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NCT Number: NCT06162585

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

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

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Key information

Conditions

Retinitis Pigmentosa Congenital, Hereditary, and Neonatal Diseases and Abnormalities Eye Diseases Eye Diseases, Hereditary Genetic Diseases, Inborn Nervous System Diseases Neurologic Manifestations Pathological Conditions, Signs and Symptoms Retinal Degeneration Retinal Diseases Retinal Dystrophies Retinitis Sensation Disorders Signs and Symptoms Vision Disorders Vision, Low

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Nanoscope Clinical Site, Arecibo, Puerto Rico

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Gene Therapy Product-MCO-010

Biological

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

Primary outcomes

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

    Time frame: 156 weeks

    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.

Secondary outcomes

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

    Time frame: 156 Weeks

    Change from baseline in BCVA over time in both eyes

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

    Time frame: 156 Weeks

    Change from baseline in multi-luminance shape discrimination test (MLSDT) scores

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

    Time frame: 156 Weeks

    Change from baseline in multi-luminance Y-Mobility Test (MLYMT) score

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

    Time frame: 156 Weeks

    Assessment of fundus photography and Optical Coherence Tomography (OCT) outcomes over time

Other outcomes

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

    Time frame: 156 Weeks

    PK parameters including change from baseline of fundus fluorescence intensity of reporter over time and PD correlation of gene expression with the efficacy measures in the study eye and fellow eye (selected sites)

Sponsors and collaborators

Lead sponsor

Nanoscope Therapeutics Inc.

Industry

Registry information

Official study title

Long Term Follow-up for Subjects Who Previously Participated in the NTXMCO-002 RESTORE Study

Acronym: REMAIN

Important dates

Study start
2023
Primary completion
2027
Study completion
2027
First posted
Dec 8, 2023
Registry last updated
Mar 24, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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