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Nervous System Diseases clinical trials at UC Health
6 in progress, 2 open to eligible people

  • EDS in Ataxia Telangiectasia Patients

    open to eligible people ages 6 years and up

    This is an international, multi-center, one-year, randomized, prospective, double-blind, placebo-controlled, phase III study, designed to assess the effect of two non-overlapping dose ranges of EDS EP, administered by IV infusion once per month, on neurological symptoms of patients with Ataxia Telangiectasia.

    at UCLA

  • Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

    open to eligible males ages 5 years and up

    This study is a long-term study of ataluren in participants with nonsense mutation Duchenne muscular dystrophy.

    at UC Davis UCSF

  • A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy

    Sorry, in progress, not accepting new patients

    HOPE-2 is a double-blind clinical trial evaluating the safety and efficacy of a cell therapy called CAP-1002 in study participants with Duchenne muscular dystrophy (DMD). Non-ambulatory and ambulatory boys and young men who meet eligibility criteria will be randomly assigned to receive either CAP-1002 or placebo every 3 months for a total of 4 doses during a 12-month period.

    at UC Davis

  • AMX0035 in Patients With Amyotrophic Lateral Sclerosis (ALS)

    Sorry, in progress, not accepting new patients

    The CENTAUR trial will be a 2:1 (active:placebo) randomized, double-blind, placebo-controlled Phase II trial to evaluate the safety and efficacy of AMX0035 for the treatment of ALS.

    at UC Irvine

  • Combination Adenovirus + Pembrolizumab to Trigger Immune Virus Effects

    Sorry, in progress, not accepting new patients

    Glioblastoma (GBM) and gliosarcoma (GS) are the most common and aggressive forms of malignant brain tumor in adults and can be resistant to conventional therapies. The purpose of this Phase II study is to evaluate how well a recurrent glioblastoma or gliosarcoma tumor responds to one injection of DNX-2401, a genetically modified oncolytic adenovirus, when delivered directly into the tumor followed by the administration of intravenous pembrolizumab (an immune checkpoint inhibitor) given every 3 weeks for up to 2 years or until disease progression. Funding Source-FDA OOPD

    at UCLA

  • Safety and Efficacy Study of VY-AADC01 for Advanced Parkinson's Disease

    Sorry, in progress, not accepting new patients

    Safety and efficacy of AADC gene transfer in participants with Parkinson's disease.

    at UCSF

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