AGN-151597 (Formerly RST-001) Phase I/II Trial for Advanced Retinitis Pigmentosa

Phase I/IIa, Open-Label, Dose-Escalation Study of Safety and Tolerability of Intravitreal RST-001 in Patients With Advanced Retinitis Pigmentosa (RP)

All participants in phase 1 and phase 2a had hand motion visual acuity or worse.

If efficacy was demonstrated from phase 1, better vision subjects could be enrolled; however, efficacy was not demonstrated.

Study RST-001-CP-0001 was an open-label, dose-escalation study to evaluate the safety and tolerability of AGN-151597 (formerly RST-001) administered as a single intravitreal injection in participants with advanced RP. Three groups of approximately 3 participants each were sequentially enrolled in the dose-escalation phase (Phase 1) of this study: Group A (low dose), Group B (mid dose), and Group C (high dose).

For each dose group, the safety and tolerability of AGN-151597 was assessed by a data safety monitoring committee (DSMC) in the first participant before the remaining participants were enrolled into the group. If the DSMC considered the safety and tolerability of all participants in the dose group to be satisfactory and enrollment stopping rules had not been met, then enrollment into the next dose group could begin.

If the DSMC considered the safety and tolerability satisfactory and the enrollment stopping rules had not been met after a minimum assessment of 1 month (to include the Month 1 Visit) from treatment of the final participant in Groups A, B, or C, then the sponsor could elect to start enrollment of up to approximately 12 participants in Phase 2a to receive AGN-151597 at the maximum tolerated dose. After completion of the 2-year core study visits, each participant could enroll in a long-term follow-up for an additional 3 years to monitor the long-term safety of AGN-151597.

Inclusion Criteria:

Participants must meet all of the following criteria.

1. Age >= 18 years.
2. Signed and dated written informed consent obtained from the patient.
3. Ability to comply with testing and all protocol tests.

Exclusion Criteria:

Any one of the following will exclude patients from being enrolled into the study:

1. Unable or unwilling to meet requirements of the study.
2. Participation in a clinical study (ocular or non-ocular) with an investigational drug, agent or therapy in the past six months.