A Phase II, Randomised, Controlled, Double Masked, Multiple Dose Study of the Safety, Tolerability and Efficacy of Intravitreal KIO-301 in Patients With Late-stage Retinitis Pigmentosa
The goal of the study is to investigate the safety, tolerability and efficacy of up to 3 doses of KIO-301 administered by intravitreal (IVT) injection bilaterally every 6 weeks in patients with late-stage retinitis pigmentosa (RP). Late-stage RP patients will include those patients with No Light Perception (NLP), or Low Vision (LV).
• Be aged 18 years or older at the time of consent.
• Provide informed consent prior to any study procedures, as stipulated by local laws, Ethics Committee (EC) and Regulatory Authority (RA) guidelines.
• Be willing and able to follow all study instructions, attend all study visits, and complete all study assessments.
• Have a clinical diagnosis of non-syndromic RP, with the exception of Usher's Syndrome Type II (USH2) which is allowed.
• Have a visual acuity as per the Berkeley Rudimentary Vision Test (BRVT) at Screening of:
‣ NLP OU confirmed by inability to see pen torch light at 25 cm OD, OS, and OU (assigned logMAR of 4.0).
⁃ LV OU limited to logMAR \> 1.6 and \< 4.0.
• Other than intravitreal corticosteroids, participants must not receive intravitreal concomitant medications from Screening until end of study.
• For Low Vision (LV) OU participants only: must pass at least one multi-luminance functional vision (MLFV) test at two successive light levels (between 1 and 500 lux), or at 1400 lux. Additionally, they must fail the same test at 0.125 and 0.35 lux.
• Must agree to follow appropriate contraception requirements from Screening until 3 months after the last dose of IMP.
‣ Participants assigned female at birth who are of child-bearing potential (OCBP) must agree to a pregnancy test at Screening and use an acceptable method of birth control including oral, transdermal, injectable, or implantable hormonal contraception, intrauterine device, abstinence from intercourse with partner assigned male at birth, or surgical sterilisation of partner assigned male at birth. Participants assigned female at birth are not OCBP if they have had a hysterectomy, bilateral oophorectomy, bilateral tubal ligation, or are post-menopausal by at least 12 months.
⁃ Participants assigned male at birth with a partner OCBP must be surgically sterile for at least 3 months prior to starting study drug, or ensure their partner uses contraception as outlined above, and must use a male condom. Participants assigned male at birth must not donate sperm from Screening until 3 months after the last dose of IMP.
⁃ Participants who have practiced true abstinence for at least 1 year due to usual and preferred lifestyle choice are exempt from contraceptive requirements. If a participant who is abstinent becomes sexually active, they must agree to use appropriate contraception as described above.