RST-001 Phase I/II Trial for Advanced Retinitis Pigmentosa

• Sponsor: AbbVie (Industry)
• Overall Status: Active, not recruiting
• CT.gov ID: NCT02556736
• Collaborator: (none)
• Enrollment: 14
• Locations: 4
• Arm: 1
• Anticipated Duration (Months): 232.3
• Patients Per Site: 3.5
• Patients Per Site Per Month: 0

Study Details

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.

Condition or Disease	Intervention/Treatment	Phase
Advanced Retinitis Pigmentosa	Drug: RST-001	Phase 1/Phase 2

Study Design

• Study Type: Interventional
• Actual Enrollment: 14 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• 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)
• Actual Study Start Date: Dec 14, 2015
• Actual Primary Completion Date: Jun 1, 2020
• Anticipated Study Completion Date: Apr 24, 2035

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Group 1 Single intravitreal injection of RST-001	Drug: RST-001; RST-001 is a gene therapeutic delivered by intravitreal injection

Outcome Measures

Primary Outcome Measures

1. Number of Participants With Any Grade 3 or Greater Adverse Event (AE) Considered Related to RST-001. [Baseline (Day 1) to 6 Months]

   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

1. Change in Ambulation [Baseline to 24 months]

   Change in ambulation scores from Baseline

2. Visual Acuity [Baseline to 24 months]

3. Change in Quality of Life [Baseline to 24 months]

   Change in quality of life, based on the National Eye Institute (NEI) Visual Functioning Questionnaire (VFQ-25)

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	University of California, San Francisco- Dept. of Ophthalmology	San Francisco	California	United States	94143
2	Duke Eye Center	Durham	North Carolina	United States	27710
3	Cincinnati Eye Institute	Cincinnati	Ohio	United States	45242
4	Retina Foundation of the Southwest	Dallas	Texas	United States	75231

Sponsors and Collaborators

• AbbVie

Investigators

• Study Director: Hanh Badger, PharmD, Allergan

Study Documents (Full-Text)

• Study Protocol - Oct 19, 2017
• Statistical Analysis Plan - Feb 22, 2019

More Information

Additional Information:

• Additional information on study locations near you may be found at AllerganClinicalTrials.com. For any study not on AllerganClinicalTrials.com, please contact [e-mail] for assistance.

Publications

None provided.

• Responsible Party: AbbVie
• ClinicalTrials.gov Identifier: NCT02556736
• Other Study ID Numbers: RST-001-CP-0001
• First Posted: Sep 22, 2015
• Last Update Posted: Apr 11, 2022
• Last Verified: Apr 1, 2022

• Keywords provided by AbbVie: gene therapy; optogenetics; channelrhodopsin; retina; retinitis pigmentosa
• Additional relevant MeSH terms: Retinitis; Retinitis Pigmentosa

Study Results

Participant Flow

Arm/Group Title	RST-001
Arm/Group Description	Single intravitreal injection of RST-001
Period Title: Overall Study
STARTED	14
COMPLETED	14
NOT COMPLETED	0

Baseline Characteristics

Arm/Group Title	RST-001
Arm/Group Description	Single intravitreal injection of RST-001
Overall Participants	14
Age (Years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [Years]	59.1 (15.47)
Sex: Female, Male (Count of Participants)
Female	4 28.6%
Male	10 71.4%
Ethnicity (NIH/OMB) (Count of Participants)
Hispanic or Latino	0 0%
Not Hispanic or Latino	14 100%
Unknown or Not Reported	0 0%
Race (NIH/OMB) (Count of Participants)
American Indian or Alaska Native	0 0%
Asian	1 7.1%
Native Hawaiian or Other Pacific Islander	0 0%
Black or African American	2 14.3%
White	11 78.6%
More than one race	0 0%
Unknown or Not Reported	0 0%

Outcome Measures

1. Primary Outcome

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

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	RST-001
Arm/Group Description	Single intravitreal injection of RST-001
Measure Participants	14
Number [Participants]	0 0%

2. Secondary Outcome

Title	Change in Ambulation
Description	Change in ambulation scores from Baseline
Time Frame	Baseline to 24 months

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title

Arm/Group Description

3. Secondary Outcome

Title	Visual Acuity
Time Frame	Baseline to 24 months

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title

Arm/Group Description

4. Secondary Outcome

Title	Change in Quality of Life
Description	Change in quality of life, based on the National Eye Institute (NEI) Visual Functioning Questionnaire (VFQ-25)
Time Frame	Baseline to 24 months

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title

Arm/Group Description

Adverse Events

Time Frame	Adverse event data was collected from the screening visit (up to 45 days prior to study drug administration) up to 12? months after drug administration.
Adverse Event Reporting Description	Adverse event data presented hear includes all adverse events with an onset date on or after the date of drug administration.
Arm/Group Title	RST-001
Arm/Group Description	Single intravitreal injection of RST-001
All Cause Mortality
	RST-001
	Affected / at Risk (%)	# Events
Total	0/14 (0%)
Serious Adverse Events
	RST-001
	Affected / at Risk (%)	# Events
Total	0/14 (0%)
Other (Not Including Serious) Adverse Events
	RST-001
	Affected / at Risk (%)	# Events
Total	9/14 (64.3%)
Eye disorders
Anterior chamber cell	1/14 (7.1%)
Blepharitis	1/14 (7.1%)
Conjunctival haemorrhage	1/14 (7.1%)
Eye discharge	1/14 (7.1%)
Eye irritation	1/14 (7.1%)
Eye pain	1/14 (7.1%)
Iritis	1/14 (7.1%)
Retinal haemorrhage	1/14 (7.1%)
Vitreal cells	1/14 (7.1%)
Vitreous detachment	1/14 (7.1%)
Vitritis	1/14 (7.1%)
Gastrointestinal disorders
Intestinal obstruction	1/14 (7.1%)
General disorders
Swelling	1/14 (7.1%)
Infections and infestations
Ear infection	1/14 (7.1%)
Oral fungal infection	1/14 (7.1%)
Sialoadenitis	1/14 (7.1%)
Tooth infection	1/14 (7.1%)
Injury, poisoning and procedural complications
Fall	1/14 (7.1%)
Post procedural haemorrhage	1/14 (7.1%)
Post-traumatic pain	1/14 (7.1%)
Investigations
Intraocular pressure increased	3/14 (21.4%)
Metabolism and nutrition disorders
Hyperkalaemia	1/14 (7.1%)
Musculoskeletal and connective tissue disorders
Arthralgia	1/14 (7.1%)
Musculoskeletal chest pain	1/14 (7.1%)
Neck Pain	1/14 (7.1%)
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Benign neoplasm of eyelid	1/14 (7.1%)
Nervous system disorders
Cerebrospinal fluid leakage	1/14 (7.1%)
Optic neuritis	1/14 (7.1%)
Product Issues
Device dislocation	1/14 (7.1%)
Renal and urinary disorders
Pollakiuria	1/14 (7.1%)

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

Allergan as the sponsor, has proprietary interest in this study. Authorship and manuscript composition will reflect joint cooperation between multiple investigators and sites and Allergan personnel. Authorship will be established prior to the writing of the manuscript. As this study involves multiple centers, no individual publications will be allowed prior to completion of the final report of the multicenter study except as agreed with Allergan.