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

April 14, 2026 updated by: Sumitomo Pharma America, Inc.

A Prospective, Open-label, Single-arm, Dose-escalation Study of the Safety and Tolerability of a Single Subretinal Uniocular Injection of Allogeneic Induced Pluripotent Stem Cell (iPSC)-Derived Retinal Sheets (DSP-3077) in Adults With Retinitis Pigmentosa (RP)

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

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.

Study Type

Interventional

Enrollment (Estimated)

12

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
    • Minnesota
      • Minneapolis, Minnesota, United States, 55455
        • Recruiting
        • University of Minnesota
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Cohort 1
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
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
Other Names:
  • Cohort 1
Experimental: Cohort 2
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
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
Other Names:
  • Cohort 2
Experimental: Cohort 3
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
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
Other Names:
  • Cohort 3

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Incidence of ocular adverse events (AEs)
Time Frame: 60 months
60 months
Incidence of ocular serious adverse events (SAEs)
Time Frame: 60 months
60 months
Incidence of ocular adverse events (AEs) leading to discontinuation
Time Frame: 60 months
60 months
Incidence of adverse events (AEs)
Time Frame: 60 months
60 months
Incidence of serious adverse events (SAEs)
Time Frame: 60 months
60 months
Incidence of adverse events (AEs) leading to discontinuation
Time Frame: 60 months
60 months
Incidence of adverse events (AEs) leading to death
Time Frame: 60 months
60 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sumitomo Pharma America, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

April 16, 2026

Primary Completion (Estimated)

October 31, 2028

Study Completion (Estimated)

October 31, 2032

Study Registration Dates

First Submitted

February 27, 2025

First Submitted That Met QC Criteria

March 17, 2025

First Posted (Actual)

March 24, 2025

Study Record Updates

Last Update Posted (Actual)

April 17, 2026

Last Update Submitted That Met QC Criteria

April 14, 2026

Last Verified

April 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • DE101101

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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