Back to resultsRST-001 Phase I/II Trial for Advanced Retinitis Pigmentosa
Primary Purpose
Advanced Retinitis Pigmentosa
Status
Active
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
RST-001
Sponsored by
About this trial
This is an interventional treatment trial for Advanced Retinitis Pigmentosa focused on measuring gene therapy, optogenetics, channelrhodopsin, retina, retinitis pigmentosa
Eligibility 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
Sites / Locations
- University of California, San Francisco- Dept. of Ophthalmology
- Duke Eye Center
- Cincinnati Eye Institute
- Retina Foundation of the Southwest
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Group 1
Arm Description
Single intravitreal injection of RST-001
Outcomes
Primary Outcome Measures
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)
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT02556736
Brief Title
RST-001 Phase I/II Trial for Advanced Retinitis Pigmentosa
Official Title
Phase I/IIa, Open-Label, Dose-Escalation Study of Safety and Tolerability of Intravitreal RST-001 in Patients With Advanced Retinitis Pigmentosa (RP)
Study Type
Interventional
2. Study Status
Record Verification Date
September 2022
Overall Recruitment Status
Active, not recruiting
Study Start Date
December 14, 2015 (Actual)
Primary Completion Date
June 1, 2020 (Actual)
Study Completion Date
September 2, 2024 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
AbbVie
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Advanced Retinitis Pigmentosa
Keywords
gene therapy, optogenetics, channelrhodopsin, retina, retinitis pigmentosa
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
14 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Group 1
Arm Type
Experimental
Arm Description
Single intravitreal injection of RST-001
Intervention Type
Drug
Intervention Name(s)
RST-001
Intervention Description
RST-001 is a gene therapeutic delivered by intravitreal injection
Primary Outcome Measure Information:
Title
Number of Participants With Any Grade 3 or Greater Adverse Event (AE) Considered Related to RST-001.
Description
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)
Time Frame
Baseline (Day 1) to 6 Months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Hanh Badger, PharmD
Organizational Affiliation
Allergan
Official's Role
Study Director
Facility Information:
Facility Name
University of California, San Francisco- Dept. of Ophthalmology
City
San Francisco
State/Province
California
ZIP/Postal Code
94143
Country
United States
Facility Name
Duke Eye Center
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
Cincinnati Eye Institute
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45242
Country
United States
Facility Name
Retina Foundation of the Southwest
City
Dallas
State/Province
Texas
ZIP/Postal Code
75231
Country
United States
12. IPD Sharing Statement
Citations:
PubMed Identifier
30218651
Citation
Simunovic MP, Shen W, Lin JY, Protti DA, Lisowski L, Gillies MC. Optogenetic approaches to vision restoration. Exp Eye Res. 2019 Jan;178:15-26. doi: 10.1016/j.exer.2018.09.003. Epub 2018 Sep 13.
Results Reference
derived
Links:
URL
http://allerganclinicaltrials.com
Description
Additional information on study locations near you may be found at AllerganClinicalTrials.com. For any study not on AllerganClinicalTrials.com, please contact IR-CTRegistration@Allergan.com for assistance.
Learn more about this trial
RST-001 Phase I/II Trial for Advanced Retinitis Pigmentosa
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