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AGN-151597

Phase 1

Advanced Retinitis Pigmentosa | Small molecule | Ophthalmology |AbbVie Inc.|Last Updated: Jan 12, 2026

Success Probability

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Market & Valuation

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Trial Design

CONTROLLEDDMC
Total Trials1
Total Enrollment14

FDA Designations

No designations recorded

Clinical trial landscape

AGN-151597 · 1 trial · 1 indication

Phase 1 1
NCT02556736AGN-151597 (Formerly RST-001) Phase I/II Trial for Advanced Retinitis PigmentosaAdvanced Retinitis Pigmentosa
COMPLETED14 Analytics
PHASE1COMPLETED
AGN-151597 (Formerly RST-001) Phase I/II Trial for Advanced Retinitis Pigmentosa
Advanced Retinitis PigmentosaUnlock trial analytics

Study Endpoints

Primary Endpoints

Number of Participants With Any Grade 3 or Greater Adverse Event (AE) Considered Related to AGN-151597
Baseline (Day 1) to 6 Months

An adverse event is any untoward medical occurrence in a subject or clinical investigation patient administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product.

Visual Acuity in the Study Eye at Baseline and Month 6
Baseline (Day 1), 6 Months

Visual acuity of the study eye (with fellow eye covered) was measured using low vision assessment of count fingers, hand motion, and light perception. For count fingers testing, the examiner's hand presenting 1, 2, or 5 fingers is held 2 feet in front of the eye being examined. If the participant correctly identifies three of five presentations, then count fingers vision is noted. If not, then the participant must be tested for hand motion vision. For hand motion testing, the examiner's hand is extended 2 feet in front of the eye and moved horizontally or vertically. If the participant correctly identifies hand movement four out of five times, then hand motion vision is noted. If not, then the participant is tested for light perception. For light perception testing, a beam of light is directed in and out of the eye at least four times from a distance of 3 feet. If the participant correctly perceives the light, vision should be recorded as yes to light perception.

Change From Baseline at Month 6 in Full Field Sensitivity in the Study Eye - Blue Light Threshold
Baseline (Day 1) to Month 6

The light sensitivity of the visual field was measured by recording the threshold at which a participant reported seeing the dimmest flash. A negative value indicates an increase in sensitivity from baseline.

Change From Baseline at Month 6 in Full Field Sensitivity in the Study Eye - Red Light Threshold
Baseline (Day 1) to Month 6

The light sensitivity of the visual field was measured by recording the threshold at which a participant reported seeing the dimmest flash. A negative value indicates an increase in sensitivity from baseline.

Change From Baseline at Month 6 in Full Field Sensitivity in the Study Eye - White Light Threshold
Baseline (Day 1) to Month 6

The light sensitivity of the visual field was measured by recording the threshold at which a participant reported seeing the dimmest flash. A negative value indicates an increase in sensitivity from baseline.

Change From Baseline at Month 6 in Ambulation in the Study Eye (Time)
Baseline (Day 1) to Month 6

Evaluated a participant's ability to navigate within a dark room. The time from the start to stop and the distance from the center of the lit panel to where the participant stopped or touched the target (panel) were recorded. The test was first performed binocularly, then on the study eye (with non-study eye patched). A negative value indicates a decrease from baseline in time or distance from start to stop (improvement).

Change From Baseline at Month 6 in Ambulation in the Study Eye (Distance)
Baseline (Day 1) to Month 6

Evaluated a participant's ability to navigate within a dark room. The time from the start to stop and the distance from the center of the lit panel to where the participant stopped or touched the target (panel) were recorded. The test was first performed binocularly, then on the study eye (with non-study eye patched). A negative value indicates a decrease from baseline in time or distance from start to stop (improvement).

Intraocular Pressure (IOP) Measurements in the Study Eye
Baseline (Day 1), 6 Months

Intraocular pressure was measured using the Goldmann applanation tonometer or a hand-held tonometer (same instrument used for each participant throughout the study, when possible). Measurements were taken at baseline (pre-injection) and at 6 months.

Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence
Baseline (Day 1), 6 Months

A standardized procedure for the collection of single, non-stereo images of the fundus of both eyes was obtained using the same equipment for each participant throughout the study. Evidence of increased inflammation, hemorrhage, retinal detachment, RPE disturbance or atrophy in the fovea, and any changes from baseline visit were documented. Any changes from baseline in fundus autofluorescence were also documented.

Change From Baseline at Month 6 in the Anatomical Parameters in the Study Eye as Measured by Spectral Domain-Optical Coherence Tomography (SD-OCT) - Total Retinal Nerve Fibre Layer Volume
Baseline (Day 1) to Month 6

Qualitative assessment of the change in retinal cross-sectional appearance in the study eye from Baseline at month 6 as Measured by SD-OCT.

Change From Baseline at Month 6 in the Anatomical Parameters in the Study Eye as Measured by Spectral Domain-Optical Coherence Tomography (SD-OCT) - Total Ganglion Cell and Inner Plexiform Layer Volume
Baseline (Day 1) to Month 6

Qualitative assessment of the change in retinal cross-sectional appearance in the study eye from Baseline at month 6 as Measured by SD-OCT.

Change From Baseline at Month 6 in the Anatomical Parameters in the Study Eye as Measured by Spectral Domain-Optical Coherence Tomography (SD-OCT) - Total Retinal Volume
Baseline (Day 1) to Month 6

Qualitative assessment of the change in retinal cross-sectional appearance in the study eye from Baseline at month 6 as Measured by SD-OCT.

Change From Baseline at Month 6 in the Anatomical Parameters in the Study Eye as Measured by Spectral Domain-Optical Coherence Tomography (SD-OCT) - Total Retinal Thickness
Baseline (Day 1) to Month 6

Qualitative assessment of the change in retinal cross-sectional appearance in the study eye from Baseline at month 6 as Measured by SD-OCT.

Secondary Endpoints

Visual Acuity in the Study Eye at Months 3, 12, and 24
3, 12, and 24 Months
Change From Baseline at Months 3, 12, and 24 in Full Field Sensitivity in the Study Eye - Blue Light Threshold
Baseline (Day 1) to Months 3, 12, and 24
Change From Baseline at Months 3, 12, and 24 in Full Field Sensitivity in the Study Eye - Red Light Threshold
Baseline (Day 1) to Months 3, 12, and 24
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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Phase 1: Low DoseEXPERIMENTALSingle intravitreal injection of AGN-151597
Phase 1: Mid DoseEXPERIMENTALSingle intravitreal injection of AGN-151597
Phase 1: High DoseEXPERIMENTALSingle intravitreal injection of AGN-151597
Phase 2: High DoseEXPERIMENTALSingle intravitreal injection of AGN-151597

Interventions

NameTypeDescription
AGN-151597DRUGAGN-151597 is a gene therapeutic delivered by intravitreal injection
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites4

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...

Countries:United States
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Frequently asked questions about AGN-151597

What is AGN-151597 used for?

AGN-151597 is an investigational small molecule being developed for the treatment of advanced retinitis pigmentosa, a group of genetic disorders that involve a breakdown and loss of cells in the retina. It is administered to patients with this condition as part of a clinical trial.

Who makes AGN-151597?

AGN-151597 is being developed by AbbVie Inc., a biopharmaceutical company traded on the New York Stock Exchange under the ticker symbol ABBV. The drug is currently in clinical development for advanced retinitis pigmentosa.

What phase is AGN-151597 in?

AGN-151597 is in Phase 1 clinical development. It has completed a Phase 1/II trial for advanced retinitis pigmentosa, but it remains investigational and has not been approved by regulatory authorities. Further clinical development may be required before it can be considered for approval.

What clinical trials is AGN-151597 in?

AGN-151597 has been studied in one clinical trial, identified as NCT02556736, titled 'AGN-151597 (Formerly RST-001) Phase I/II Trial for Advanced Retinitis Pigmentosa.' This trial was conducted in the United States, enrolled 14 participants, and has been completed.

Is AGN-151597 the same as RST-001?

Yes, AGN-151597 was formerly known as RST-001. The clinical trial NCT02556736 is titled 'AGN-151597 (Formerly RST-001) Phase I/II Trial for Advanced Retinitis Pigmentosa,' confirming that the drug was previously referred to by the name RST-001.