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

Retinitis Pigmentosa Clinical Trial

— REMAIN  

Official title:

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

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT06162585
• Other study ID #: NTXLTFU-008
• Status: Enrolling by invitation
• Start date: December 8, 2023
• Est. completion date: September 2027

Study information

• Verified date: December 2023
• Source: Nanoscope Therapeutics Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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

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.

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 18
• Est. completion date: September 2027
• Est. primary completion date: July 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Eye Diseases
• Eye Diseases, Hereditary
• Genetic Diseases, Inborn
• Retinal Degeneration
• Retinal Diseases
• Retinal Dystrophies
• Retinitis
• Retinitis Pigmentosa

Intervention

Biological:
• Gene Therapy product-MCO-010
Safety evaluation to monitor long term effects of previously injected MCO-010 in RP patients

Locations

Country	Name	City	State
Puerto Rico	Nanoscope Clinical Site	Arecibo
United States	Nanoscope Clinical Site	Beverly Hills	California
United States	Nanoscope Clinical Site	Fargo	North Dakota
United States	Nanoscope Clinical Site	Houston	Texas
United States	Nanoscope Clinical Site	McAllen	Texas
United States	Nanoscope Clinical Site	Pensacola	Florida

Sponsors (1)

Lead Sponsor	Collaborator
Nanoscope Therapeutics Inc.

Countries where clinical trial is conducted

United States,  Puerto Rico, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Assessment of the long-term pharmacokinetic (PK) and pharmacodynamic (PD) impact of previous treatment with MCO-010 on gene reporter expression	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)	156 Weeks
Primary	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	Evaluation of long-term effects on visual acuity of previous treatment with a single intravitreal injection of MCO-010	Change from baseline in BCVA over time in both eyes	156 Weeks
Secondary	Evaluation of long-term effects on shape discrimination at multiple light levels of previous treatment with a single intravitreal injection with MCO-010	Change from baseline in multi-luminance shape discrimination test (MLSDT) scores	156 Weeks
Secondary	Evaluation of long-term effects on navigation/mobility at multiple light levels of previous treatment with a single intravitreal injection with MCO-010	Change from baseline in multi-luminance Y-Mobility Test (MLYMT) score	156 Weeks
Secondary	Exploration of the long-term impact of previous treatment with MCO-010 on retinal thickness and retinal anatomy	Assessment of fundus photography and Optical Coherence Tomography (OCT) outcomes over time	156 Weeks