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rAAV2tYF-GRK1-hRPGRco · 1 trial · 1 indication
LLVA(Low Luminance Visual Acuity) will be determined by adding a neutral density filter to the refraction using standard ETDRS (Early Treatment of Diabetic Retinopathy) visual acuity or tumbling "E" chart
| Arm | Type | Description |
|---|---|---|
| Group 1: Dose | ACTIVE_COMPARATOR | Male participants 12-50 years of age treated by subretinal injection with the of AGTC-501 |
| Group 2: Dose | ACTIVE_COMPARATOR | Male participants 12-50 years of age treated by subretinal injection with the dose of AGTC-501 |
| Group 3: Control | OTHER | Male participants 12-50 years of age in the untreated control group. Participants in the control group will be followed for a minimum of 24 months. After all participants have reached Month 12, participants in the control group will be given the option to receive the study drug in the fellow eye, if eligible. |
| Name | Type | Description |
|---|---|---|
| rAAV2tYF-GRK1-hRPGRco | BIOLOGICAL | Adeno-associated virus vector expressing a human RPGR gene |
| Control | DRUG | Untreated Control Group 3 |
General Inclusion Criteria: 1. Provide written informed consent or assent (per local regulation), prior to the conduct of any study-related procedure. Participants who provide assent must have a parent, guardian, or legal representative provide written informed consent. 2. Be between 12 and 50 year...
rAAV2tYF-GRK1-hRPGRco is an investigational gene therapy being developed for X-Linked Retinitis Pigmentosa, a rare inherited retinal disease. It is designed to deliver a functional RPGR gene to retinal cells via subretinal injection. The drug is currently in Phase 2 clinical development and has not been approved by regulatory authorities.
rAAV2tYF-GRK1-hRPGRco works by delivering a copy of the RPGR gene using an adeno-associated viral vector. The vector is engineered to target retinal cells, where the gene is intended to produce the RPGR protein that is deficient or defective in X-Linked Retinitis Pigmentosa. This approach aims to restore retinal cell function and slow disease progression.
rAAV2tYF-GRK1-hRPGRco is being developed by Elevance Health, Inc., a company traded on the New York Stock Exchange under the ticker ELV. The drug is in Phase 2 clinical trials for X-Linked Retinitis Pigmentosa.
rAAV2tYF-GRK1-hRPGRco is currently in Phase 2 clinical development. It is an investigational drug and has not been approved by the FDA or other regulatory agencies. The ongoing Phase 2 trial is active but not recruiting participants, with an enrollment target of 85 patients.
rAAV2tYF-GRK1-hRPGRco is being evaluated in a Phase 2 clinical trial registered as NCT04850118. This trial is assessing the safety and efficacy of a single subretinal injection of the drug in participants with X-Linked Retinitis Pigmentosa. The study is active but not recruiting, with 85 enrolled participants across the United States, Australia, Canada, and the United Kingdom.
Yes, rAAV2tYF-GRK1-hRPGRco is also known as AGTC-501. The Phase 2 clinical trial NCT04850118 is titled 'A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With XLRP,' confirming that both names refer to the same investigational gene therapy.