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

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: NCT04945772
Recruitment Status : Completed
First Posted : June 30, 2021
Last Update Posted : March 22, 2024
Sponsor:
Information provided by (Responsible Party):
Nanoscope Therapeutics Inc.

Study Description
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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 (MCO-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-MCO-010 Procedure: Sham Injection Phase 2

Detailed Description:
This multicenter, randomized, double-masked, sham-controlled, dose-ranging study will evaluate 2 dose levels of MCO-010 in up to 18 subjects with retinitis pigmentosa (9 per dose). An additional 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 100 weeks following treatment with MCO-010.
Study Design
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Study Type : Interventional  (Clinical Trial)
Actual 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 MCO-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 MCO-010 Optogenetic Therapy in Adults With Retinitis Pigmentosa [RESTORE]
Actual Study Start Date : July 13, 2021
Actual Primary Completion Date : February 27, 2023
Actual Study Completion Date : January 18, 2024

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: MCO-010- High Dose
Participants receive 1.2E11gc/eye of MCO-010
 Biological: Gene Therapy Product-MCO-010
The MCO-010 is an adeno-associated virus serotype 2-based vector carried multi-characteristic opsin (MCO) gene expression cassette
Experimental: MCO-010- Low Dose
Participants receive 0.9E11gc/eye of MCO-010
 Biological: Gene Therapy Product-MCO-010
The MCO-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


Outcome Measures
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Primary Outcome Measures :
  1. Efficacy of a single intravitreal injection of Multi-Characteristic Opsin (MCO-010) as assessed by best corrected visual acuity. [ Time Frame: Week 52 ]
    Change from Baseline in the Freiburg Visual Acuity (quantitative LogMAR) score for the study eye at Week 52.


Secondary Outcome Measures :
  1. Efficacy of MCO-010 as assessed by best corrected visual acuity. [ Time Frame: Week 76 ]
    Change from Baseline in the Freiburg Visual Acuity (quantitative LogMAR) score for the study eye at Week 76.

  2. Efficacy of MCO-010 as assessed by mobility testing. [ Time Frame: Weeks 16,24,32,52,76,100 ]
    Change from Baseline in Multi-Luminance Y-Mobility Test score. Range: -1 to 5, higher score means better outcome.

  3. Efficacy of MCO-010 as assessed by mobility testing. [ Time Frame: Weeks 16,24,32,52,76,100 ]
    Proportion of subjects with Multi-Luminance Y-Mobility Test score scores of 2 or more light level improvement from Baseline. Range: 0% to100%, higher score means better outcome.

  4. Efficacy of MCO-010 as assessed by static shape recognition assay. [ Time Frame: Weeks 16,24,32,52,76,100 ]
    Proportion of subjects with Multi-Luminance Shape Discrimination Test scores of 2 or more light level improvements from Baseline. Range: 0% to 100%, higher score means better outcome.

  5. Efficacy of MCO-010 as assessed by static shape recognition assay. [ Time Frame: Weeks 16,24,32,52,76,100 ]
    Change from Baseline in Multi-Luminance Shape Discrimination Test score. Range: 0 to 5, higher score means better outcome.

  6. Efficacy of MCO-010 as assessed on visual field. [ Time Frame: Weeks 16,24,32,52,76,100 ]
    Change from Baseline in Visual Fields measured by Humphrey 30-2 perimetry.


Other Outcome Measures:
  1. Efficacy of MCO-010 as assessed by a composite of functional assessments. [ Time Frame: Week 52 ]
    Proportion of subjects demonstrating a ≥2 unit improvement from Baseline in EITHER the MLYMT OR the MLSDT score at Week 52.

  2. Safety of MCO-010. [ Time Frame: 100 weeks ]
    Incidences, nature, and severity of ocular and non-ocular treatment emergent adverse events (TEAEs).


Eligibility Criteria
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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:

  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) in the study eye and no better than 1.6 LogMAR (Snellen equivalent 20/800) in the fellow eye during screening.

Exclusion Criteria:

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

  1. Prior participation in gene therapy program
  2. 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).
  3. Presence of any complicating systemic diseases such as malignancies whose treatment could affect central nervous system function.
  4. Active ocular inflammation or recurrent history of idiopathic or autoimmune associated uveitis.
  5. Having received retinal prothesis (such as ARGUS-II) or any gene or stem cell therapy (ocular or non-ocular).
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): NCT04945772


Locations
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United States, California
Nanoscope Clinical Site
Beverly Hills, California, United States, 90211
United States, Florida
Nanoscope Clinical Site
Pensacola, Florida, United States, 32503
United States, North Dakota
Nanoscope Clinical Site
Fargo, North Dakota, United States, 58103
United States, Texas
Nanoscope Clinical Site
Houston, Texas, United States, 77030
Nanoscope Clinical Site
McAllen, Texas, United States, 78503
Puerto Rico
Nanoscope Clinical Site
Arecibo, Puerto Rico, 00612
Sponsors and Collaborators
Nanoscope Therapeutics Inc.
Investigators
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Study Chair: Dr Samarendra Mohanty Nanoscope Therapeutics Inc.
More Information
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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: March 22, 2024
Last Verified: March 2024
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: 12 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
Visual Acuity
Multi-Luminance Y Mobility Test (MLYMT)
Multi-Luminance Shape Discrimination Test (MLSDT)
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