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RECRUITINGPhase 1 / Phase 2INTERVENTIONAL

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

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)

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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.

Who May Be Eligible (Plain English)

Who May Qualify: - Participant is \>= 18 years of age at the time of signing the willing to sign a consent form. - 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. Who Should NOT Join This Trial: - 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. ...See full criteria on ClinicalTrials.gov Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
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.

Treatments Being Tested

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

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

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

Locations (1)

Massachusetts Eye and Ear
Boston, Massachusetts, United States