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NCT Number: NCT06388200

A Phase 3 Study Of OCU400 Gene Therapy for the Treatment Of Retinitis Pigmentosa

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.

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This study is active but is not currently recruiting participants.

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Key information

Conditions

Retinitis Pigmentosa Congenital, Hereditary, and Neonatal Diseases and Abnormalities Eye Diseases Eye Diseases, Hereditary Genetic Diseases, Inborn Retinal Degeneration Retinal Diseases Retinal Dystrophies

Age range

3 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Calgary Retina Consultants, Calgary, Alberta, Canada

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About this study

A total of one hundred and forty (140) RP participants will be enrolled in this study into RHO arm or Gene agnostic arm. RHO arm will only enroll participants with confirmed genetic diagnosis of mutation in RHO gene; whereas Gene Agnostic arm will enroll RP Participants based on clinical diagnosis of RP and a confirmed genetic diagnosis with a gene associated with RP.

Subjects in each arm will be randomized into treatment and control groups with a 2:1 ratio. Subjects in the treatment group will receive a sequential, bilateral sub-retinal injection of OCU400 if both eyes meet inclusion criteria. Control or untreated group subjects will receive OCU400 subretinal injection after completion of 12-month follow-up.

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • 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

Exclusion criteria

  • 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

Treatment and study plan

Sub-Retinal Administration of OCU400-301

Genetic

Sub-Retinal Administration of OCU400-301

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

    Time frame: 52 weeks

    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)

Secondary outcomes

  1. 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

    Time frame: 52 weeks

    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, as measured by the ability of a study participant to navigate through a maze in Luminance Dependent Navigation Assessment (LDNA).

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

    Time frame: 52 weeks

    Change from Baseline in visual function in patients with retinitis pigmentosa when treatment group were compared to untreated controls in all RP subjects, as assessed by binocular low luminance visual acuity when using the Early Treatment Diabetic Retinopathy Study (ETDRS) chart letter score at Week 52.

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

    Time frame: 52 weeks

    Change from baseline in LDNA Lux-level results in all RP subjects in treated subjects when compared to the untreated controls at weeks 12, 24, 36, and 52

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

    Time frame: 52 weeks

    Change in LLVA letter scores in Gene Agnostic Arm subjects will be compared to controls

Other outcomes

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

    Time frame: 52 weeks

    Evaluate the efficacy of OCU400 gene therapy in treatment group when compared to untreated controls (in pooled analysis from patients in RHO arm and gene agnostic arm) with Retinitis Pigmentosa through week 52 as indicated by:

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

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

    Time frame: 52 weeks

    Evaluate the efficacy of OCU400 gene therapy in treatment group when compared to untreated controls (in pooled analysis from patients in RHO arm and gene agnostic arm) with Retinitis Pigmentosa through week 52 as indicated by:

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

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

    Time frame: 52 weeks

    Evaluate the efficacy of OCU400 gene therapy in treatment group when compared to untreated controls (in pooled analysis from patients in RHO arm and gene agnostic arm) with Retinitis Pigmentosa through week 52 as indicated by:

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

  4. Improvement in mean retinal sensitivity from baseline as measured by static perimetry in the 30-degree

    Time frame: 52 weeks

    Evaluate the efficacy of OCU400 gene therapy in treatment group when compared to untreated controls (in pooled analysis from patients in RHO arm and gene agnostic arm) with Retinitis Pigmentosa through week 52 as indicated by:

    Improvement in mean retinal sensitivity from baseline as measured by static perimetry in the 30-degree.

  5. Change from baseline in hyperfluorescent ring as measured by Wide field-FAF

    Time frame: 52 weeks

    Evaluate the efficacy of OCU400 gene therapy in treatment group when compared to untreated controls (in pooled analysis from patients in RHO arm and gene agnostic arm) with Retinitis Pigmentosa through week 52 as indicated by:

    Change from baseline in hyperfluorescent ring as measured by Wide field-FAF.

  6. Change from baseline in area of RP atrophy in macular region as measured by Wide field-FAF

    Time frame: 52 weeks

    Evaluate the efficacy of OCU400 gene therapy in treatment group when compared to untreated controls (in pooled analysis from patients in RHO arm and gene agnostic arm) with Retinitis Pigmentosa through week 52 as indicated by:

    Change from baseline in area of RP atrophy in macular region as measured by Wide field-FAF.

  7. Change from baseline in hypo autofluorescence assessments in peripheral retina as measured by Wide field-FAF

    Time frame: 52 weeks

    Evaluate the efficacy of OCU400 gene therapy in treatment group when compared to untreated controls (in pooled analysis from patients in RHO arm and gene agnostic arm) with Retinitis Pigmentosa through week 52 as indicated by:

    Change from baseline in hypo autofluorescence assessments in peripheral retina as measured by Wide field-FAF.

  8. Change from baseline in hyper autofluorescence assessments in peripheral retina as measured by Wide field-FAF

    Time frame: 52 weeks

    Evaluate the efficacy of OCU400 gene therapy in treatment group when compared to untreated controls (in pooled analysis from patients in RHO arm and gene agnostic arm) with Retinitis Pigmentosa through week 52 as indicated by change from baseline in hyper autofluorescence assessments in peripheral retina as measured by Wide field-FAF.

  9. Changes in the Ellipsoid zone area from baseline as measured by SD-OCT

    Time frame: 52 weeks

    Evaluate the efficacy of OCU400 gene therapy in treatment group when compared to untreated controls (in pooled analysis from patients in RHO arm and gene agnostic arm) with Retinitis Pigmentosa through week 52 as indicated by changes in Ellipsoid zone area from baseline as measured by SD-OCT.

  10. Changes in the outer segment length from baseline as measured by SD-OCT

    Time frame: 52 weeks

    Evaluate the efficacy of OCU400 gene therapy in treatment group when compared to untreated controls (in pooled analysis from patients in RHO arm and gene agnostic arm) with Retinitis Pigmentosa through week 52 as indicated by changes in the outer segment length as measured by SD-OCT.

  11. Changes in the outer segment volume from baseline as measured by SD-OCT

    Time frame: 52 weeks

    Evaluate the efficacy of OCU400 gene therapy in treatment group when compared to untreated controls (in pooled analysis from patients in RHO arm and gene agnostic arm) with Retinitis Pigmentosa through week 52 as indicated by changes in the outer segment volume from baseline as measured by SD-OCT.

Sponsors and collaborators

Lead sponsor

Ocugen

Industry

Registry information

Official study title

A Phase 3, Multi-Center, Randomized Study to Assess The Efficacy, Safety and Tolerability of Subretinal OCU400 Gene Therapy for the Treatment of Retinitis Pigmentosa

Acronym: liMeliGhT

Important dates

Study start
2024
Primary completion
2027
Study completion
2027
First posted
Apr 29, 2024
Registry last updated
Mar 5, 2026

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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