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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02556736
Recruitment Status : Active, not recruiting
First Posted : September 22, 2015
Results First Posted : June 23, 2021
Last Update Posted : October 6, 2022
Sponsor:
Information provided by (Responsible Party):
AbbVie

Study Description
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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
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Study Type : Interventional  (Clinical Trial)
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 : December 14, 2015
Actual Primary Completion Date : June 1, 2020
Estimated Study Completion Date : September 2, 2024

Resource links provided by the National Library of Medicine


Arms and Interventions
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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
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Primary Outcome Measures :
  1. Number of Participants With Any Grade 3 or Greater Adverse Event (AE) Considered Related to RST-001. [ Time Frame: 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)



Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02556736


Locations
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United States, California
University of California, San Francisco- Dept. of Ophthalmology
San Francisco, California, United States, 94143
United States, North Carolina
Duke Eye Center
Durham, North Carolina, United States, 27710
United States, Ohio
Cincinnati Eye Institute
Cincinnati, Ohio, United States, 45242
United States, Texas
Retina Foundation of the Southwest
Dallas, Texas, United States, 75231
Sponsors and Collaborators
AbbVie
Investigators
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Study Director: Hanh Badger, PharmD Allergan
  Study Documents (Full-Text)

Documents provided by AbbVie:
Study Protocol  [PDF] October 19, 2017
Statistical Analysis Plan  [PDF] February 22, 2019

More Information
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Additional Information:

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: AbbVie
ClinicalTrials.gov Identifier: NCT02556736    
Other Study ID Numbers: RST-001-CP-0001
First Posted: September 22, 2015    Key Record Dates
Results First Posted: June 23, 2021
Last Update Posted: October 6, 2022
Last Verified: September 2022
Keywords provided by AbbVie:
gene therapy
optogenetics
channelrhodopsin
retina
retinitis pigmentosa
Additional relevant MeSH terms:
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Retinitis
Retinitis Pigmentosa
Retinal Diseases
Eye Diseases
 Eye Diseases, Hereditary
Retinal Dystrophies
Retinal Degeneration
Genetic Diseases, Inborn