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6 Usher Syndrome Trials

Power is an online platform that helps thousands of Usher Syndrome patients discover FDA-reviewed trials every day. Every trial we feature meets safety and ethical standards, giving patients an easy way to discover promising new treatments in the research stage.

Usher syndrome (USH) causes extensive degeneration in the cochlear nerve (CN), especially in CN fibers innervating the base of the cochlea. As the first step toward developing evidence-based practice for managing implant patients with USH, this study evaluates local neural health, as well as the neural encoding of temporal and spectral cues at the CN in implanted patients with USH. Aim 1 will determine local CN health in patients with USH by assessing the sensitivity of the electrically evoked compound action potential to changes in interphase gap and pulse polarity. Aim 2 will determine group differences in neural encoding of temporal and spectral cues at the CN between patients with USH and patients with idiopathic hearing loss. Aim 3 will use supervised machine learning techniques to develop an objective tool for assessing the electrode-neuron interface at individual electrode locations.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Unphased
Age:1 - 85
Sex:All
44 Participants Needed
This trial is testing if N-acetylcysteine (NAC) can help slow down vision loss in people with Retinitis Pigmentosa (RP). RP is a genetic condition that causes gradual vision loss. NAC reduces damage caused by too much oxygen around eye cells, potentially protecting vision. N-acetylcysteine (NAC) has been shown to reduce oxidative damage and increase cone function/survival in studies related to RP.
Pivotal Trial
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 3
Age:18 - 65
Sex:All
438 Participants Needed
The purpose of this Phase 2b study is to evaluate the safety and tolerability of Ultevursen administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene. This is a multicenter Double-masked, Randomized, Sham-controlled study which will enroll 81 subjects.
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:8+
Sex:All
81 Participants Needed
This trial will use stem cells from patients' own bone marrow to treat severe vision loss caused by retinal or optic nerve damage. The stem cells will be injected into the eye to help repair the damaged tissues. SCOTS is the largest ophthalmology stem cell study registered at the National Institutes of Health, using autologous bone marrow-derived stem cells for retinal and optic nerve diseases.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Unphased
Age:18+
Sex:All
500 Participants Needed
Study OpCT-001-101 is a Phase 1/2a first-in-human, multisite, 2-part interventional study to evaluate the safety, tolerability, and the effect on clinical outcomes of OpCT-001 in up to approximately 54 adults with primary photoreceptor (PR) disease. Phase 1 will focus on safety and features a dose-escalation design. Phase 2 is designed to gather additional safety data and assess the effect of OpCT-001 on measures of visual function, functional vision, and anatomic measures of engraftment in different clinical subgroups.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:18+
Sex:All
54 Participants Needed
Primary Objective: To evaluate the long-term safety and tolerability of SAR421869 in patients with Usher syndrome Type 1B Secondary Objective: To assess long-term safety and biological activity of SAR421869
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:18+
Sex:All
9 Participants Needed
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Frequently Asked Questions

How much do Usher Syndrome clinical trials pay?

Each trial will compensate patients a different amount, but $50-100 for each visit is a fairly common range for Phase 2–4 trials (Phase 1 trials often pay substantially more). Further, most trials will cover the costs of a travel to-and-from the clinic.

How do Usher Syndrome clinical trials work?

After a researcher reviews your profile, they may choose to invite you in to a screening appointment, where they'll determine if you meet 100% of the eligibility requirements. If you do, you'll be sorted into one of the treatment groups, and receive your study drug. For some trials, there is a chance you'll receive a placebo. Across Usher Syndrome trials 30% of clinical trials have a placebo. Typically, you'll be required to check-in with the clinic every month or so. The average trial length for Usher Syndrome is 12 months.

How do I participate in a study as a "healthy volunteer"?

Not all studies recruit healthy volunteers: usually, Phase 1 studies do. Participating as a healthy volunteer means you will go to a research facility several times over a few days or weeks to receive a dose of either the test treatment or a "placebo," which is a harmless substance that helps researchers compare results. You will have routine tests during these visits, and you'll be compensated for your time and travel, with the number of appointments and details varying by study.

What does the "phase" of a clinical trial mean?

The phase of a trial reveals what stage the drug is in to get approval for a specific condition. Phase 1 trials are the trials to collect safety data in humans. Phase 2 trials are those where the drug has some data showing safety in humans, but where further human data is needed on drug effectiveness. Phase 3 trials are in the final step before approval. The drug already has data showing both safety and effectiveness. As a general rule, Phase 3 trials are more promising than Phase 2, and Phase 2 trials are more promising than phase 1.

Do I need to be insured to participate in a Usher Syndrome medical study ?

Clinical trials are almost always free to participants, and so do not require insurance. The only exception here are trials focused on cancer, because only a small part of the typical treatment plan is actually experimental. For these cancer trials, participants typically need insurance to cover all the non-experimental components.

What are the newest Usher Syndrome clinical trials ?

Most recently, we added OpCT-001 for Retinitis Pigmentosa, Ultevursen for Retinitis Pigmentosa and N-acetylcysteine for Retinitis Pigmentosa to the Power online platform.