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

NCT06891885 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: DE101101
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 2

Brief 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.

Conditions

Retinitis Pigmentosa

Outcome Measures

Primary Outcomes (7)

• 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

Study Arms (3)

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

Drug: DSP-3077 Retinal Sheet Cohort 1

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

Drug: DSP-3077 Retinal Sheet Cohort 2

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

Drug: DSP-3077 Retinal Sheet Cohort 3

Interventions

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

Also known as: Cohort 1

Cohort 1

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

Also known as: Cohort 2

Cohort 2

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

Also known as: Cohort 3

Cohort 3

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Sumitomo Pharma America, Inc.: lead

Study Sites (2)

Massachusetts Eye and Ear

Boston, Massachusetts, 02114, United States

RECRUITING

University of Minnesota

Minneapolis, Minnesota, 55455, United States

RECRUITING

MeSH Terms

Conditions

Retinitis Pigmentosa

Interventions

KPNA1 protein, human

Condition Hierarchy (Ancestors)

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

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 27, 2025
• First Posted: March 24, 2025
• Study Start: April 16, 2026
• Primary Completion (Estimated): October 31, 2028
• Study Completion (Estimated): October 31, 2032
• Last Updated: April 17, 2026

Record last verified: 2026-04

Locations

US (2)