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Efficacy and Safety of of vMCO-010 Optogenetic Therapy in Adults With Retinitis Pigmentosa [RESTORE] (RESTORE)

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ClinicalTrials.gov Identifier: NCT04945772
Recruitment Status : Recruiting
First Posted : June 30, 2021
Last Update Posted : September 24, 2021
Sponsor:
Information provided by (Responsible Party):
Nanoscope Therapeutics Inc.

Brief Summary:
The purpose of the study is to evaluate the safety and efficacy of a single intravitreal injection of virally-carried Multi-Characteristic Opsin (vMCO-010).

Condition or disease Intervention/treatment Phase
Retinitis Pigmentosa Retinitis Retinal Diseases Eye Diseases Eye Diseases, Hereditary Retinal Dystrophies Retinal Degeneration Biological: Gene Therapy Product-vMCO-010 Procedure: Sham Injection Phase 2

Detailed Description:
This multicenter, randomized, double-masked, sham-controlled, dose-ranging study will evaluate 2 dose levels of vMCO-010 in up to 18 subjects with retinitis pigmentosa (9 per dose). Nine subjects will receive sham injection. Subjects with a confirmed diagnosis of Advanced Retinitis Pigmentosa (RP) based on clinical examination, dilated fundus examination, and genetic testing will be considered for participation in this study. All subjects will continue to be assessed for 52 weeks following treatment with vMCO-010.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 27 participants
Allocation: Randomized
Intervention Model: Sequential Assignment
Intervention Model Description: Following a 1:1:1 block randomization schema, 9 subjects will be enrolled in each vMCO-010 treatment group, and 9 subjects will be enrolled in the sham-controlled group.
Masking: Triple (Participant, Investigator, Outcomes Assessor)
Masking Description: Treatment assignment will be unknown (or masked) to the study participants, the evaluating physician (non-injecting), outcomes assessor, the sponsor and its agents.
Primary Purpose: Treatment
Official Title: A Phase 2b Randomized, Double-Masked, Sham-Controlled, Study to Evaluate the Efficacy and Safety of Intravitreal Injection of vMCO-010 Optogenetic Therapy in Adults With Retinitis Pigmentosa [RESTORE]
Actual Study Start Date : July 13, 2021
Estimated Primary Completion Date : January 1, 2023
Estimated Study Completion Date : January 1, 2023


Arm Intervention/treatment
Experimental: vMCO-010- High Dose
Participants receive 1.2E11gc/eye of vMCO-010
Biological: Gene Therapy Product-vMCO-010
The vMCO-010 is an adeno-associated virus serotype 2-based vector carried multi-characteristic opsin (MCO) gene expression cassette

Experimental: vMCO-010- Medium Dose
Participants receive 0.9E11gc/eye of vMCO-010
Biological: Gene Therapy Product-vMCO-010
The vMCO-010 is an adeno-associated virus serotype 2-based vector carried multi-characteristic opsin (MCO) gene expression cassette

Sham Comparator: Sham Injection
Participants receive sham injection
Procedure: Sham Injection
Sham Injection




Primary Outcome Measures :
  1. Efficacy of a single intravitreal injection of virally-carried Multi-Characteristic Opsin (vMCO-010) assessed by Y- Mobility Test. [ Time Frame: 52 weeks ]
    Change from Baseline in Y-Mobility Test compared to week 52.

  2. Type, severity, and incidence of ocular and systemic adverse events (AEs) [ Time Frame: 52 weeks ]
    Type, severity, and incidence of ocular and systemic adverse events (AEs), specifically those related to intravitreal injection of vMCO-010


Secondary Outcome Measures :
  1. Effect of vMCO-010 as assessed by static shape recognition assay [ Time Frame: 52 Weeks ]
    Change from baseline compared to Week 52 in the % shape recognition accuracy, measured with Static Shape recognition assay, performed at different light intensities

  2. Effect of vMCO-010 on determination of optical flow direction [ Time Frame: 52 Weeks ]
    Change from baseline compared to week 52 in accuracy determination of optical flow direction

  3. Effect of vMCO-010 as assessed by Freiburg Visual Acuity [ Time Frame: 52 Weeks ]
    Change from baseline compared to week 52 in Freiburg Visual Acuity.

  4. Effect of vMCO-010 on pupillary light reflex [ Time Frame: 52 Weeks ]
    Change from baseline compared to week 52 in the % change in pupil size.

  5. Effect of vMCO-010 on light sensitivity [ Time Frame: 52 Weeks ]
    Change from baseline compared to week 52 in the Full-field Stimulus Threshold (FST).

  6. To evaluate the effect of vMCO-010 on visual field [ Time Frame: 52 Weeks ]
    Change from baseline compared to week 52 in visual field measured by perimetry.

  7. Effect of vMCO-010 on functional vision outcomes [ Time Frame: 52 Weeks ]
    Change from baseline compared to week 52 in activities of daily living using the National Eye Institute Visual Function Questionnaire-25. Each question has several responses scored on a scale from 0 to 5, 0 to 6, or 0 to 10, and the Values are calculated in percentages.

