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Gene Therapy

RST-001 for Retinitis Pigmentosa

Phase 1 & 2
Waitlist Available
Research Sponsored by Allergan
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Age must be greater than or equal to 18 years
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline to 24 months
Awards & highlights
No Placebo-Only Group

Summary

This trial is enrolling 12 patients with varying degrees of vision impairment. Half of the patients will have very poor vision, while the other half will have slightly better vision. The details of the intervention are not specified.

Who is the study for?
This trial is for adults over 18 with advanced retinitis pigmentosa. Participants must be able to follow the study's procedures and have given written consent. Those with very limited vision (no better than hand motion) or moderately limited vision (up to 20/200) can join. People who've been in another drug study within the last six months cannot participate.
What is being tested?
The trial is testing RST-001, a new potential treatment for retinitis pigmentosa. It involves a small group of just 12 patients in Phase I/II to assess how well it works and its safety at different stages of visual impairment.
What are the potential side effects?
Since this summary does not provide specific side effects of RST-001, we can assume that as an investigational therapy for a condition affecting the eyes, potential side effects may include eye irritation, discomfort, or changes in vision.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I am 18 years old or older.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline to 24 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline to 24 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Number of Participants With Any Grade 3 or Greater Adverse Event (AE) Considered Related to RST-001.
Secondary study objectives
Change in Ambulation
Change in Quality of Life
Visual Acuity

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: Group 1Experimental Treatment1 Intervention
Single intravitreal injection of RST-001

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
Gene therapy for Retinitis Pigmentosa (RP) involves delivering a functional copy of the defective gene to retinal cells using viral vectors like adeno-associated viruses (AAV). This treatment aims to restore or improve visual function by halting or reversing the degeneration of photoreceptor cells, which are crucial for vision. This approach is significant for RP patients as it targets the underlying genetic cause of the disease, offering the potential for long-term preservation or improvement of vision, rather than merely managing symptoms.
Gene therapy reforms photoreceptor structure and restores vision in NPHP5-associated Leber congenital amaurosis.Pharmacological and rAAV gene therapy rescue of visual functions in a blind mouse model of Leber congenital amaurosis.

Find a Location

Who is running the clinical trial?

AllerganLead Sponsor
781 Previous Clinical Trials
276,640 Total Patients Enrolled
1 Trials studying Retinitis Pigmentosa
21 Patients Enrolled for Retinitis Pigmentosa
AbbVieLead Sponsor
1,017 Previous Clinical Trials
520,012 Total Patients Enrolled
Hanh Badger, PharmDStudy DirectorAllergan
1 Previous Clinical Trials
80 Total Patients Enrolled

Media Library

RST-001 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT02556736 — Phase 1 & 2
Retinitis Pigmentosa Research Study Groups: Group 1
Retinitis Pigmentosa Clinical Trial 2023: RST-001 Highlights & Side Effects. Trial Name: NCT02556736 — Phase 1 & 2
RST-001 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02556736 — Phase 1 & 2
~1 spots leftby Oct 2025