Clinical Trial test gene therapy treatment Haemophilia B

On 28th June 2018, the EPA received an application from UniQure Biopharma B.V., Paasheuvelweg 25A, 1105 BP Amsterdam, Netherlands, seeking consent to carry out a clinical trial in St James's Hospital in Dublin.

 

Objective of the field study

The objective of this proposed clinical trial is to test the safety and efficacy of a single administration of a gene therapy treatment in patients suffering from severe or moderately severe Haemophilia B. 

 

Location and Duration

It is proposed that this study will be carried out from 1 January 2019 - 31 December 2021 at St James's Hospital, James' Street, Dublin 8.

 

Legislation

Any person or organisation proposing to carry out a deliberate release of a genetically modified organism (GMO) for purposes other than placing on the market (performance of clinical trials) must comply with the legislative requirements set out under Part II of the GMO (Deliberate Release) Regulations S.I. No 500 of 2003.

 

Role of the EPA

In accordance with the Regulations, it is the role of the Agency to evaluate the risks posed by the proposed deliberate release for human health and the environment.

The Agency must respond to the applicant in writing within 90 days of receipt of the application.  This does not include any period during which the Agency is awaiting receipt of any further information on the notification, which it my have required from the applicant.

In its reponse, the Agency may indicate that consent to the proposed deliberate release is:-

  • granted with or without conditions; or,

  • refused, and the reasons for the refusal.

In reaching its decision, the Agency will take into consideration the views expressed by:

  • the GMO Advisory Committee, a statutory body set up to advise the Agency on any aspect of its functions in relation to GMOs; and,

 

Here below we provide access to the application documentation:

  • Summary Notification Information Format (SNIF);

  • Technical dossier on the proposed deliberate release / Environmental Risk Assessment.

 

Newspaper notice

The applicant published a notice of the proposed deliberate release, in the Dubin Gazette City Edition (5th July 2018) inviting members of the public to make representations to the EPA within 28 days of publication of the notice in a newspaper (i.e. up to 5pm on 2nd August 2018).

 

GMO Register Entry

The proposed clinical trial has been assigned GMO Register No G0667-01.  The Register provides significant details about the trial.  The register entry will be updated as further details become available.