AGN-151597 (Formerly RST-001) Phase I/II Trial for Advanced Retinitis Pigmentosa

NCTID	NCT02556736 (View at clinicaltrials.gov)
Description	All participants in phase 1 and phase 2a had hand motion visual acuity or worse. If efficacy was demonstrated from phase 1, better vision subjects could be enrolled; however, efficacy was not demonstrated. (Show More)
Development Status 🔄	Inactive
Indication	Advanced Retinitis Pigmentosa
Disease Ontology Term	DOID:10584
Drug Product Name 🔄	AGN-151597
Drug Product Alias 🔄	RST-001
Sponsor	AbbVie
Funder Type	Industry
Recruitment Status	Completed
Enrollment Count	14 (ACTUAL)
Results Posted	View Results

Target Gene/Variant	ChR2
Therapeutic Modality	Gene transfer
Therapy Route	in vivo
Mechanism of Action	Overexpression of protective allele/gene
Route of Administration	Intravitreal
Drug Product Type	Viral vector
Target Tissue/Cell	Retinal ganglion
Gene Delivery System Type	Viral transduction
Vector Type 🔄	Adeno-associated virus
Viral Vector Subtype	AAV2.7m8
Dose 1 🔄	4.3E10 vg/eye (n=3)
Dose 2 🔄	1.4E11 vg/eye (n=4)
Dose 3 🔄	4.3E11 vg/eye (n=7)

Eligible Age	>=18 Years
Standard Ages	Adult, Older adult
Sexes Eligible for Study	ALL
Eligibility Criteria	Inclusion Criteria: Participants must meet all of the following criteria. 1. Age \>= 18 years. 2. Signed and dated written informed consent obtained from the patient. 3. Ability to comply with testing and all protocol tests. Exclusion Criteria: Any one of the following will exclude patients from being enrolled into the study: 1. Unable or unwilling to meet requirements of the study. 2. Participation in a clinical study (ocular or non-ocular) with an investigational drug, agent or therapy in the past six months. View Inclusion and Exclusion Criteria at ClinicalTrials.gov

No.of Trial Sites	4
Locations	United States

Has US IND	True
FDA Designations	Orphan Drug Designation
Recent Updates	Clinical trial was initially sponsored by Allergan (prior to its acquisition by AbbVie). Development of AGN-151597 was discontinued for business reasons