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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

ACTIVE_NOT_RECRUITING

Total Enrollment

18 participants

Study Classification

OBSERVATIONAL

Study Start Date

2023-12-08

Study Completion Date

2027-09-30

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

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

NCT04945772

Retinitis Pigmentosa Retinitis Retinal Diseases +4 more

COMPLETED PHASE2

RESCUE and REVERSE Long-term Follow-up

NCT03406104

Leber Hereditary Optic Neuropathy

COMPLETED PHASE3

A Long-Term Follow-Up Study in Subjects Who Received vMCO-I Administered Via Intravitreal Injection

NCT05921162

Retinitis Pigmentosa Retinal Disease Retinal Degeneration

ENROLLING_BY_INVITATION

Genetic Load and Phenotype in Aggressive AMD

NCT01650948

Age-related Macular Degeneration

COMPLETED

A Follow-up Study to Evaluate the Safety of RetinoStat® in Patients With Age-Related Macular Degeneration

NCT01678872

Age-Related Macular Degeneration

ACTIVE_NOT_RECRUITING PHASE1

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

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

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

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.

Gene Therapy product-MCO-010

Intervention Type BIOLOGICAL

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

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.

Gene Therapy product-MCO-010

Intervention Type BIOLOGICAL

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

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Gene Therapy product-MCO-010

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

Intervention Type BIOLOGICAL

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No