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

Primary Purpose

Retinitis Pigmentosa, Retinitis, Retinal Diseases

Status
Active
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Gene Therapy Product-MCO-010
Sham Injection
Sponsored by
Nanoscope Therapeutics Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Retinitis Pigmentosa focused on measuring 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)

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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).

Sites / Locations

  • Nanoscope Clinical Site
  • Nanoscope Clinical Site
  • Nanoscope Clinical Site
  • Nanoscope Clinical Site
  • Nanoscope Clinical Site
  • Nanoscope Clinical Site

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Sham Comparator

Arm Label

MCO-010- High Dose

MCO-010- Medium Dose

Sham Injection

Arm Description

Participants receive 1.2E11gc/eye of MCO-010

Participants receive 0.9E11gc/eye of MCO-010

Participants receive sham injection

Outcomes

Primary Outcome Measures

Efficacy of a single intravitreal injection of Multi-Characteristic Opsin (MCO-010) assessed by Multi Luminance Y- Mobility Test.
Change from Baseline in Multi Luminance Y-Mobility Test (MLYMT) score for the study eye at week 52

Secondary Outcome Measures

Proportion of Subjects with MLYMT Scores
Proportion of subjects with MLYMT scores of 2 or more light level improvement at week 52 using the study eye
Proportion of Subjects with MLSDT Scores
Proportion of subjects with Multi-Luminance Shape Discrimination Test (MLSDT) scores of 2 or more light level improvements at week 52 using the study eye
Change from baseline in MLYMT score
Change from baseline in the MLYMT score at Week 52 when using both eyes
Change from Baseline in MLYMT score
Change from baseline in MLYMT at Weeks 16, 24, 36, 52, 76 and 100
Proportion of MLYMT responders to treatment
Proportion of MLYMT responders to treatment at Weeks 16, 24, 36, 52, 76 and 100
Effect of MCO-010 as assessed by static shape recognition assay
Change from baseline in accuracy in determination of 3-dimensional shapes using the MLSDT at Weeks 16, 24, 36, 52, 76 and 100
Proportion of MLSDT responders to treatment
Proportion of MLSDT responders to treatment at Weeks 16, 24, 36, 52, 76 and 100
Effect of MCO-010 as assessed by Freiburg Visual Acuity
Change from baseline in Freiburg Visual Acuity (quantitative LogMAR score) at Weeks 16, 24, 36, 52, 76 and 100
To evaluate the effect of MCO-010 on visual field
Change from baseline in Visual Fields measured by Humphrey 30-2 perimetry at Weeks 16, 36, 52, 76 and 100

Full Information

First Posted
June 15, 2021
Last Updated
May 30, 2023
Sponsor
Nanoscope Therapeutics Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT04945772
Brief Title
Efficacy and Safety of MCO-010 Optogenetic Therapy in Adults With Retinitis Pigmentosa [RESTORE]
Acronym
RESTORE
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]
Study Type
Interventional

2. Study Status

Record Verification Date
May 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
July 13, 2021 (Actual)
Primary Completion Date
March 28, 2023 (Actual)
Study Completion Date
March 1, 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Nanoscope Therapeutics Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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).
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). 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 MCO-010.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Retinitis Pigmentosa, Retinitis, Retinal Diseases, Eye Diseases, Eye Diseases, Hereditary, Retinal Dystrophies, Retinal Degeneration
Keywords
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)

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Sequential Assignment
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
ParticipantInvestigatorOutcomes 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.
Allocation
Randomized
Enrollment
27 (Actual)

