Treatment Trials

4 Clinical Trials for Various Conditions

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ACTIVE_NOT_RECRUITING
SAD of IVT VP-001 in PRPF31 Mutation-Associated Retinal Dystrophy Subjects
Description

A Phase 1 Open-Label, Single Arm Dose Escalation Study to Evaluate the Safety and Tolerability of Intravitreally Administered VP-001 in Participants with Confirmed PRPF31 Mutation-Associated Retinal Dystrophy

NOT_YET_RECRUITING
A Study of Two Doses of VP-001 Administered Intravitreally in Participants with Confirmed PRPF31 Mutation-Associated Retinal Dystrophy Previously Treated with VP001
Description

This is a repeat-dose, open-label, four arm safety and efficacy study of two doses of VP-001 administered intravitreally in participants with confirmed PRPF31 mutation-associated Retinal Dystrophy and previously treated with VP001.

RECRUITING
MAD of IVT VP-001 in PRPF31 Mutation-Associated Retinal Dystrophy Subjects (Wallaby)
Description

A Phase 1 Open-Label, Multiple Ascending Dose Study to Evaluate the Safety and Tolerability of Intravitreally Administered VP-001 in Participants with Confirmed PRPF31 Mutation-Associated Retinal Dystrophy

ACTIVE_NOT_RECRUITING
Natural History of PRPF31 Mutation-Associated Retinal Dystrophy
Description

The purpose of this study is to characterize the natural history through temporal systemic evaluation of subjects identified with PRPF31 mutation-associated retinal dystrophy, also called retinitis pigmentosa type 11, or RP11. Assessments will be completed to measure and evaluate structural and functional visual changes including those impacting patient quality of life associated with this inherited retinal condition and observing how these changes evolve over time.