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CIL 34546-NSR-REP-01

Gene therapy study on choroideremia : to assess the safety and tolerability of the AAV.REP1 vector, administered at two different doses to the retina in patients with a diagnosis of choroideremia.

Type: Interventional

Status of the trial: Completed

Orphan Drug Recognition: Yes


  • Opening Date: 2017-06-01
  • Closing Date: 2018-08-31


Inclusion: Visual Acuity between 20/200 and 20/40

Exclusion: age < 18 years

More information

Children: No

Adults: Yes

Funder Type: industry

Within ERN-EYE members

Principal investigators

Other investigators


  • Retinal Rare Eye Diseases (WG1)

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This ERN is supported by: