Safety of a one-time DSP-3077 retinal sheet injection in adults with retinitis pigmentosa
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)
This will test whether a single subretinal injection of DSP-3077 retinal sheets is safe and well tolerated in adults with retinitis pigmentosa.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 12 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Sumitomo Pharma America, Inc. Industry-sponsored |
| Locations | 2 sites (Boston, Massachusetts and 1 other locations) |
| Trial ID | NCT06891885 on ClinicalTrials.gov |
What this trial studies
This open-label, single-arm Phase 1/2a dose-escalation trial gives a single unilateral subretinal injection of iPSC-derived retinal sheets (DSP-3077) to 12 adults with nonsyndromic retinitis pigmentosa across three cohorts defined by baseline visual acuity. Each cohort of four participants receives one of two dose levels and participants are followed closely with frequent visits early after surgery then tapering to monthly, quarterly, and semiannual visits over approximately 60 months, with additional long-term safety follow-up planned up to 15 years. The primary objective is to measure safety and tolerability, with secondary assessments of engraftment, potential therapeutic response, and delivery device performance. The trial is sponsored by Sumitomo Pharma America and conducted at Massachusetts Eye and Ear and the University of Minnesota.
Who should consider this trial
Good fit: Ideal candidates are adults (≥18) with nonsyndromic retinitis pigmentosa who meet the study eye visual acuity ranges, are in generally good physical health, and can commit to long-term follow-up.
Not a fit: Patients with other eye diseases that impair vision, significant systemic illnesses, or those who do not meet the required study eye visual acuity ranges are unlikely to receive benefit from this intervention.
Why it matters
Potential benefit: If successful, the transplanted retinal sheets could engraft and potentially preserve or improve vision for people with retinitis pigmentosa.
How similar studies have performed: Early-phase human trials of retinal cell transplants have shown feasibility and acceptable short-term safety but limited and variable evidence for meaningful vision restoration, so this approach remains experimental.
Eligibility criteria
Show full inclusion / exclusion criteria
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.
Where this trial is running
Boston, Massachusetts and 1 other locations
- Massachusetts Eye and Ear — Boston, Massachusetts, United States (Recruiting)
- University of Minnesota — Minneapolis, Minnesota, United States (Recruiting)
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.