Title

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)
  • Phase

    Phase 1/Phase 2
  • Study Type

    Interventional
  • Intervention/Treatment

    rst-001 ...
  • Study Participants

    14
Currently enrolling a total of 12 patients for Phase 2a of the study: 6 patients must have VA of no-better-than hand motion in the study eye, and 6 patients must have VA in the study eye to range from no-worse-than count fingers to 20/200 vision.
Study Started
Dec 14
2015
Primary Completion
Jun 01
2020
Study Completion
Sep 02
2024
Anticipated
Results Posted
Jun 23
2021
Last Update
Oct 06
2022

Drug RST-001

RST-001 is a gene therapeutic delivered by intravitreal injection

Group 1 Experimental

Single intravitreal injection of RST-001

Criteria

Inclusion criteria:

Participants must meet all of the following criteria.

Age >= 18 years
Signed and dated written informed consent obtained from the patient.
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:

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

Summary

RST-001

All Events

Event Type Organ System Event Term RST-001

Number of Participants With Any Grade 3 or Greater Adverse Event (AE) Considered Related to RST-001.

The U.S. Dept. of Health and Human Services' Common Terminology Criteria for Adverse Events grades AEs from 1 (Mild) to 5 (Death related to AE). Grade 3 is defined as "Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self care Activities of Daily Living (ADL)

RST-001

Age, Continuous

59.1
Years (Mean)
Standard Deviation: 15.47

Ethnicity (NIH/OMB)

Race (NIH/OMB)

Sex: Female, Male

Overall Study

RST-001