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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT02556736
Recruitment Status : Recruiting
First Posted : September 22, 2015
Last Update Posted : March 7, 2018
Information provided by (Responsible Party):

Brief Summary:
RST-001 is a gene therapy given as an injection into the eye.

Condition or disease Intervention/treatment Phase
Advanced Retinitis Pigmentosa Drug: RST-001 Phase 1 Phase 2

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 21 participants
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
Estimated Primary Completion Date : August 28, 2019
Estimated Study Completion Date : October 31, 2033

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

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

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

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

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
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

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 identifier (NCT number): NCT02556736

Contact: Clinical Trials Registry Team 877-277-8566

United States, Texas
Retina Foundation of the Southwest Recruiting
Dallas, Texas, United States, 75231
Contact: David Birch, PhD   
Sponsors and Collaborators
Study Director: Werner Schmidt Allergan

Additional Information:
Responsible Party: Allergan Identifier: NCT02556736     History of Changes
Other Study ID Numbers: RST-001-CP-0001
First Posted: September 22, 2015    Key Record Dates
Last Update Posted: March 7, 2018
Last Verified: March 2018

Keywords provided by Allergan:
gene therapy
retinitis pigmentosa

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