Neuropathy clinical trials at UCLA

3 in progress, 2 open to eligible people

Showing trials for

  • DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants, Adolescents and Adults With Hearing Loss Due to Otoferlin Mutations

    open to all eligible people

    Regeneron is conducting a study of an investigational new drug called DB-OTO. DB-OTO is a gene therapy that is being developed to treat pediatric and adult participants who have severe-to profound and profound hearing loss due to changes in the otoferlin gene. The purpose of this study is to: - Learn about the safety of DB-OTO - Determine how well DB-OTO is tolerated (does not cause ongoing discomfort) - Evaluate the efficacy of DB-OTO (how well DB-OTO works)

    Los Angeles, California and other locations

  • Long-term Safety of Eplontersen Treated aTTR Patients and in Liver Transplant and Severely Hepatic Impaired Subpopulations

    open to eligible people ages 18 years and up

    The aim of this observational cohort study is to characterise use of eplontersen in patients with prior liver transplant or with pre-existing severe hepatic impairment, as well as to assess long-term safety among all new users of eplontersen; all are areas of missing information Primary objectives are: 1. To describe demographic and clinical characteristics of patients at eplontersen initiation, including the prevalence of prior liver transplant (overall and by reason for liver transplant), and the prevalence of severe hepatic impairment; and to describe patients in these subgroups (prior liver transplant, severe hepatic impairment). 2. To describe long-term safety in patients who initiate eplontersen treatment, including onset of new clinical events, abnormal laboratory values and serious adverse events.

    Los Angeles, California and other locations

  • Intranasal Cenegermin in Adult Participants With Non-Arteritic Anterior Ischemic Optic Neuropathy (NAION)

    Sorry, not currently recruiting here

    This is a phase 3, randomized, multicenter, vehicle-controlled, double-masked study to evaluate the efficacy and safety of intranasal cenegermin compared with vehicle control in adult participants with NAION. Approximately 272 participants who meet all eligibility criteria will be randomly assigned in a 1:1 ratio to receive either cenegermin treatment (Group 1) or the vehicle control (Group 2).

    Los Angeles, California and other locations

Our lead scientists for Neuropathy research studies include .

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