8. Arms, Groups, and Interventions

Arm Title
MCO-010- High Dose
Arm Type
Experimental
Arm Description
Participants receive 1.2E11gc/eye of MCO-010
Arm Title
MCO-010- Medium Dose
Arm Type
Experimental
Arm Description
Participants receive 0.9E11gc/eye of MCO-010
Arm Title
Sham Injection
Arm Type
Sham Comparator
Arm Description
Participants receive sham injection
Intervention Type
Biological
Intervention Name(s)
Gene Therapy Product-MCO-010
Intervention Description
The MCO-010 is an adeno-associated virus serotype 2-based vector carried multi-characteristic opsin (MCO) gene expression cassette
Intervention Type
Procedure
Intervention Name(s)
Sham Injection
Intervention Description
Sham Injection
Primary Outcome Measure Information:
Title
Efficacy of a single intravitreal injection of Multi-Characteristic Opsin (MCO-010) assessed by Multi Luminance Y- Mobility Test.
Description
Change from Baseline in Multi Luminance Y-Mobility Test (MLYMT) score for the study eye at week 52
Time Frame
100 weeks
Secondary Outcome Measure Information:
Title
Proportion of Subjects with MLYMT Scores
Description
Proportion of subjects with MLYMT scores of 2 or more light level improvement at week 52 using the study eye
Time Frame
100 weeks
Title
Proportion of Subjects with MLSDT Scores
Description
Proportion of subjects with Multi-Luminance Shape Discrimination Test (MLSDT) scores of 2 or more light level improvements at week 52 using the study eye
Time Frame
100 weeks
Title
Change from baseline in MLYMT score
Description
Change from baseline in the MLYMT score at Week 52 when using both eyes
Time Frame
100 weeks
Title
Change from Baseline in MLYMT score
Description
Change from baseline in MLYMT at Weeks 16, 24, 36, 52, 76 and 100
Time Frame
100 weeks
Title
Proportion of MLYMT responders to treatment
Description
Proportion of MLYMT responders to treatment at Weeks 16, 24, 36, 52, 76 and 100
Time Frame
100 weeks
Title
Effect of MCO-010 as assessed by static shape recognition assay
Description
Change from baseline in accuracy in determination of 3-dimensional shapes using the MLSDT at Weeks 16, 24, 36, 52, 76 and 100
Time Frame
100 Weeks
Title
Proportion of MLSDT responders to treatment
Description
Proportion of MLSDT responders to treatment at Weeks 16, 24, 36, 52, 76 and 100
Time Frame
100 weeks
Title
Effect of MCO-010 as assessed by Freiburg Visual Acuity
Description
Change from baseline in Freiburg Visual Acuity (quantitative LogMAR score) at Weeks 16, 24, 36, 52, 76 and 100
Time Frame
100 Weeks
Title
To evaluate the effect of MCO-010 on visual field
Description
Change from baseline in Visual Fields measured by Humphrey 30-2 perimetry at Weeks 16, 36, 52, 76 and 100
Time Frame
100 Weeks
Other Pre-specified Outcome Measures:
Title
Proportion of subjects demonstrating a ≥2 unit in either MLYMT or MLSDT Score
Description
Proportion of subjects demonstrating a ≥2 unit improvement from baseline in EITHER the MLYMT OR the MLSDT score at Week 52 in the study eye
Time Frame
100 weeks
Title
To evaluate the effect of MCO-010 on the listed safety endpoints
Description
Incidences, nature, and severity of ocular and non-ocular treatment emergent adverse events (TEAEs), Serious Adverse Events (SAEs); intraocular inflammation graded through ocular exam; intraocular pressure and retinal thickness
Time Frame
100 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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: Age ≥ 18 years Able to comprehend and give informed consent. Confirmed diagnosis of Advanced Retinitis Pigmentosa (RP) based on clinical examination, dilated fundus examination, and genetic testing. 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. 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: Prior participation in gene therapy program 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. Presence of an active implantable medical device 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). Presence of any complicating systemic diseases such as malignancies whose treatment could affect central nervous system function. Subjects who are positive for syphilis, hepatitis B, C, and human immunodeficiency virus (HIV) will be excluded. Subjects who have undergone ocular surgery in the study eye within three months prior to Day 0. Presence of narrow iridocorneal angles contraindicating pupillary dilation in the study eye. 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. 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. Current evidence of retinal detachment in the study eye that significantly affects central vision. Current use of hydroxychloroquine, chloroquine, or any related retina-toxic compounds. Active ocular inflammation or recurrent history of idiopathic or autoimmune associated uveitis. Having received retinal prothesis (such as ARGUS-II) or any gene or stem cell therapy (ocular or non-ocular).
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Dr Samarendra Mohanty
Organizational Affiliation
Nanoscope Therapeutics Inc.
Official's Role
Study Chair
Facility Information:
Facility Name
Nanoscope Clinical Site
City
Beverly Hills
State/Province
California
ZIP/Postal Code
90211
Country
United States
Facility Name
Nanoscope Clinical Site
City
Pensacola
State/Province
Florida
ZIP/Postal Code
32503
Country
United States
Facility Name
Nanoscope Clinical Site
City
Fargo
State/Province
North Dakota
ZIP/Postal Code
58103
Country
United States
Facility Name
Nanoscope Clinical Site
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Nanoscope Clinical Site
City
McAllen
State/Province
Texas
ZIP/Postal Code
78503
Country
United States
Facility Name
Nanoscope Clinical Site
City
Arecibo
ZIP/Postal Code
00612
Country
Puerto Rico

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing 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.
IPD Sharing Time Frame
12 months after the study is completed
IPD Sharing Access Criteria
Efficacy and Safety Results

Learn more about this trial

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

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