A Phase 1/2 Study in Healthy Volunteers and Participants With Autosomal Dominant Retinitis Pigmentosa (RHO-adRP)

A regiszterben nem jelölt feltételek

• Phase 1b/2 is active. Inclusion
• Phase 1b/2 (Multiple Ascending Dose Escalation in RHO-adRP Patients) Inclusion:
• Participants must have a diagnosis of Rhodopsin-associated, autosomal dominant Retinitis Pigmentosa confirmed by a genetic test that is implemented in accordance with the relevant national requirements
• Male or female greater than or equal to age 18 (inclusive) at the time of signing the informed consent form
• Have a body weight \>50kg, with a body mass index (BMI) from 18.0 to 35.0 kg/m\^2 (inclusive) at screening
• Best Corrected Visual Acuity (BCVA) using the Early Treatment Diabetic Retinopathy Study (ETDRS) \> 55
• Other than their diagnosis of RHO-adRP, be in good general health, as determined by the Investigator
• Have sufficient venous access to allow blood sampling for pharmacokinetic analysis and safety laboratory samples
  • Phase 1b/2 (Multiple Ascending Dose Escalation in RHO-adRP Patients) Exclusion:
• Females who are pregnant or breastfeeding
• Evidence of any other significant ocular (other than RHO-adRP) and non-ocular disease/ disorder
• Lifetime history of ocular surgery
• Any prior or current ophthalmologic gene therapy
• Has a history of major surgery, severe trauma, or bone fracture within 12 weeks before the first dose of the investigational product or planned surgery within 4 weeks after the end of the study