What side effects are associated with this type of intervention?

Gene therapies for Retinitis Pigmentosa, such as Ultevursen, often involve intravitreal injections and can have side effects including subretinal hemorrhage, inflammation, and resistance to drug injection. Other common side effects observed in similar treatments include mild to moderate ocular discomfort, increased intraocular pressure, and potential for cataract formation. These side effects are generally manageable but require careful monitoring by healthcare providers.

What prior FDA approvals exist?

The FDA has approved voretigene neparvovec (Luxturna) for the treatment of retinal dystrophy due to confirmed biallelic RPE65 mutations, which is a form of gene therapy similar to Ultevursen. Luxturna works by delivering a normal copy of the RPE65 gene directly to retinal cells, thereby improving vision. While Luxturna targets RPE65 mutations, Ultevursen is being studied for its efficacy in treating RP due to mutations in exon 13 of the USH2A gene, representing a similar approach in gene therapy for retinal diseases.

What other types of research exist?

Promising novel alternatives to Ultevursen for Retinitis Pigmentosa patients include: 1) Antisense oligonucleotide therapies, such as those targeting specific mutations in the USH2A gene. 2) CRISPR/Cas9-based gene editing, which aims to correct genetic mutations at the DNA level. 3) RNA interference (RNAi) therapies, which can silence the expression of mutant genes. 4) Optogenetics, which involves using light to control cells within the retina to restore vision. These approaches are in various stages of research and clinical development and may offer new hope for RP patients in the near future.

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Antisense Oligonucleotide

Ultevursen for Vision Impairments

Q: What is the mechanism of action of this treatment?

Gene therapy for Retinitis Pigmentosa (RP) aims to correct or compensate for the genetic mutations causing the disease. Treatments like Ultevursen, which target mutations in exon 13 of the USH2A gene, work by delivering a functional copy of the gene or modifying the existing gene to restore normal function. This approach is significant for RP patients because it addresses the root cause of the disease at the molecular level, potentially halting or reversing the progression of vision loss. By targeting specific genetic mutations, gene therapy offers a personalized treatment option that can lead to more effective and lasting outcomes compared to traditional therapies.

Phase 2 & 3

Waitlist Available

Research Sponsored by ProQR Therapeutics

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Male or female, ≥ 18 years of age OR a minor (12 to < 18 years) with permission from a parent or legal guardian. The lower age limit for pediatric populations is subject to local regulatory and ethics committee requirements.

An adult willing to comply with the protocol, follow study instructions, attend study visits as required and willing and able to complete all study assessments. OR A minor able to complete all study assessments and comply with the protocol and has a parent or caregiver willing and able to follow study instructions, and attend study visits with the subject as required.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 27 months

Summary

This trial tests a drug called ultevursen, which is injected into the eye, for patients with a specific genetic form of Retinitis Pigmentosa. The drug targets and modifies the genetic mutation causing their vision loss.

Who is the study for?

This trial is for adults and minors with parental consent who have Retinitis Pigmentosa due to USH2A gene mutations. Participants must have a certain level of vision in both eyes, no treatment history with genetic or stem-cell therapy, and not be allergic to the study medication components.

What is being tested?

The trial tests Ultevursen's effectiveness, safety, and tolerability when injected into the eye compared to a sham procedure. It targets patients with specific genetic mutations affecting their vision.

What are the potential side effects?

While side effects are not detailed here, similar treatments may cause eye discomfort or inflammation, temporary visual disturbances post-injection, potential allergic reactions, or increased intraocular pressure.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am 18 or older, or between 12 and 17 with parental consent.

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I am willing and able to follow the study's requirements and attend all visits.

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I have RP with Usher syndrome type 2 or a form of RP without other symptoms, confirmed by eye and ear tests.

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I have a specific genetic mutation in the USH2A gene.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 27 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~27 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 27 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

Mean change from baseline in BCVA

Secondary study objectives

Change from baseline in Full-field Stimulus Threshold (FST)

Change from baseline in Low Luminance Visual Acuity (LLVA)

Change from baseline in Microperimetry

+6 more

Other study objectives

Change from baseline in mobility course score

Trial Design

3Treatment groups

Experimental Treatment

Placebo Group

Group I: Ultevursen 60/60 µgExperimental Treatment1 Intervention

60 µg loading dose administered on Day 1, 60 µg maintenance dose administered at Month 3 and every 6 months thereafter

Group II: Ultevursen 180/60 µgExperimental Treatment1 Intervention

180 µg loading dose administered on Day 1, 60 µg maintenance dose administered at Month 3 and every 6 months thereafter

Group III: Sham-procedurePlacebo Group1 Intervention

Sham-procedure (no experimental drug administered) on Day 1, Month 3 and every 6 months thereafter

0 / 12Eligibility 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) aims to correct or compensate for the genetic mutations causing the disease. Treatments like Ultevursen, which target mutations in exon 13 of the USH2A gene, work by delivering a functional copy of the gene or modifying the existing gene to restore normal function. This approach is significant for RP patients because it addresses the root cause of the disease at the molecular level, potentially halting or reversing the progression of vision loss. By targeting specific genetic mutations, gene therapy offers a personalized treatment option that can lead to more effective and lasting outcomes compared to traditional therapies.