Clinical Trial Summary

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Phase

Phase 1/Phase 2

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Funding Agency/Sponsor

Industry

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Disease

Other

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Enrollment Eligibility

Inclusion Criteria:
- Male or female
- At least 3 years of age at screening with CEP290-related retinal degeneration caused
by a homozygous or compound heterozygous mutation involving c.2991+1655A>G in IVS26 of
the CEP290 gene.
- Visual Acuity:
- Sentinel participant will have severe vision loss with a logMAR BCVA of ≥1.6 to
3.9 (20/800 or worse to LP) in the study eye
- Non-sentinel participants must have BCVA between 1.0 - 3.0 logMAR in the study
eye
Exclusion Criteria:
- Other known disease-causing mutations
- Achieves a passing score for the mobility course at the most difficult level
- In either eye, active systemic or ocular/intraocular infection or inflammation
- In either eye, history of steroid-responsive intraocular pressure with increases > 25
mm Hg following corticosteroid exposure
- Any vaccination/immunization in the last 28 days before screening
- Inability or unwillingness to take oral prednisone
- Prior gene therapy or oligonucleotide treatment

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