Clinical trial NCT06162585: Non-Interventional Long Term Follow-up Study of Participants Previously Enrolled in the RESTORE Study
Official title: Long Term Follow-up for Subjects Who Previously Participated in the NTXMCO-002 RESTORE Study
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
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
Lead sponsor: Nanoscope Therapeutics Inc.
Sponsor class: INDUSTRY
First posted: 2023-12-08
Enrollment: 18
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
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.

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

Detailed description: This study is designed to follow subjects with Retinitis Pigmentosa (RP) previously enrolled in study NTXMCO-002 (RESTORE, NCT04945772). In that study, 18 of 27 enrolled subjects received MCO-010, an ambient light-activated, Multi-Characteristic Opsin (MCO) transgene in an adeno-associated virus serotype 2 (AAV2) vector via intravitreal injection (IVT) and 9 of 27 received a sham injection. Those who received the sham injection will not be continued in the long-term, follow-up study for drug safety. MCO-010 has the potential to restore vision irrespective of the underlying gene mutation, and because it is directed at bipolar retinal cells, intact photoreceptors are not required. Further details on MCO-010 and the underlying disease under investigation are included in the protocol for RESTORE and are not repeated herein.

The current study is a non-interventional long-term safety follow-up of the subjects who completed RESTORE, in accordance with FDA guidance on recipients of human gene therapy products.

Eligibility criteria: Inclusion Criteria:

* 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.

Exclusion Criteria:

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

Primary outcomes:
measure: Assessment of the long-term safety of previous treatment with a single intravitreal injection of MCO-010
timeFrame: 156 weeks
Secondary outcomes:
measure: Evaluation of long-term effects on visual acuity of previous treatment with a single intravitreal injection of MCO-010
timeFrame: 156 Weeks
measure: Evaluation of long-term effects on shape discrimination at multiple light levels of previous treatment with a single intravitreal injection with MCO-010
timeFrame: 156 Weeks
measure: Evaluation of long-term effects on navigation/mobility at multiple light levels of previous treatment with a single intravitreal injection with MCO-010
timeFrame: 156 Weeks
measure: Exploration of the long-term impact of previous treatment with MCO-010 on retinal thickness and retinal anatomy
timeFrame: 156 Weeks
Other outcomes:
measure: Assessment of the long-term pharmacokinetic (PK) and pharmacodynamic (PD) impact of previous treatment with MCO-010 on gene reporter expression
timeFrame: 156 Weeks
Locations:
city: Beverly Hills
country: United States
facility: Nanoscope Clinical Site
state: California
city: Pensacola
country: United States
facility: Nanoscope Clinical Site
state: Florida
city: Fargo
country: United States
facility: Nanoscope Clinical Site
state: North Dakota
city: Houston
country: United States
facility: Nanoscope Clinical Site
state: Texas
city: McAllen
country: United States
facility: Nanoscope Clinical Site
state: Texas
city: Arecibo
country: Puerto Rico
facility: Nanoscope Clinical Site
Study officials:
affiliation: Nanoscope Therapeutics Inc.
name: Samuel Barone, MD
role: STUDY_DIRECTOR
References: