Why is the WINGS study being done?

The WINGS study is designed to obtain information on the safety and the efficacy of an experimental drug called QR-313. This drug is meant for patients who have confirmed Recessive Dystrophic Epidermolysis Bullosa (RDEB) with at least one mutation in exon 73 of the COL7A1 gene.

Who can take part in the WINGS study?*

The main eligibility criteria include:

  • 6 years of age and older
  • RDEB caused by a mutation(s) in exon 73 of the COL7A1 gene
  • At least one skin area (called a target wound area) that shows dynamic wound healing with an open wound that has healing potential
  • No suspicion of current squamous cell carcinoma (SCC)

* Other criteria may apply

What does participation in the study involve?

Throughout the study, you will maintain your normal Epidermolysis Bullosa (EB) wound care routine. Participation in this study is expected to take between 14 to 24 weeks. Once eligibility has been confirmed, the study can be broadly broken down into different stages:

  • Screening Period: Identification and observation of potential target wound areas for a minimum of 2 weeks and up to 8 weeks
  • Treatment Period: Selection of 2 target wound areas to receive treatment 2 or 3 times a week with study drug (QR-313 or matching placebo gel with no active drug) for 4 weeks per wound area
  • Follow up Period: Observation of each target wound area for 8 weeks after treatment.

Certain study visits will need to take place at the study site, but there is an option for some study visits at home by a member of the study staff (home nurse).

I don’t know the mutation that causes my RDEB, can I still take part in the WINGS study?

Potentially yes. After you agree to participate in the study (Informed Consent), there is a Screening Period where you will be assessed according to eligibility criteria and the required assessments will be done. This will include genetic testing to confirm that you have RDEB with at least one mutation in exon 73 of the COL7A1 gene. Your study doctor will inform you of the results and will answer any questions you may have.

Will it cost me anything to be in the study?

The study drug will be given to you for free. Everything related with this study (including genetic testing) and that are not a part of your normal EB wound care routine will be paid for you. Reasonable travel expenses and overnight accommodation will be covered for you and your accompanying person and you both will get a daily allowance for food and drinks.

More information

Please visit clinicaltrials.gov for more information about WINGS and talk to your EB doctor if you have any questions about the study.

QR-313 for RDEB

QR-313 is an investigational drug developed by Wings Therapeutics, meant for RDEB patients due to a mutation in a specific part of the COL7A1 gene called exon 73.

What is RDEB?

Epidermolysis bullosa (EB) is a group of rare genetic skin blistering diseases of which Recessive Dystrophic EB (RDEB) is one of the most severe forms.