  8. Pharmacokinetic (PK) impact of vMCO-010 on gene reporter expression [ Time Frame: 52 Weeks ]
    Change from baseline compared to week 52 in PK parameters including the rate of increase of fluorescence intensity of reporter measured by fluorescence funduscopy.



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:

The subject population includes subjects with advanced RP. Subjects are eligible to be included in the study only if all of the following criteria apply:

  1. Age ≥ 18 years
  2. Able to comprehend and give informed consent.
  3. Confirmed diagnosis of Advanced Retinitis Pigmentosa (RP) based on clinical examination, dilated fundus examination, and genetic testing.
  4. Best-Corrected (Freiburg) Visual Acuity worse than 1.9 LogMAR (Snellen equivalent 20/1600, Count Fingers/ Hand Motion) in the study eye and no better than 1.6 LogMAR (Snellen equivalent 20/800) in the fellow eye during screening.
  5. Presence of retinal inner nuclear and nerve fiber layers on optical coherence tomography (OCT) testing in the study eye during screening as determined by the Investigator and confirmed by the Sponsor or designee.

Exclusion Criteria:

Subjects are excluded from the study if any of the following criteria apply:

  1. Prior participation in gene therapy program
  2. Individuals who refuse or are incapable of performing mobility testing or pass the mobility testing at 0.3 or 1 lux as determined by the Investigator and confirmed by the Sponsor or designee during screening, will be excluded.
  3. Presence of an active implantable medical device
  4. Pre-existing conditions in the study eye such as glaucoma, diseases affecting the optic nerve causing significant visual field loss, active uveitis, corneal or lenticular opacities).
  5. Presence of any complicating systemic diseases such as malignancies whose treatment could affect central nervous system function.
  6. Subjects who are positive for syphilis, hepatitis B, C, and human immunodeficiency virus (HIV) will be excluded.
  7. Subjects who have undergone ocular surgery in the study eye within three months prior to Day 0.
  8. Presence of narrow iridocorneal angles contraindicating pupillary dilation in the study eye.
  9. Presence of disorders of the ocular media in the study eye which could interfere with visual acuity and other ocular assessments, including OCT, during the study period.
  10. Presence of vitreo-macular adhesion or traction, epiretinal membrane, macular pucker and macular hole in the study eye, evident by ophthalmoscopy and/or by OCT examinations and assessed by the investigator to significantly affect central vision.
  11. Current evidence of retinal detachment in the study eye that significantly affects central vision.
  12. Current use of hydroxychloroquine, chloroquine, or any related retina-toxic compounds.
  13. Active ocular inflammation or recurrent history of idiopathic or autoimmune associated uveitis.
  14. Having received retinal prothesis (such as ARGUS-II) or any gene or stem cell therapy (ocular or non-ocular).

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): NCT04945772


Contacts
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Contact: Kristen Peterson 701-483-3599 info@nanostherapeutics.com

Locations
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United States, California
Nanoscope Clinical Site Recruiting
Beverly Hills, California, United States, 90211
Contact: Janet Kurokouchi    213-483-8810      
United States, Florida
Nanoscope Clinical Site Not yet recruiting
Miami, Florida, United States, 33136
Contact: Rosa Potyra    305-243-2020      
Nanoscope Clinical Site Recruiting
Pensacola, Florida, United States, 32503
Contact: Jeanine Strickland         
United States, North Dakota
Nanoscope Clinical Site Recruiting
Fargo, North Dakota, United States, 58103
Contact: Tim Dahl    701-478-0061      
United States, Texas
Nanoscope Clinical Site Recruiting
Houston, Texas, United States, 77030
Contact: Jessica Monroe    713-524-3434      
Nanoscope Clinical Site Recruiting
McAllen, Texas, United States, 78503
Contact: Yesenia Salinas    956-631-8875      
Puerto Rico
Nanoscope Clinical Site Recruiting
Arecibo, Puerto Rico, 00612
Contact: Diane Perez    (787) 879-7722      
Sponsors and Collaborators
Nanoscope Therapeutics Inc.
Investigators
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Study Chair: Dr. Samarendra Mohanty Nanoscope Therapeutics Inc.
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Responsible Party: Nanoscope Therapeutics Inc.
ClinicalTrials.gov Identifier: NCT04945772    
Other Study ID Numbers: NTXMCO-002.
First Posted: June 30, 2021    Key Record Dates
Last Update Posted: September 24, 2021
Last Verified: September 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: The results of the clinical trial will be made available when the study is completed. The results will be published on this site and be available to conference presentations and publications.
Time Frame: 6 months after the study is completed
Access Criteria: Efficacy and Safety Results

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Nanoscope Therapeutics Inc.:
Retinitis Pigmentosa
Eye Diseases Hereditary
Eye Diseases
Retinal Degeneration
Inherited Retinal Diseases
Rod & cone dystrophies
Optogenetics
Gene Therapy
AAV vectors
Intravitreal Injections
Low Vision
Multi-Characteristic Opsin
No Light Perception
Low-Vision Multi-Parameter Test (LVMPT)
Additional relevant MeSH terms:
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Eye Diseases
Retinitis
Retinitis Pigmentosa
Retinal Diseases
Retinal Degeneration
Retinal Dystrophies
Eye Diseases, Hereditary
Genetic Diseases, Inborn