Evidence map›Paper›PMID 32693833›Full record

Trial reportOrphanet journal of rare diseases2020

Wound closure in epidermolysis bullosa: data from the vehicle arm of the phase 3 ESSENCE Study.

Dedee F Murrell, Amy S Paller, Christine Bodemer, John Browning, Milos Nikolic, Jay A Barth, Hjalmar Lagast, Eva Krusinska, Allen Reha, ESSENCE Study Group

Registry-linked trialOpen access · goldAbstract readMulticenter StudyRandomized Controlled Trial
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Orphanet journal of rare diseases, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02384460 (A Phase 3, Multi-center, Randomized, Double-Blind, Placebo Controlled Study of the Efficacy and Safety of SD-101 Cream in Patients With Epidermolysis Bullosa), which is not on this map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed
1.0field-weighted citation impact, top 28% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT02384460 phase3completednot on this map

A Phase 3, Multi-center, Randomized, Double-Blind, Placebo Controlled Study of the Efficacy and Safety of SD-101 Cream in Patients With Epidermolysis Bullosa

TypeinterventionalSponsorScioderm, Inc.Ran2015 to 2017Enrolled169ConditionsEpidermolysis BullosaArmsSD-101-6.0 cream, Placebo (SD-101-0.0) cream
3 · Its place in the literature

Who cites it

3 citing papers in PubMed, 14 citations in OpenAlex.

  1. Article
  2. Review
  3. ABCB5Cytotherapy · 2023
    Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

10 authors at 5 institutions in 3 countries.

Dedee F MurrellUniversity of New South Wales, Sydney, NSW, Australia.
Amy S PallerDepartments of Dermatology and Pediatric Dermatology, Northwestern University Feinberg School of Medicine, 676 N. St. Clair, Suite 1600, Chicago, IL, 60611-2997, USA. apaller@northwestern.edu.
Christine BodemerEB Expert Centre (MAGEC), Department of Dermatology, Necker-Enfants Malades Hospital, Paris Centre University, Paris, France.
John BrowningTexas Dermatology & Laser Specialists, San Antonio, TX, USA.
Milos NikolicClinical Center of Serbia, Department of Dermatology, University of Belgrade, Belgrade, Serbia.
Jay A BarthAmicus Therapeutics, Inc., Cranbury, NJ, USA.
Hjalmar LagastAmicus Therapeutics, Inc., Cranbury, NJ, USA.
Eva KrusinskaAmicus Therapeutics, Inc., Cranbury, NJ, USA.
Allen RehaAmicus Therapeutics, Inc., Cranbury, NJ, USA.
ESSENCE Study Group
Amicus Therapeutics (United States) · USNorthwestern University · USTexas Dermatology and Laser Specialists · USUniversity of Belgrade · RSUNSW Sydney · AU

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

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 cm

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.

Indexed as

Epidermolysis BullosaDouble-Blind MethodHumansInfant, NewbornWound HealingEpidermolysis bullosaNatural historyWound closure

Identifiers

PMID32693833
PMCPMC7374851
OpenAlexW3044462916

What OpenQuestion holds

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Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.