Non-Interventional Long Term Follow-up Study of Participants Previously Enrolled in the RESTORE Study

NCT06162585 | Active Not Recruiting FDA Drug

• 1 other identifier: NTXLTFU-008
• Study Type: observational
• Target: 18
• Locations: 2 countries
• Sites: 6

Brief Summary

This study will be conducted following Good Clinical Practice (GCP) and International Conference on Harmonization (ICH) guidelines. Eligible subjects will be consented to return for scheduled study visits for this study following their completion in study NTXMCO-002 (RESTORE). They will not receive a second treatment with MCO-010 (or a repeated sham injection) in this study

Conditions

Retinitis Pigmentosa; Retinitis; Retinal Diseases; Eye Diseases; Eye Diseases, Hereditary; Retinal Dystrophies; Retinal Degeneration

Keywords

Gene Therapy; AAV Vectors; Low Vision; Intravitreal Injections; Optogenetics; Multi-Characteristic Opsin; Multi-Luminance Y Mobility Test; Multi-Luminance Shape Discrimination Test; Visual Acuity

Outcome Measures

Primary Outcomes (1)

• Assessment of the long-term safety of previous treatment with a single intravitreal injection of MCO-010

  Delayed adverse events. Incidence, nature, and severity of selected adverse events (AEs); all serious adverse events (SAEs); all ocular AEs including intraocular inflammation graded through ocular exam; non-ocular AEs with a common terminology criteria for adverse events (CTCAE) grade of 3 or greater; AEs of special interest (AESIs) including new malignancies, new incidence or exacerbation of any pre-existing neurologic disorder or rheumatologic or other autoimmune disorder, new incidence of hematologic disorder or new infection regardless of suspected relatedness to treatment with MCO-010.

  156 weeks

Secondary Outcomes (4)

• Evaluation of long-term effects on visual acuity of previous treatment with a single intravitreal injection of MCO-010

  156 Weeks

• Evaluation of long-term effects on shape discrimination at multiple light levels of previous treatment with a single intravitreal injection with MCO-010

  156 Weeks

• Evaluation of long-term effects on navigation/mobility at multiple light levels of previous treatment with a single intravitreal injection with MCO-010

  156 Weeks

• Exploration of the long-term impact of previous treatment with MCO-010 on retinal thickness and retinal anatomy

  156 Weeks

Other Outcomes (1)

• Assessment of the long-term pharmacokinetic (PK) and pharmacodynamic (PD) impact of previous treatment with MCO-010 on gene reporter expression

  156 Weeks

Study Arms (2)

Observation of Participants exposed 1.2E11gc/eye of MCO-010

This is a long-term follow-up observational study of participants who previously received 1.2E11gc/eye of MCO-010. No investigational product will be administered in this study.

Biological: Gene Therapy product-MCO-010

Observation of Participants exposed to 0.9E11gc/eye of MCO-010

This is a long-term follow-up observational study of participants who previously received 0.9E11gc/eye of MCO-010 No investigational product will be administered in this study.

Biological: Gene Therapy product-MCO-010

Interventions

Gene Therapy product-MCO-010 BIOLOGICAL

Safety evaluation to monitor long term effects of previously injected MCO-010 in RP patients

Observation of Participants exposed 1.2E11gc/eye of MCO-010; Observation of Participants exposed to 0.9E11gc/eye of MCO-010

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

This study population will be comprised of the 18 adult subjects with advanced retinitis pigmentosa, previously dosed with MCO-010 in the RESTORE study.

You may qualify if:

• Previously enrolled in study NTXMCO-002 (RESTORE)
• Able to comprehend and give informed consent.
• Able to comply with testing and all protocol tests.
• Agree to participate for the full 3-year duration of follow-up to the best of their ability and barring any unforeseen circumstances.

You may not qualify if:

• Not applicable. Subjects will be included in this study and will be consented after completion of all assessments at their final RESTORE study visit

Sponsors & Collaborators

• Nanoscope Therapeutics Inc.: lead

Study Sites (6)

Nanoscope Clinical Site

Beverly Hills, California, 90211, United States

Location

Nanoscope Clinical Site

Pensacola, Florida, 32503, United States

Location

Nanoscope Clinical Site

Fargo, North Dakota, 58103, United States

Location

Nanoscope Clinical Site

Houston, Texas, 77030, United States

Location

Nanoscope Clinical Site

McAllen, Texas, 78503, United States

Location

Nanoscope Clinical Site

Arecibo, 00612, Puerto Rico

Location

MeSH Terms

Conditions

Retinitis Pigmentosa; Retinitis; Retinal Diseases; Eye Diseases; Eye Diseases, Hereditary; Retinal Dystrophies; Retinal Degeneration; Vision, Low

Condition Hierarchy (Ancestors)

Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Vision Disorders; Sensation Disorders; Neurologic Manifestations; Nervous System Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Officials

• Samuel Barone, MD

  Nanoscope Therapeutics Inc.

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 30, 2023
• First Posted: December 8, 2023
• Study Start: December 8, 2023
• Primary Completion (Estimated): July 1, 2027
• Study Completion (Estimated): September 1, 2027
• Last Updated: March 24, 2025

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will share
• Time Frame: Within a year from the long term monitoring data availability
• Access Criteria: IPD sharing access will be subject to data transfer agreement. IPD generated as part of this clinical study may be subject to patient confidentiality.

Locations

US (5); PR (1)