Basic Information


GTO ID GTC0267
Trial ID NCT02556736
Disease Retinitis Pigmentosa
Altered gene ChR2
Therapeutic/Target gene Therapeutic gene
TherapyGene transfer
Treatment RST-001
PhasePhase1|Phase2
Recruitment statusActive, Not Recruiting
TitlePhase I/IIa, Open-Label, Dose-Escalation Study of Safety and Tolerability of Intravitreal RST-001 in Patients With Advanced Retinitis Pigmentosa (RP)
Year2015
CountryUnited States
Company sponsorAbbVie
Other ID(s)RST-001-CP-0001
Vector information
Vectoradeno-associated virus
ConstructAAV2-ChR2
Vector typeadeno-associated virus serotype 2 (AAV2) vector
Transgene/Inserted genea photosensitivity gene, channelrhodopsin-2 (ChR2)

Clinical Result

Cohort 1
Administration route intravitreal injection
Pts 14
Age Adult, Older_Adult
Adverse reactions 9/14(Eye disorders; Gastrointestinal disorders; General disorders; Infections and infestations; Injury, poisoning and procedural complications; Metabolism and nutrition disorders; Neoplasms benign, malignant and unspecified, incl cysts and polyps; Nervous system disorders; Renal and urinary disorders)
References PMID: 35204770

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