Clinical Studies

Parkinson's Disease:

Validating the Montreal Cognitive Assessment for the Diagnosis of Mild Cognitive Impairment in Parkinson's Disease

This is a longitudinal study sponsored by the Michael J. Fox Foundation. We are investigating whether a short paper and pencil test can determine changes in memory and thinking in individuals with Parkinson's disease. This study requires two visits per year for three years.

You may be eligible to participate if you:
1. Are at least 65 years old
2. Have been diagnosed with Parkinson's disease
3. Have color vision
4. Have a friend or family member who can answer questions about you. 


Finding the Optimum Regimen for Duchenne Muscular Dystrophy (FOR DMD) - The Finding the Optimum Regimen for Duchenne Muscular Dystrophy (FOR DMD) study will compare three ways of giving corticosteroids to boys with Duchenne Muscular Dystrophy (DMD) to determine which of the three ways increases muscle strength the most and which causes the fewest side effects. Using the results of this study, the investigators aim to provide patients and families with clearer information about the best way to take these drugs.

H6D-MC-LVJJ A Randomized, Double-Blind, Placebo Controlled, Phase 3 Trial of Tadalafil for Duchenne Muscular Dystrophy - H6D-MC-LVJJ is a 3 arm study to determine the efficacy and safety of Tadalafil administered orally once daily in ambulatory boys with DMD who are already receiving treatment with corticosteroids. The primary objective is to test the hypothesis that once daily Tadalafil administered orally for 48 weeks lessens the decline in ambulatory ability as measured by the 6MWD compared to placebo in boys with Duchenne muscular dystrophy (DMD). Two doses of Tadalafil (0.3mg/kg and 0.6mg/kg) will be compared to placebo.

BEL115123 A Randomized, Placebo Controlled, Double-Blind Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacodynamics of Belimumab in Subjects with Generalized Myasthenia Gravis (MG) - The purpose of this study is to test the safety and efficacy of belimumab to treat MG. Belimumab will be compared with a placebo (which contains no active medication). The effects of the drug, both good and bad, will be compared with the effects in people who are not being given the drug. You have a 50-50 chance of being placed in either group. About 50 people (adult women and men) with MG in at least 4 countries will take part in this study.

A Sample Repository for the Examination of Biomarkers for Amyotrophic Lateral Sclerosis (ALS) and Other Motor Neuron Diseases - This research is being done to find biological markers (biomarkers) of MND within blood, or cerebrospinal fluid (CSF). Biomarkers are measurable indicators of disease vulnerability (reflecting a risk to contract the disease), diagnosis (whether disease is present), and/or progression (worsening of disease). They may take a variety of forms, although this study focuses on protein biomarkers. Information gathered from this work may help clinicians and researchers create better treatment plans, develop new drugs, and perform further research.

Using Next Generation Sequencing to Unravel the Pathogenesis of Sporadic Inclusion Body Myositis (IBM) - The International IBM Consortium Genetic Study - Inclusion Body Myositis can cause muscle weakness, and may even lead to disability. It is a muscle disorder still without effective treatment. The aim of this study is to better understand what the cause of Inclusion Body Myositis is, by collecting clinical information and performing genetic studies on a blood, muscle or skin tissue sample. Approximately 20-40 people will take part in this research study at the Penn State Milton S. Hershey Medical Center.

POMPE Registry - The Pompe Registry was initiated worldwide in 2004 as part of an effort to help health care providers who are involved in treating or diagnosing Pompe disease to better understand the disease and its management. The Pompe Registry is open to all people with Pompe disease, regardless of whether they are receiving treatment and regardless of the type of treatment. Data that are regarded as standard assessments for Pompe patients, such as cardiac, respiratory, neuromuscular, sensory, Quality of Life and Enzyme Replacement Therapy (ERT) status will be collected and comprise the Pompe Registry database.

For any questions concerning the above studies, please contact Anne Morris at (717) 531-0003 x289123 or email:


Multiple Sclerosis:

STRATIFY-2: This clinical trial, closed to enrollment, involves a yearly blood draw to identify the JCV antibody, which is present in over 50% of patients diagnosed with multiple sclerosis. The purpose of this trial is to better understand whether antibodies to JCV may be used to predict whether a patient is at higher or lower risk for developing PML. Progressive multifocal leukoencephalopathy (PML) is a brain infection associated in some patients receiving Natalizumab (Tysabri®) therapy.  (P.I. Gary Thomas, MD).

Transition:  This is a two year observational study for MS patients who formerly used Tysabri as their disease modifying therapy, but have switched to Gilenya within the past 12 months. Most visits are standard of care and the medication is obtained commercially through your insurance. The purpose of this study is to evaluate the overall safety profile of Gilenya as well as the incidence of selected safety outcomes in patients previously exposed to Tysabri. (P.I. Elisabeth Lucassen, MD).

Baclofen GRS: The sponsor of this study has developed an investigational type of extended release Baclofen for MS patients with spasticity. This 21 week trial compares the treatment with Baclofene extended-release capsules versus down-titration to placebo in subjects stabilized on Baclofen ER with respect to efficacy of treatment of their spasticity, long-term efficacy over 12 weeks, and determination of a safety profile. Assessment of your spasticity is performed each visit. A daily diary is provided to document daytime and nighttime spasticity occurrences. The purpose of this study is to show that the extended release Baclofen, taken once each evening, will not only allow for more consistent control of spasms throughout the day and night, but possibly allow undisturbed sleep, and minimize the daytime sedation that is associated with Baclofen. (P.I. Elisabeth Lucassen, MD).

PreferMS: This one year trial is CLOSED to enrollment for relapsing remitting MS subjects naive to treatment or treated with no more than one disease modifying therapy. Qualified subjects are randomized to either one of the injectable MS medications (Copaxone, Betaseron, Extavia, Rebif or Avonex), or to Gilenya. The purpose of this study is to evaluate the effectiveness of Gilenya versus the injectables by patient retention on treatment over 12 months. (P.I. Elisabeth Lucassen, MD).

For more information on any of these Multiple Sclerosis trials, please contact Jen Crossen at 717-531-7591.