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Ongoing clinical trials

Below you will find a list of EB clinical trials being conducted around the world.

Each official clinical trial website will include details on who is conducting it, what its purpose is, how the outcomes will be measured, and who is eligible to partake. Don’t be put off by any technical terms. There are glossaries out there to help you through, including the excellent Clinical Trials Glossary from EURORDIS.

If you are interested in taking part in a clinical trial, it is advisable to discuss the possibility with your doctor first. Many of the trials listed provide contact details for someone who can give you more information.

If you are an investigator conducting a clinical trial please contact us to make sure we have up-to-date and accurate information on your trial.

 

DELIVERS Study: Safety and Efficacy of Diacerein 1% Ointment Topical Formulation Compared to Placebo for Subjects With Epidermolysis Bullosa Simplex (EBS)
WhatThe purpose of this study is to compare the efficacy of Diacerein 1% Ointment to Control Ointment when applied once-daily for 8 weeks in subjects with EBS. It is a potentially disease-modifying therapy that may block an important inflammatory signaling pathway associated with EBS, which could strengthen epidermal tissue and support healing. Previously, oral forms of diacerein has been studied for the treatment of joint diseases such as osteoarthritis and other conditions including eczema, rash, and pruritus.
WhereVarious locations in the USA, and Europe (including Austria, The Netherlands, Germany, France, and the UK) as well as in Israel, and Australia.
WhenThis study is currently at the stage of recruiting participants.
WhoPeople with EBS, 4 years and over, who fit the criteria
See full details here:
ClinicalTrials.gov
Deliversebs.com
Last updated 18.10.2017
WINGS Study: QR-313 for Recessive Dystrophic Epidermolysis Bullosa (RDEB) due to mutation(s) in exon 73 of the COL7A1 gene
WhatProQR Therapeutics is currently recruiting for WINGS, a Phase 1b/2 research study of QR-313 in patients aged 6 years and older with Recessive Dystrophic Epidermolysis Bullosa (RDEB) due to mutation(s) in a specific part of the COL7A1 gene called exon 73. QR-313 gel will be applied directly onto wounded skin. QR-313 is an experimental drug designed to exclude exon 73 (exon skipping) from the COL7A1 mRNA (the ‘blueprint’ for making proteins) with the aim to restore functionality of collagen type VII protein. This in turn may potentially lead to the formation of anchoring fibrils that bind the dermal (inner) and epidermal (outer) skin layers together, improved wound healing and skin strength.The main objectives of WINGS are to evaluate safety after administration of QR-313 to wounded skin and to evaluate whether QR-313 is able to exclude (skip) exon 73 from the COL7A1 mRNA. The effects of QR-313 on wound healing, on skin strength and on the presence of collagen type VII protein and anchoring fibrils following treatment will also be assessed. During the study, QR-313 or a matching placebo gel, will be applied to wounded skin two to three times a week (at the time of changing of wound dressings) for up to four weeks with an eight week follow up period.
WhereVarious locations in the USA and Europe.
WhenThis study is currently recruiting participants.
WhoPeople with RDEB, aged 6 years and older and meet all enrollment criteria
See full details here:
ClinicalTrials.gov
Last updated 17.09.2018
WINGS Study: QR-313 for Recessive Dystrophic Epidermolysis Bullosa (RDEB) due to mutation(s) in exon 73 of the COL7A1 gene
WhatProQR Therapeutics is currently recruiting for WINGS, a Phase 1b/2 research study of QR-313 in patients aged 6 years and older with Recessive Dystrophic Epidermolysis Bullosa (RDEB) due to mutation(s) in a specific part of the COL7A1 gene called exon 73. QR-313 gel will be applied directly onto wounded skin. QR-313 is an experimental drug designed to exclude exon 73 (exon skipping) from the COL7A1 mRNA (the ‘blueprint’ for making proteins) with the aim to restore functionality of collagen type VII protein. This in turn may potentially lead to the formation of anchoring fibrils that bind the dermal (inner) and epidermal (outer) skin layers together, improved wound healing and skin strength.The main objectives of WINGS are to evaluate safety after administration of QR-313 to wounded skin and to evaluate whether QR-313 is able to exclude (skip) exon 73 from the COL7A1 mRNA. The effects of QR-313 on wound healing, on skin strength and on the presence of collagen type VII protein and anchoring fibrils following treatment will also be assessed. During the study, QR-313 or a matching placebo gel, will be applied to wounded skin two to three times a week (at the time of changing of wound dressings) for up to four weeks with an eight week follow up period.
WhereVarious locations in the USA and Europe.
WhenThis study is currently recruiting participants.
WhoPeople with RDEB, aged 6 years and older and meet all enrollment criteria
See full details here:
ClinicalTrials.gov
Last updated 17.09.2018

 

Amryt Pharma EASE Study: Double blind, Randomised, Vehicle controlled, Phase III, Efficacy and Safety Study with 24-month Open-label Follow-up of Oleogel S10 in Patients with Inherited Epidermolysis Bullosa
WhatThe purpose of this study is to compare the efficacy, safety and tolerability of Oleogel-S10 versus placebo in the promotion of healing of Epidermolysis Bullosa partial thickness wounds. At the end of the 90 day double-blind period patients in both treatment arms will enter an open-label follow-up phase for 24 months.
Note: the treatment has already been approved in Europe for use in the treatment of partial thickness wounds (PTW) in adults under the brand name Episalvan.
WhereArgentina, Australia, Austria, Belgium, Brazil, Chile, Colombia, Croatia, Czech Rep., Denmark, France, Germany, Greece, Hong Kong, Hungary, Ireland, Italy, Romania, Russia, Serbia, Singapore, Spain, Switzerland, Ukraine, UK, USA
WhenRecruitment ongoing
WhoPeople with DEB or JEB, 4 years and over, who meet the eligibility criteria
See full details here
Last updated 10.09.2018
Gene Transfer for RDEB
WhatThis is a trial of an ex-vivo (out of the body) gene transfer. Specifically, the study aims to take some of the patients own skin cells, correct the faulty collagen 7 gene in the lab, grow the cells into 'sheets' and then graft them back to the patient's skin.
This type of approach is a combination of a cell therapy and a gene therapy (in this case the investigators refer to it as a gene 'transfer' as they do not want to call it a therapy until it is proven effective).
WhereStanford University, California, US
WhenThis trial is currently at the stage of recruiting participants (02/2015)
WhoPeople over 18 with RDEB, who fit the criteria
See full details here
Stem Cell Transplant for EB
WhatThis trial aims to test the effectiveness of treating severe EB, with stem cells found in bone marrow or umbilical cord blood (hematopoietic stem cells). Participants undergo a full bone marrow transplant. This trial represents a slightly modified approach to a previous trial undertaken by this group. In this trial participants are also treated with a type of stem cells called mesenchymal stem cells.
WhereMasonic Cancer Center, University of Minnesota, US
WhenThis trial is currently at the stage of recruiting participants (04/2015)
WhoPeople with a severe form of EB, up to 25 years of age, who fit the criteria
See full details here

Disclaimer

In so far as possible, we have tried to ensure that the details provided are accurate. However, to a large extent we depend on investigators making up-to-date information available through the relevant websites or through contact with us. If you are interested in a clinical trial, please confirm details with the relevant investigators.

While we have been careful to only include clinical trials in this list that have been approved by appropriate authorities, it is unfortunately not possible for us to take the step of endorsing particular clinical trials. Please consider this list simply an information resource on current and recent EB clinical trials.