A Study to Investigate the Safety of DSP-3077 After a Unilateral Eye Injection in Male and Female Participants 18 Years of Age or Older With Retinitis Pigmentosa
DSP-3077 Retinal Sheet Cohort 1: 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
DSP-3077 Retinal Sheet Cohort 2: 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
DSP-3077 Retinal Sheet Cohort 3: 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
Study summary
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.
Eligibility
Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
Inclusion Criteria:
* Participant is \>= 18 years of age at the time of signing the informed consent.
* Participant has a clinical diagnosis of nonsyndromic retinitis pigmentosa.
* Participant is willing to consent to genetic testing, if not already done.
* Cohorts 1 and 2: Participant will have BCVA in the study eye between hand motion and 20 ETDRS letter score (approximately \<= 20/400 Snellen equivalent), inclusive at Screening and Baseline.
* Cohort 3: Participant will have BCVA in the study eye between 20 ETDRS letter score (approximately \>= 20/400 Snellen equivalent) and 35 ETDRS letter score (approximately \<= 20/200 Snellen equivalent), inclusive at Screening.
* Participant is in good physical health, based on medical history, physical examination, vital signs, electrocardiogram (ECG), and clinical laboratory tests at Screening.
Exclusion Criteria:
* Participant has an eye disease or visual disorder other than RP that impairs visual function (eg, retinal vascular disease, glaucoma).
* Participant has any other eye condition (eg, ocular media opacity, nystagmus), which in the opinion of the investigator, would preclude an accurate evaluation at any time during the study and/or make surgical delivery more challenging.
* Participant has any clinically significant unstable medical condition or any clinically significant chronic disease that in the opinion of the investigator, would limit the participant's ability to complete and/or participate in the study.
* Female participant who is pregnant or lactating or planning to become pregnant.
* Participant has received treatment with any nonapproved, experimental, or investigational therapy in either eye (systemic, topical, intravitreal) and/or received treatment in an interventional clinical trial for an eye disease or disorder within 90 days or 5 half-lives, whichever is longer, prior to Screening.
* Participant has previously received cell therapy, gene augmentation therapy, genome editing therapy, or any subretinal administered therapy for an eye disease or disorder.
Primary outcome measure(s)
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
Trial sites (2)
Facility
City
Region
Status
Massachusetts Eye and Ear
Boston
Massachusetts
Recruiting
University of Minnesota
Minneapolis
Minnesota
Recruiting
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This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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