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Nervous System Diseases clinical trials at UC Health
6 in progress, 3 open to new patients

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

    “Preliminary results show that the study drug (CAP-1002) may help improve weakened skeletal and heart muscle caused by DMD.”

    open to eligible males ages 10 years and up

    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)

    open to eligible people ages 18–80

    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

    open to eligible people ages 18 years and up

    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

  • Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Dystrophinopathy

    Sorry, in progress, not accepting new patients

    The main goal of this Phase 3 extension study is to obtain long term safety of ataluren in boys with nonsense mutation dystrophinopathy as determined by adverse events and laboratory abnormalities. The study will also assess changes in physical function, pulmonary function and other important clinical and laboratory measures.

    at UCLAUC Davis

  • 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

  • Telerehabilitation for Patients With Stroke

    Sorry, not yet accepting patients

    The aim of the current protocol is to study 40 patients, each for 90 days, to address hypotheses related to the ability of a telerehabilitation system to (a) improve motor status and disability, (b) collect various forms of patient data from the home, (c) improve risk factor knowledge and control, and (d) assess patient compliance with home-based telerehabilitation. Patients who have returned to their home after stroke will be provided with a telehealth system and be asked to use it 6 days/week for 90 days, during which time subjects will use this system for daily rehabilitation therapy, assessments, and education--all on one platform.

    at UC Irvine