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Trial record 1 of 1 for:    retrosense
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RST-001 Phase I/II Trial for Retinitis Pigmentosa

This study is currently recruiting participants.
See Contacts and Locations
Verified October 2016 by RetroSense Therapeutics
Sponsor:
Information provided by (Responsible Party):
RetroSense Therapeutics
ClinicalTrials.gov Identifier:
NCT02556736
First received: September 21, 2015
Last updated: October 7, 2016
Last verified: October 2016
  Purpose

RST-001 is a gene therapy given as an injection into the eye and delivers a gene encoding a photo switch, channelrhodopsin-2, (optogenetics) to cells in the retina of the eye. When expressed, the channelrhodopsin-2 protein can depolarize in response to light thus generating a signal that is transmitted to the brain.

The study is composed of two parts. An initial dose-ranging study (part 1) is proposed whereby three dose levels of RST-001 will be studied in three separate groups of adult patients with advanced disease. This first part of the study is aimed at determining a single dose of the experimental agent which is safe and well tolerated, to further evaluate in a fourth group of patients. The second part of the study is aimed at obtaining additional safety data at the highest tolerated dose and providing important additional clinical data to guide the design of future efficacy studies.


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

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Phase I/IIa, Open-Label, Dose-Escalation Study of Safety and Tolerability of Uniocular Intravitreal RST-001 in Patients With Retinitis Pigmentosa (RP)

Resource links provided by NLM:


Further study details as provided by RetroSense Therapeutics:

Primary Outcome Measures:
  • Safety [ Time Frame: 6 months ]
    Any Grade 3 (National Cancer Institute [NCI] grading system) or greater adverse event considered related to injected test article


Estimated Enrollment: 15
Study Start Date: November 2015
Estimated Study Completion Date: April 2017
Estimated Primary Completion Date: April 2017 (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.
  4. Diagnosis of advanced retinitis pigmentosa (RP) defined as:

    1. Clinical diagnosis of advanced RP
    2. Prior documented retinal electrophysiological evidence of rod-cone photoreceptor degeneration
    3. Baseline evidence of <10microvolts maximal b-wave ERG response
  5. Visual acuity in the study eye of no-better-than hand motion
  6. Visual acuity in the non-study eye of no-better-than finger counting
  7. Presence of retinal ganglion cells and/or retinal nerve fiber layer on SD-OCT testing
  8. The presence of macular edema due to RP will not be considered an exclusion criteria
  9. Women of childbearing potential must have a negative pregnancy test at screening and at baseline, and agree to use an effective form of contraception, or be surgically sterile or postmenopausal (defined as last menstrual period greater than two years prior to baseline. Acceptable methods of contraception include hormonal contraception (i.e., birth control pills, injected hormones, dermal patch or vaginal ring), intrauterine device, barrier methods (diaphragm, condom) with spermicide, or surgical sterilization (tubal ligation).
  10. Males must use two forms of contraception (including one barrier method for three months following agent administration if their partner is of child-bearing potential), or must be surgically sterile.

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;
  3. Concurrent participation in another interventional clinical ocular study;
  4. Prior participation in any gene or stem cell therapy (ocular or other);
  5. Pre-existing eye conditions that would preclude the planned treatment (i.e. injection) or interfere with the interpretation of study endpoints or surgical complications (example would include (but not limited to) glaucoma, diseases affecting the optic nerve causing significant visual field loss, active uveitis, corneal or lenticular opacities);
  6. Complicating systemic diseases or clinically significant abnormal baseline laboratory values. Complicating systemic diseases would include those in which the disease itself, or the treatment for the disease, can alter ocular function. Examples are malignancies whose treatment could affect central nervous system function (for example, radiation treatment of the orbit; leukemia with central nervous system/optic nerve involvement). Also excluded would be subjects with any immunological response dysfunction, for example, immuno-compromising diseases or use of immunosuppressive medications, among others. Subjects who are positive for hepatitis B, C, and HIV will be excluded.;
  7. Cataract surgery, intraocular and/or peri-ocular injection in the study eye within the prior six months;
  8. Prior vitrectomy or aphakia in the study eye;
  9. Known sensitivity to any component of the study agent or medications planned for use in the peri-operative period.
  10. Current pregnancy or breastfeeding;
  11. Individuals of childbearing potential who are pregnant or unwilling to use effective contraception for the duration of the study;
  12. Subjects will be excluded if immunological studies show presence of neutralizing antibodies to AAV2 above 1:1000;
  13. Use of anti-platelet agents that may alter coagulation within 28 days prior to study agent administration;
  14. Any other condition that would not allow the potential subject to complete follow-up examinations during the course of the study and, in the opinion of the investigator, makes the potential subject unsuitable for the study.
  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: David G Birch, PhD 214-363-3911 ext 105

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
RetroSense Therapeutics
  More Information

Additional Information:
Responsible Party: RetroSense Therapeutics
ClinicalTrials.gov Identifier: NCT02556736     History of Changes
Other Study ID Numbers: RST-001-CP-0001
Study First Received: September 21, 2015
Last Updated: October 7, 2016

Keywords provided by RetroSense Therapeutics:
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 June 28, 2017