Safety of a one-time DSP-3077 retinal sheet injection in adults with retinitis pigmentosa

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• Massachusetts Eye and Ear
  Boston, Massachusetts, United States Recruiting
• University of Minnesota
  Minneapolis, Minnesota, United States Recruiting

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.