cm2 present for ≥21 days. Time to complete target wound closure and the proportion of patients with target wound closure over time were analyzed overall and by parameters including patient age and baseline body surface area index (BSAi) of total wound burden (< 5% and ≥ 5%). Changes in BSAi of lesional skin, pain, and itching were also assessed.ResultsThe vehicle-control arm included 87 patients. Mean (standard deviation [SD]) time to target wound closure within 3 months was 53.6 (28.6) days, with a range of 14 to 142 days. The proportion of patients with target wound closure increased over time from 7.1% at day 14 to 53.6% at month 3. Mean (SD) changes from baseline in BSAi of total wound burden and BSAi of lesional skin at month 3 were -2.3% (6.3) and -5.0% (13.5) of total body coverage, respectively. Reductions in pain and itching were observed at day 7 and maintained for 3 months. Faster healing times and a greater proportion of patients with wound closure were observed in patients aged 1 month to < 2 years; those with wounds < 30 days old, and in those with BSAi of total body wound burden < 5%.ConclusionsTreatment response observed in the vehicle-control arm of the ESSENCE study was unexpectedly high and may have been due to unforeseen benefits of vehicle or enhanced wound care provided by the clinical trial staff. These observations will help inform the study design of future trials in patients with EB.Trial registrationClinicalTrials.gov , NCT02384460 ; Date of registration: February 13, 2015; First participant enrollment: March 11, 2015.

Project description:Epidermolysis bullosa (EB) is characterized by skin fragility with blister formation occurring spontaneously or following minor trauma such as gentle pressure or friction. Current physiotherapy practice is based on anecdotal care, clinical expertise and creative problem solving with caregivers and individuals with EB. Evidence based intervention is needed to establish a foundation of knowledge and to guide international practitioners to create and improve standards of care to effectively work with individuals living with EB. This clinical practice guideline (CPG) was created for the purpose of providing evidence based interventions and best clinical practices for the physiotherapy management of individuals with EB. A survey was conducted within the EB community and six outcomes were identified as a priority to address in physiotherapy management, including (1) attaining developmental motor milestones, (2) identifying safe and functional mobility in the natural environment, (3) encouraging ambulation endurance, (4) supporting safe ability to bear weight, (5) improving access to physiotherapy services, and (6) optimizing interaction with the community. A systematic literature review was conducted and articles were critically analyzed by an international panel consisting of thirteen members: healthcare professionals (including physiotherapist, doctors, and occupational therapist), caregivers, and individuals with EB. Recommendations were formulated from evidence and panel consensus. An external panel of twelve were invited to improve the quality and gather feedback on draft manuscript and recommendations. This CPG describes the development of recommendations for physiotherapy management including several best practice interventions. This guideline lays the foundational work for physiotherapist throughout the world to provide high quality services while improving and maintaining functional mobility and independence within the EB community. The CPG outlines limitations in the evidence available and possible future research needed to improve physiotherapy practice.

Dataset Information

Efficacy and tolerability of the investigational topical cream SD-101 (6% allantoin) in patients with epidermolysis bullosa: a phase 3, randomized, double-blind, vehicle-controlled trial (ESSENCE study).

Publications

Efficacy and tolerability of the investigational topical cream SD-101 (6% allantoin) in patients with epidermolysis bullosa: a phase 3, randomized, double-blind, vehicle-controlled trial (ESSENCE study).

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