STAR (Clinical Trial)

Full Study Name

STAR - A Single-Masked, Randomised, Controlled, Parallel Group, Phase 3 Clinical Trial Of Retinal Gene Therapy For Choroideremia Using An Adeno-Associated Viral Vector (AAV2) Encoding Rab Escort Protein 1 (REP1)

Local Principal Investigator

Prof Paulo Stanga

Study Status


Main Aims

The objective of the study is to evaluate the efficacy and safety of a single sub-retinal injection of AAV2-REP1 in subjects with choroideremia (CHM).

Inclusion Criteria

• Are willing and able to give informed consent for participation in the study
• Are male and ≥18 years of age
• Have a genetically-confirmed diagnosis of CHM
• Have active disease clinically visible within the macular region in the study eye
• Have a BCVA of 34-73 ETDRS letters (equivalent to worse than or equal to 6/12 or 20/40 Snellen acuity, but better than or equal to 6/60 or 20/200 Snellen acuity) in the study eye.

Exclusion Criteria

• Have a history of amblyopia in the eligible eye(s)
• Are unwilling to use barrier contraception methods, if relevant
• Have any other significant ocular or non-ocular disease/disorder which, in the opinion of the investigator, may either put the subjects at risk because of participation in the study, or may influence the results of the study, or the subject’s ability to participate in the study. This would include having a contraindication to oral corticosteroid (eg prednisolone/prednisone), such as a history of gastric ulcer or significant side effects.
• Have participated in another research study involving an investigational product in the past 12 weeks or received a gene/cell-based therapy at any time previously.

Open Sites

MFT, Oxford University Hospital, EU and worldwide sites

For more information click here