Efficacy & Safety of RPh201 Treatment in Patients With Previous Nonarteritic Anterior Ischemic Optic Neuropathy (NAION)

Phase 2

Terminated

• Conditions: Nonarteritic Anterior Ischemic Optic Neuropathy
• Interventions: Drug: RPh201 Cohort A; Other: Placebo Cohort A; Drug: RPh201 Cohort B; Other: Placebo Cohort B

• Registration Number: NCT03547206
• Lead Sponsor: Regenera Pharma Ltd

Brief Summary

This study is designed as a double-masked, randomized, placebo-controlled, clinical study to evaluate the efficacy and safety of subcutaneous (SC) administration of RPh201 in participants with previous NAION. All participants enrolled in Cohort A of the study will have a documented history of NAION for at least 12 months and at most, five years prior to enrollment. Participants enrolled in Cohort B of the study will have a documented history of NAION for at least 6 months and at most, three years prior to enrollment.

Detailed Description

This study is designed as a double-masked, randomized, placebo-controlled, clinical study to evaluate the efficacy and safety of SC administration of RPh201 in participants with previous NAION.

Following a screening phase of 1-8 weeks, participants will attend a baseline visit in which they will undergo testing and visual function assessments. Participants then will be randomized to receive RPh201 or control.

Cohort A After randomization, participants will begin a 26-week schedule consisting of twice-weekly treatment. Participants will return to the clinic for visits at Week 1, Week 4, Week 12 and Week 26 and Week 52

Cohort B After randomization, participants will begin a 12-week schedule consisting of four-times-per-week treatment. Participants will return to the clinic for visits at Week 4 and Week 12.

Safety and efficacy parameters will be recorded throughout the duration of the study.

Study Timeline

• Study First Submitted: 2018-05-24
• Study First Submitted QC: 2018-05-24
• Study First Posted: 2018-06-06
• Study Start: 2018-07-10
• Primary Completion: 2020-09-27
• Study Completion: 2020-09-27
• Status Verified: 2020-10
• Last Update Submitted: 2020-10-08
• Last Update Posted: 2020-10-12

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 165

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
RPh201 Cohort A	RPh201 Cohort A	A 26-week schedule consisting of twice-weekly subcutaneous administration of 400 μL of the Investigational Medical Product (IMP) (20 mg RPh201).
Placebo Cohort A	Placebo Cohort A	A 26-week schedule consisting of twice-weekly subcutaneous administration of 400 μL of the vehicle control.
RPh201 Cohort B	RPh201 Cohort B	A 12-week schedule consisting of four-times-per-week subcutaneous administration of 400 μL of the Investigational Medical Product (IMP) (20 mg RPh201).
Placebo Cohort B	Placebo Cohort B	A 12-week schedule consisting of four-times-per-week subcutaneous administration of 400 μL of the vehicle control.

Primary Outcome Measures

Name	Time	Method
The change in number of best-corrected visual acuity (BCVA) letters from baseline to Week 26 (Cohort A) measured using electronic visual acuity (EVA).	Week 26 or Week 12	Visual acuity
The change in number of best-corrected visual acuity (BCVA) letters from baseline to Week 12 (Cohort B) measured using electronic visual acuity (EVA).	Week 12	Visual acuity

Secondary Outcome Measures

Name	Time	Method
The proportion of study eyes improving by a 15-letter score or more in BCVA from baseline to Week 26 using EVA (Cohort A).	Week 26	Visual acuity
The proportion of study eyes improving by a 15-letter score or more in BCVA from baseline to Week 12 (Cohort B).	Week 12	Visual acuity
The proportion of study eyes improving from baseline in five or more locations of the Humphrey visual field (HVF) 24-2 full-threshold with the size V stimulus on the glaucoma change probability map (GCPM) at the 5% level by group.	Week 26	Humphrey visual field (HVF)
The proportion of study eyes improving by a 10-letter score or more in BCVA from baseline to Week 26 (Cohort A) using EVA.	Week 26	Visual acuity
The proportion of study eyes improving by a 10-letter score or more in BCVA from baseline to Week 12 (Cohort B) using EVA.	Week 12	Visual acuity

Trial Locations

Locations (14)

Emory University; 🇺🇸 Atlanta, Georgia, United States

Neuro-Eye Clinical Trials, Inc.; 🇺🇸 Houston, Texas, United States

New York Eye and Ear Infirmary of Mount Sinai; 🇺🇸 New York, New York, United States

UCLA Doheny Eye Center; 🇺🇸 Pasadena, California, United States

The Eye Care Group; 🇺🇸 Waterbury, Connecticut, United States

Bethesda Neurology, LLC; 🇺🇸 Rockville, Maryland, United States

Wills Eye Hospital; 🇺🇸 Philadelphia, Pennsylvania, United States

Byers Eye Institute at Stanford University; 🇺🇸 Palo Alto, California, United States

NorthShore Medical Group; 🇺🇸 Glenview, Illinois, United States

Anne Bates Leach Eye Hospital/Bascom Palmer Eye Institute; 🇺🇸 Miami, Florida, United States

Washington University Ophthalmology; 🇺🇸 Saint Louis, Missouri, United States

Charleston Neuroscience Institute; 🇺🇸 Ladson, South Carolina, United States

University of Virginia; 🇺🇸 Charlottesville, Virginia, United States

Massachusetts Eye and Ear Infirmary; 🇺🇸 Boston, Massachusetts, United States