General Information |
| Summary |
Study OpCT-001-101 is a Phase 1/2a first-in-human, multisite, 2-part interventional study to evaluate the safety, tolerability, and the effect on clinical outcomes of OpCT-001 in up to approximately 54 adults with primary photoreceptor (PR) disease. Phase 1 will focus on safety and features a dose-escalation design. Phase 2 is designed to gather additional safety data and assess the effect of OpCT-001 on measures of visual function, functional vision, and anatomic measures of engraftment in different clinical subgroups. |
| Description |
Phase 1 will include 4 planned dose levels to be administered across 4 cohorts (Cohorts 1 through 4). Participants will be assigned to receive OpCT-001.
Dose escalation in Phase 1 will be conducted using a standard 3+3 scheme in which a total of 12 to 24 legally blind participants (~3 to 6 per cohort) will receive OpCT-001. Phase 2 is planned to enroll a maximum of 15 participants per cohort in 2 cohorts (Cohorts 5 and 6) to evaluate 2 dose levels of OpCT-001 that will be selected based on Phase 1 safety and tolerability data. Phase 2 participants will be randomized 1:1 to either Cohort 5 or Cohort 6. Phase 2 participants and the investigator/study site personnel outside of the surgical team will be masked to OpCT-001 dose assignments. |
| Clinical trials phase |
Phases 1/2 |
| Start date (estimated) |
2025-02-01 |
| End date (estimated) |
2030-08-31 |
| Clinical feature |
| Label |
hereditary retinal dystrophy |
| Link |
http://purl.obolibrary.org/obo/DOID_8500 |
|
Administrative Information |
| NCT number |
NCT06789445 |
| ICTRP weblink |
https://trialsearch.who.int/Trial2.aspx?TrialID=NCT06789445 |
| Other study identifiers |
|
| Source weblink |
https://clinicaltrials.gov/study/NCT06789445 |
| Regulatory body approval |
| Name |
Food and Drug Administration (FDA) |
| Country |
|
|
| Public contact |
| Email |
apd86@med.miami.edu |
| Public email |
apd86@med.miami.edu |
| First name |
Adriana |
| Last name |
Drada |
| Phone |
+ 1 305-482-4292 |
| City |
Miami |
| Country |
|
|
| Sponsors |
BlueRock Therapeutics
|
Cells |
| Which differentiated cell type is used |
| Label |
photoreceptor cell |
| Link |
http://purl.obolibrary.org/obo/CL_0000210 |
| Description |
A cell specialized in detecting light stimuli that are involved in visual perception. |
|
Recruitment |
| Recruitment Status |
Not yet recruiting |
| Estimated number of participants |
54 |
