NCT06388200

Brief Summary

This is a Phase 3 study to Assess the Efficacy, Safety and Tolerability of OCU400 in patients with retinitis pigmentosa (RP) associated with RHO mutations and patients with any other RP associated mutation with a clinical phenotype of RP. This is a multicenter, assessor blinded and randomized study which will enroll 140 subjects. Study has completed enrollment of all 140 subjects.

Trial Health

78
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
140

participants targeted

Target at P25-P50 for phase_3

Timeline
8mo left

Started Jun 2024

Typical duration for phase_3

Geographic Reach
2 countries

17 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress75%
Jun 2024Feb 2027

First Submitted

Initial submission to the registry

April 9, 2024

Completed
20 days until next milestone

First Posted

Study publicly available on registry

April 29, 2024

Completed
2 months until next milestone

Study Start

First participant enrolled

June 18, 2024

Completed
2.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 12, 2027

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

February 12, 2027

Last Updated

March 5, 2026

Status Verified

March 1, 2026

Enrollment Period

2.7 years

First QC Date

April 9, 2024

Last Update Submit

March 2, 2026

Conditions

Retinitis Pigmentosa

Keywords

Modified Gene therapyRetinitis PigmentosaRHORPNR2E3Subretinal Injection

Outcome Measures

Primary Outcomes (1)

  • Change in functional vision from baseline to week 52 in pooled analysis when study eyes in treatment group were compared to study eyes in untreated control

    Change in functional vision from baseline to week 52 in pooled analysis from retinitis pigmentosa subjects when study eyes in treatment group were compared to study eyes in untreated control, as measured by the ability of a study participant to navigate through a maze in Luminance Dependent Navigation Assessment (LDNA)

    52 weeks

Secondary Outcomes (4)

  • Change in functional vision from baseline to week 52 in all the treated eyes from RP subjects (study eyes + fellow treated eyes) when compared to all the eyes (study eyes and fellow eyes) in untreated control group

    52 weeks

  • Change from Baseline in visual function in patients with retinitis pigmentosa when treatment group were compared to untreated controls

    52 weeks

  • Change from baseline in LDNA Lux-level results in treated subjects when compared to the untreated controls

    52 weeks

  • Number of participants with change in visual function in Gene Agnostic Arm as assessed by LLVA letter scores

    52 weeks

Other Outcomes (11)

  • Change from baseline in Patients Global Impression of Change (PGIC) score

    52 weeks

  • Change from baseline in Best corrected visual acuity (BCVA) measured by ETDRS

    52 weeks

  • Improvement in Low luminance deficit (LLD) from baseline measured by ETDRS

    52 weeks

  • +8 more other outcomes

Study Arms (4)

RHO Arm

EXPERIMENTAL

Subjects will receive a sub-retinal injection of OCU400-301 modifier gene therapy product with a concentration of 2.5 x 10\^10 vg/eye

Genetic: Sub-Retinal Administration of OCU400-301

Gene Agnostic Arm

EXPERIMENTAL

Subjects will receive a sub-retinal injection of OCU400-301 modifier gene therapy product with a concentration of 2.5 x 10\^10 vg/eye

Genetic: Sub-Retinal Administration of OCU400-301

Control for RHO Arm

NO INTERVENTION

Will not receive any active study intervention

Control for Gene Agnostic Arm

NO INTERVENTION

Will not receive any active study intervention

Interventions

Sub-Retinal Administration of OCU400-301GENETIC

Sub-Retinal Administration of OCU400-301

Gene Agnostic ArmRHO Arm

Eligibility Criteria

Age3 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Males or females ≥ 3 years of age
  • Confirmed genetic diagnosis of autosomal dominant RHO mutation with clinical diagnosis of RP
  • Clinical Diagnosis of Syndromic or Non-Syndromic RP with/without confirmed genetic diagnosis of any other RP associated mutation (except AD-NR2E3)
  • BCVA ≤ 80 letters and ≥25 letters as measured by an ETDRS chart
  • Visual field of \>5° in any meridian as measured by a III4e isopter or equivalent
  • Able to perform a Luminance LDNA at certain light intensity at the Screening visit
  • Presence of photoreceptors as determined by SD-OCT

You may not qualify if:

  • Subject lacks evidence of outer nuclear layer
  • Previous treatment with a gene-therapy or cell therapy product or treatment with any investigational drug or ocular device within one year.
  • History of any corticosteroid contraindication, corticosteroid related IOP spikes or uncontrolled glaucoma.
  • Cataract surgery within 3 months. YAG capsulotomy within 1 month. Any other intraocular surgery within 6 months.
  • Active ocular/intraocular infection, any history of rhegmatogenous retinal detachment or Current retinal detachment or retinal implant.
  • Breast-feeding, pregnancy, sperm donation or inability to practice strict contraception

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (17)

Associated Retina Consultants

Phoenix, Arizona, 85020, United States

Location

University of Southern Califormia

La Jolla, California, 92093, United States

Location

University of Southern California, Roski Eye Insitute

Los Angeles, California, 90033, United States

Location

Advanced Research, LLC.

Deerfield Beach, Florida, 33064, United States

Location

Bascom Palmer Eye Institute, University of Miami, Miller School of Medicine

Miami, Florida, 33136, United States

Location

Erie Retina Research LLC

Erie, Pennsylvania, 16507, United States

Location

Vanderbilt Eye Institute

Nashville, Tennessee, 37232, United States

Location

Retina Consultants of Texas

Bellaire, Texas, 77401, United States

Location

Retina Foundation of the Southwest

Dallas, Texas, 75231, United States

Location

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

Valley Retina Institute

McAllen, Texas, 78503, United States

Location

Gundersen Health System

La Crosse, Wisconsin, 54601, United States

Location

Calgary Retina Consultants

Calgary, Alberta, T2H OC8, Canada

Location

University of Alberta

Edmonton, Alberta, T6G 3E1, Canada

Location

The University of British Columbia

Vancouver, British Columbia, V5Z 3N9, Canada

Location

Sunnybrook Health Sciences Centre

Toronto, Ontario, M4N 3M5, Canada

Location

Research Institute of the McGill University Health Centre

Montreal, Quebec, H2W 1L4, Canada

Location

MeSH Terms

Conditions

Retinitis Pigmentosa

Condition Hierarchy (Ancestors)

Eye Diseases, HereditaryEye DiseasesRetinal DystrophiesRetinal DegenerationRetinal DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Huma Qamar

    Ocugen

    STUDY CHAIR

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
SINGLE
Who Masked
OUTCOMES ASSESSOR
Masking Details
This is outcome assessor blinded study
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR
Expanded Access
Yes

Study Record Dates

First Submitted

April 9, 2024

First Posted

April 29, 2024

Study Start

June 18, 2024

Primary Completion (Estimated)

February 12, 2027

Study Completion (Estimated)

February 12, 2027

Last Updated

March 5, 2026

Record last verified: 2026-03

Data Sharing

IPD Sharing
Will not share

Locations

US(12)CA(5)