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

This study is currently recruiting participants.
See Contacts and Locations
Verified July 2017 by Allergan
Sponsor:
Information provided by (Responsible Party):
Allergan
ClinicalTrials.gov Identifier:
NCT02556736
First received: September 21, 2015
Last updated: July 7, 2017
Last verified: July 2017
  Purpose
RST-001 is a gene therapy given as an injection into the eye.

Condition Intervention Phase
Advanced Retinitis Pigmentosa Drug: RST-001 Phase 1 Phase 2

Study Type: Interventional
Study Design: 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)

Resource links provided by NLM:


Further study details as provided by Allergan:

Primary Outcome Measures:
  • Safety [ Time Frame: 6 month from baseline ]
    Absence of any grade 3 or greater AE considered related to RST-001.


Secondary Outcome Measures:
  • To establish the maximum tolerated dose of RST-001 [ Time Frame: Up to 24 months from the baseline ]
    Determined by a safety review

  • To evaluate the preliminary efficacy of RST-001 in patients with advanced RP [ Time Frame: Up to 24 months from the baseline ]
    Preliminary efficacy data will be obtained through ocular evaluations (e.g., number of participants with changes in visual function)


Estimated Enrollment: 15
Actual Study Start Date: December 14, 2015
Estimated Study Completion Date: February 28, 2021
Estimated Primary Completion Date: August 28, 2019 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Group 1
Single intravitreal injection of RST-001
Drug: RST-001
RST-001 is a gene therapeutic delivered by intravitreal injection

  Eligibility

Ages Eligible for Study:   18 Years and older   (Adult, Senior)
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02556736

Contacts
Contact: Clinical Trials Registry Team 877-277-8566 IR-CTRegistration@Allergan.com

Locations
United States, Texas
Retina Foundation of the Southwest Recruiting
Dallas, Texas, United States, 75231
Contact: David Birch, PhD    214-363-3911 ext 105      
Sponsors and Collaborators
Allergan
Investigators
Study Director: Werner Schmidt Allergan
  More Information

Additional Information:
Responsible Party: Allergan
ClinicalTrials.gov Identifier: NCT02556736     History of Changes
Other Study ID Numbers: RST-001-CP-0001
Study First Received: September 21, 2015
Last Updated: July 7, 2017

Keywords provided by Allergan:
gene therapy
optogenetics
channelrhodopsin
retina
retinitis pigmentosa

Additional relevant MeSH terms:
Retinitis
Retinitis Pigmentosa
Retinal Diseases
Eye Diseases
Eye Diseases, Hereditary
Retinal Dystrophies
Retinal Degeneration
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on August 23, 2017