Phase 2
N=14
AGN-151597 (Formerly RST-001) Phase I/II Trial for Advanced Retinitis Pigmentosa
Advanced Retinitis Pigmentosa
Bottom Line
View on ClinicalTrials.gov: NCT02556736 ↗Enrolled (actual)
14
Serious AEs
14.3%
Results posted
Jun 2021
Primary outcome: Primary: Number of Participants With Any Grade 3 or Greater Adverse Event (AE) Considered Related to AGN-151597 — 0; 0; 0; 0 participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- AGN-151597 (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- AbbVie
- Primary completion
- Jun 2020
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Any Grade 3 or Greater Adverse Event (AE) Considered Related to AGN-151597 |
0; 0; 0; 0 | — |
| PRIMARY Visual Acuity in the Study Eye at Baseline and Month 6 |
0; 0; 0; 0; 0; 0 | — |
| PRIMARY Change From Baseline at Month 6 in Full Field Sensitivity in the Study Eye - Blue Light Threshold |
0.404; -6.600; 1.235 | — |
| PRIMARY Change From Baseline at Month 6 in Full Field Sensitivity in the Study Eye - Red Light Threshold |
0.903; -10.900; -0.232 | — |
| PRIMARY Change From Baseline at Month 6 in Full Field Sensitivity in the Study Eye - White Light Threshold |
0.109; -6.572; -4.700; -1.887 | — |
| PRIMARY Change From Baseline at Month 6 in Ambulation in the Study Eye (Time) |
-15.50; -7.25; -0.50; -4.50 | — |
| PRIMARY Change From Baseline at Month 6 in Ambulation in the Study Eye (Distance) |
-0.45; 0.16; -0.27; 0.02 | — |
| PRIMARY Intraocular Pressure (IOP) Measurements in the Study Eye |
13.3; 13.5; 18.7; 14.0; 13.3; 15.3 | — |
| PRIMARY Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence |
0; 0; 0; 1; 0; 0 | — |
| PRIMARY 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 |
1.760; 0.005 | — |
| PRIMARY 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 |
0.180; -0.055 | — |
| PRIMARY 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 |
0.570 | — |
| PRIMARY 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 |
14.0; 60.0 | — |
| SECONDARY Visual Acuity in the Study Eye at Months 3, 12, and 24 |
0; 0; 0; 0; 0; 0 | — |
| SECONDARY Change From Baseline at Months 3, 12, and 24 in Full Field Sensitivity in the Study Eye - Blue Light Threshold |
-1.097; -10.050; -0.475; 0.008; -7.300; -0.905 | — |
| SECONDARY Change From Baseline at Months 3, 12, and 24 in Full Field Sensitivity in the Study Eye - Red Light Threshold |
1.259; -5.700; -1.046; -0.301; -5.100; 1.537 | — |
| SECONDARY Change From Baseline at Months 3, 12, and 24 in Full Field Sensitivity in the Study Eye - White Light Threshold |
-0.666; -1.988; -7.500; -0.066; -0.351; -4.414 | — |
| SECONDARY Change From Baseline at Months 3, 12, and 24 in Ambulation Light-Guided Walking Test in the Study Eye (Time) |
-19.00; -6.00; 0.00; 1.50; -15.50; -12.00 | — |
| SECONDARY Change From Baseline at Months 3, 12, and 24 in Ambulation Light-Guided Walking Test in the Study Eye (Distance) |
-0.57; 0.07; -0.16; -0.01; -0.26; 0.20 | — |
| SECONDARY Change From Baseline at Months 3, 6, and 24 in Object Detection and Discrimination Scores |
-68.939; 5.998; -1.865; -18925.624; 10323.422; -124.924 | — |
| SECONDARY Change From Baseline at Months 3, 6, and 24 in Visual Evoked Potential (VEP) Scores in the Study Eye (SE/0.974 Amplitude) |
0.0; 1.0; 47.0 | — |
| SECONDARY Change From Baseline at Months 3, 6, and 24 in Visual Evoked Potential (VEP) Scores in the Study Eye (SE/0.649 Amplitude) |
0.0; 0.0; 10.3 | — |
| SECONDARY Change From Baseline at Months 3, 6, and 24 in Visual Evoked Potential (VEP) Scores in the Study Eye (SE/0.216 Amplitude) |
-4.0; 0.0; 40.5 | — |
| SECONDARY Change From Baseline at Months 3, 6, and 24 in Visual Evoked Potential (VEP) Scores in the Study Eye (SE/0.974 Latency) |
-1.0; 25.0; 40.0 | — |
| SECONDARY Change From Baseline at Months 3, 6, and 24 in Visual Evoked Potential (VEP) Scores in the Study Eye (SE/0.649 Latency) |
13.0; 15.0; -6.3 | — |
| SECONDARY Change From Baseline at Months 3, 6, and 24 in Visual Evoked Potential (VEP) Scores in the Study Eye (SE/0.216 Latency) |
-21.0; 7.0; 22.0 | — |
| SECONDARY Change From Baseline at Months 6 and 24 in Electroretinogram (ERG) Scores in the Study Eye (Amplitude) |
0.0; 0.0; 0.0; 0.0; 0.0; 0.0 | — |
| SECONDARY Change From Baseline at Months 6 and 24 in Electroretinogram (ERG) Scores in the Study Eye (Latency) |
0.0; 0.0; 0.0; 0.0; 0.0; 0.0 | — |
| SECONDARY Change From Baseline at Months 3, 6, and 24 in Composite Score of National Eye Institute (NEI) Visual Functioning Questionnaire (VFQ-25) Scores |
4.3; 5.5; -5.3; 0.9; 4.8; 6.2 | — |
| SECONDARY Anatomical Parameters as Measured in the Study Eye by Color Fundus Photography and Autofluorescence |
0; 0; 0; 1; 0; 0 | — |
| SECONDARY Change From Baseline at Months 3, 12, and 24 in the Anatomical Parameters in the Study Eye as Measured by Spectral Domain-OCT (SD-OCT) - Volume |
0.350; 0.060; 0.210; 0.070; 0.945; -0.040 | — |
| SECONDARY Change From Baseline at Months 3, 12, and 24 in the Anatomical Parameters in the Study Eye as Measured by Spectral Domain-OCT (SD-OCT) - Total Retinal Thickness |
29.0; 63.0; 12.0; 9.5; 17.0; -1.0 | — |
Summary
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.
Eligibility Criteria
Inclusion Criteria
Participants must meet all of the following criteria.
- Age >= 18 years.
- Signed and dated written informed consent obtained from the patient.
- 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:
- Unable or unwilling to meet requirements of the study.
- Participation in a clinical study (ocular or non-ocular) with an investigational drug, agent or therapy in the past six months.
Data sourced from ClinicalTrials.gov (NCT02556736). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.