Clinical Research

Clinical research in the Department of Neurology continues to expand and significant numbers of patients with neurologic diseases are enrolled in ongoing clinical trials. This is an invaluable resource for the continued development of research in the department and attracts patients to our medical center. The clinical research program includes two major research centers: The UPMC Stroke Institute and the NIA-funded Alzheimer’s Disease Research Center (ADRC). Other areas of clinical research concentration include epilepsy, multiple sclerosis, headache and neuromuscular disorders. Some current projects include, but are not limited to:

Dr. Sara Berman is the site project director for the Dominantly-Inherited Alzheimer’s Network (DIAN) NIA grant and is the site project director for the first DIAN treatment trial in FY 17. She continues this work with the Grifols multicenter, randomized, controlled study to evaluate the efficacy and safety of short-term plasma exchange followed by long-term plasmapheresis with the infusion of human albumin combined with intravenous immunoglobin in patients with mild-moderate Alzheimer’s disease and initiated a new trial with Lundbeck in patients with mild to moderate AD.

Drs. Paula Clemens and Larry Wechsler are the Directors of the University of Pittsburgh site in the NINDS-funded NeuroNEXT, a national clinical trials network established to coordinate Phase 1-2 neuroscience clinical trials and Stroke Net.

Dr. Paula Clemens is the site project director for the Pompe Study. The purpose of this study is to determine if co-administration of investigational new drugs ATB200 and AT2221 is safe in adults with Pompe disease.

Dr. Paula Clemens is the site project director for the AMICUS study, “Open-label, Ascending-Dose, First-inHuman Study to Assess the Safety, Tolerability, and Pharmacokinetics of Intravenous Infusions of ATB200 Alone and ATB200 Co-administered with Oral AT2221 in Adult Subjects with Pompe Disease who were Previously Treated with Alglucosidase alfa”.

Dr. Paula Clemens received funding from TRiNDS LLC for a Phase II, Dose Finding Study to Access the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NS-065/NCNP-01 in Boys with Duchenne Muscular Dystrophy. The University of Pittsburgh is the coordinating center.

Dr. Islam Zayden is the site P.I. for the Open-Label, multicenter, expanded access program for Ocrelizumab in patients with primary progress Multiple Sclerosis study. The purpose of this study is to collect safety data and to monitor the clinical conditional of patients with PPMS who are taking ocrelizumab based on MRI, EDSS score, or clinical judgment.

Dr. Oscar Lopez is the Director of the NIA-funded Alzheimer Disease Research Center (ADRC), which has been successfully renewed through 2020. He completed a project examining amyloid deposition, vascular disease and clinical progress of AD. He is also the project leader on a PiB PET imaging Program Project Grant. He also completed a trial (Cytox) comparing the functional integrity of the mTOR pathway with the PET scan amyloid status (positive or negative) in subjects with a diagnosis of MCI. Several pharmaceutical trials to improve function in patients with AD are being conducted.

Dr. Oscar Lopez, Co-I, received funding from Cleveland Clinic for a U01. Biomarkers for the Lewy Body Dementia. This study enrolls subjects that have a baseline evaluation and at least two years of follow up data and biofluid collection. The study will evaluate motor and behavioral evaluations.

Dr. Oscar Lopez is the site PI for the ADNI 3 study. This study continues the previously funding ADNA-1, ADNI-GO, and ADNI-2 studies that have been combined public/private collaborations between academia and industry to determine the relationships between the clinical, cognitive, imaging, genetic and biochemical biomarker characteristics of the entire spectrum of Alzheimer’s disease (AD). The overall goal of the study is to continue to discover, optimize, standardize, and validate clinical trial measures and biomarkers used in AD research.

Dr. Oscar Lopez and Dr. Beth Snitz received funding for an R01 from NIA. Role of Midlife Cardiovascular Disease on Alzheimer’s Pathology and Cerebrovascular Reactivity in the Young-Old to further the understanding of the relationship between AD pathology and neurodegeneration, cerebrovascular reactivity and midlife CaVD.

Dr. Ayra Puwanant received funding from MDA. Sub Regional Body Composition and Clinical Endpoint in Myotonic Dystrophy to test the hypothesis that sub-regional LTM in the preferentially affected muscles may provide a sensitive endpoint that can be bridged to meaningful clinical outcomes for clinical trials in DM. The study cohort involves 20 individuals with DM1 and 20 individuals with DM2, and 20 age-matched healthy individuals.

Dr. Beth Snitz received funding from NIH for an R01. Alzheimer Neuroimaging-Biomarkers in Pre-Clinical Cognitive Decline from a Population-Based Study to will investigate in vivo brain imaging of the two neuropathological hallmarks of Alzheimer Disease (AD) in older adults evidencing pre-clinical cognitive decline. Appling these imaging tools to a carefully selected and characterized group of recruited participants, leveraging at least four years of serial, annual cognitive data from a population-based study to define preclinical cognitive decline.

Dr. Beth Snitz, Co-I, received funding on R01 from NIH. Connectomics of Brain Aging and Dementia to implement the HCP LifeSpan imaging protocol, and will use the HCP behavioral and cognitive assessments.

Dr. Suski is the site P.I. for the RESTORE study (a clinical study of patients with symptomatic neurogenic orthostatic hypotension to assess sustained effects of Droxidopa therapy). The purpose of this study is to evaluate the time to treatment in patients with PD, MSA, PAF, NDAN, or DBH Deficiency who have been previously stabilized with droxidopa therapy for symptoms of orthostatic hypotension.

Dr. Wechsler is the Director of the University of Pittsburgh NINDS funded Stroke Trials Network as a Regional Coordinating Center (RCC). The primary goals of the StrokeNet are to maintain a dynamic, collaborative and sustainable administrative structure to rapidly implement stroke clinical trials, enrolling patients across a wide range of adult and pediatric populations to promote the career development of the next generation of clinical investigators in the neurosciences.

Dr. Wechsler is the P.I. for the Sanbio Stem Cell Trial study. The SanBio Stem Cell trial is a DoubleBlind, Controlled Phase 2b Study of the Efficacy of Modified Stem Cells (SB623) in Patients with Chronic Motor Deficit from Ischemic Stroke.

Dr. Zongqi Xia received funding from NINDS for an R01. Integrating Electronic Health Records and Genomics to Predict Multiple Sclerosis Drug Response to gain insights into the factors that determine treatment response and enable physicians to match an individual MS patients clinical and genomic profile with uniquely tailored therapy to maximize effectiveness, delay disease progression and reduce overall costs.