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Article History

Received: 22 March 2018

Accepted: 9 November 2018

First Online: 26 November 2018

Ethics approval and consent to participate

: The study was approved on 19 October 2015 by the UK’s Medicines & Healthcare Products Regulatory Agency (Reference 45,268/0001/001–0001) in compliance with the European Clinical Trials Directive and the Medicines for Human Use (Clinical Trials) Regulations 2004 and its subsequent amendments. The study was provided with ethical approval by the NHS Health Research Authority and local NHS Research Ethics Committee (Integrated Research Application System Project Code 186163, REC Reference 15/EE/0334). The study is registered on(Identifier; NCT02739217 -) and European Union Drug Regulating Authorities Clinical Trials (EudraCT Number 2015–001625-16).Subjects are only included in this study if informed consent is obtained in advance of any study-specific procedures and documented in an IEC-approved information sheet and consent form. Given the nature and severity of Alström Syndrome and the resultant loss of sight, subjects are not expected to be able to read a normal information sheet and consent form. Accordingly, provision are made to provide the contents of the information sheet consent form in a format that is suitable for the individual (e.g., in large font, in braille, or verbally or in an audio format) and for consent to be witnessed.

: By providing informed consent, participants agree to have study results published in form of scientific papers. Data is anonymised before analysis and publication.

: JM is an employee of Prometic, the developer of PBI-4050. The other authors declare no competing interest.

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