What side effects are associated with this type of intervention?

Common treatments for Retinitis Pigmentosa include gene therapy and vitamin supplementation. Gene therapy, which involves the use of viral vectors to deliver genetic material, can cause immune reactions and surgical complications. Vitamin A supplementation may help in some cases but carries risks of toxicity, such as liver damage and increased intracranial pressure. Vitamin E and dietary modifications are generally safer but their effectiveness is less certain.

What prior FDA approvals exist?

The FDA has approved voretigene neparvovec (Luxturna) for the treatment of Retinitis Pigmentosa and Leber congenital amaurosis caused by confirmed biallelic RPE65 mutations. This gene therapy involves a subretinal injection that delivers a normal copy of the RPE65 gene to retinal cells, aiming to restore visual function. This approval marks a significant advancement in the treatment of inherited retinal diseases using gene therapy.

What other types of research exist?

Promising novel alternatives to gene therapy for Retinitis Pigmentosa patients include stem cell-based therapies and retinal implants. Stem cell therapies involve the transplantation of retinal pigment epithelial cells derived from human embryonic stem cells, which have shown potential in clinical trials to restore vision. Retinal implants, such as the Argus II Retinal Prosthesis System, provide artificial vision by electrically stimulating the remaining retinal cells. Both approaches are advancing and may offer viable treatment options by 2024.

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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 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline to 24 months

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

Side effects data

From 2024 Phase 1 & 2 trial • 14 Patients • NCT02556736

21%

Intraocular pressure increased

Iritis

Hyperkalaemia

Arthralgia

Musculoskeletal chest pain

Neck Pain

Benign neoplasm of eyelid

Device dislocation

Pollakiuria

Eye irritation

Eye pain

Vitreal cells

Ear infection

Oral fungal infection

Sialoadenitis

Tooth infection

Fall

Post procedural haemorrhage

Post-traumatic pain

Cerebrospinal fluid leakage

Optic neuritis

Conjunctival haemorrhage

Retinal haemorrhage

Vitreous detachment

Vitritis

Intestinal obstruction

Swelling

Anterior chamber cell

Blepharitis

Eye discharge

100%

80%

60%

40%

20%

Study treatment Arm

RST-001

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

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

RST-001

2015

Completed Phase 2

~20

0 / 1Eligibility criteria met

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.