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The Goal of this study is to evaluate the safety, tolerability, and clinical responses following single dose of DSP-3077. Study enrolls both male and female patients in 3 cohorts with each cohort defined by visual acuity (VA) criteria and dose level of DSP-3077. Each cohort will include 4 participants.
This is a Phase 1/2a, open-label, single-arm, uncontrolled, dose-escalation study evaluating 2 dose levels of iPSC-derived retinal sheets (DSP-3077) administered with a single subretinal uniocular injection in adults with RP. The total duration of participant participation will be approximately 67 months from start of Screening through end of Extension Observation Period Part B. After an initial 2-week period of frequent visits after surgery, the visit frequency will be approximately monthly through Month 4, every 3 months through Month 24, and every 6 months through Month 60. After completion of Study DE101101 (Month 60 Visit or ET Visit) and under a separate process, the Sponsor will collect long-term data following treatment with DSP-3077, annually from 6 years to 15 years after DSP-3077 administration to further characterize the long-term safety of DSP-3077.
Participants will be treated in 3 cohorts, with each cohort defined by visual acuity (VA) criteria and dose level of DSP-3077. Each cohort will include 4 participants for a total of 12 participants.
Primary objective is to evaluate the safety and tolerability of 2 dose levels of DSP-3077 in adults with RP. Other objectives are to evaluate the safety, engraftment, and potential therapeutic response of 2 dose levels of DSP-3077 in adults with RP and to evaluate DSP-3077-delivery device performance.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cohort 1 | Experimental | Best Corrected Visual Acuity (BCVA) in the study eye between hand motion and 20 Early Treatment Diabetic Retinopathy Study (ETDRS) letter score (approximately ≤ 20/400 Snellen equivalent), inclusive at Screening and Baseline |
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| Cohort 2 | Experimental | Best Corrected Visual Acuity (BCVA) in the study eye between hand motion and 20 Early Treatment Diabetic Retinopathy Study (ETDRS) letter score (approximately ≤ 20/400 Snellen equivalent), inclusive at Screening and Baseline |
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| Cohort 3 | Experimental | Best Corrected Visual Acuity (BCVA) in the study eye between 20 Early Treatment Diabetic Retinopathy Study (ETDRS) letter score (approximately ≥ 20/400 Snellen equivalent) and 35 ETDRS letter score (approximately ≤ 20/200 Snellen equivalent), inclusive at Screening; BCVA in the study eye between 10 ETDRS letter score (approximately ≥ 20/640 Snellen equivalent) and 35 ETDRS letter score (approximately ≤ 20/200 Snellen equivalent) at Baseline |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| DSP-3077 Retinal Sheet Cohort 1 | Drug | For each participant only 1 eye will receive a low dose (>= 0.8 to < 2.4 mm2) of single subretinal injection of DSP-3077 using the DSP-3077-delivery device on Day 1 |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of ocular adverse events (AEs) | 60 months | |
| Incidence of ocular serious adverse events (SAEs) | 60 months | |
| Incidence of ocular adverse events (AEs) leading to discontinuation | 60 months | |
| Incidence of adverse events (AEs) | 60 months | |
| Incidence of serious adverse events (SAEs) | 60 months | |
| Incidence of adverse events (AEs) leading to discontinuation | 60 months | |
| Incidence of adverse events (AEs) leading to death | 60 months |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Massachusetts Eye and Ear | Recruiting | Boston | Massachusetts | 02114 | United States |
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| DSP-3077 Retinal Sheet Cohort 2 | Drug | For each participant only 1 eye will receive a high dose (>= 2.4 to < 6.4 mm2) of single subretinal injection of DSP-3077 using the DSP-3077-delivery device on Day 1 |
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| DSP-3077 Retinal Sheet Cohort 3 | Drug | For each participant only 1 eye will receive a high dose (>= 2.4 to < 6.4 mm2) of single subretinal injection of DSP-3077 using the DSP-3077-delivery device on Day 1 |
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| University of Minnesota | Recruiting | Minneapolis | Minnesota | 55455 | United States |
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| ID | Term |
|---|---|
| D012174 | Retinitis Pigmentosa |
| ID | Term |
|---|---|
| D015785 | Eye Diseases, Hereditary |
| D005128 | Eye Diseases |
| D058499 | Retinal Dystrophies |
| D012162 | Retinal Degeneration |
| D012164 | Retinal Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| ID | Term |
|---|---|
| C423142 | KPNA1 protein, human |
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