Neurology Research at Dartmouth Hitchcock Medical Center Timothy G. Lukovits, MD Disclosures: • I have no financial disclosures • I am the local PI for the DIAS4, POINT and IRIS trials • I will only be discussing non-FDA approved treatments in the setting of clinical trials Neurology Research Overview • More than 35 Active Clinical Trials and Research Studies • Full age range of participants: children through seniors • Collaborative research effort with other departments within the medical center as well as Dartmouth College • 10 full and part time research coordinators – Provide assistance with both pharmaceutical sponsored trials as well as investigator initiated trials – Provide Institutional Review Board management and trial management Epilepsy trials now enrolling – UCB N01358 (Brivaracetam) – Cognitive development effects of Rufinamide for Lennox-Gastaut Syndrome – SABRIL Vision Study – HOBSCOTCH UCB N01358 (Brivaracetam) • Efficacy and Safety Study in Subjects With Partial Onset Seizures • Ages 16 – 80 • 1 or 2 concomitant AEDs, including VNS • 8 Type I seizures over 8-week baseline period • Doses of 100 and 200mg/day compared to placebo • Participants eligible for open-label extension study Adjunctive Rufinamide in Lennox-Gastaut Syndrome • Cognitive development effects and safety, and pharmacokinetics of adjunctive Rufinamide in subjects with inadequately controlled LGS. • Ages 1 – 3 • 1 to 3 concomitant AEDs • Randomized to adjunctive Rufinamide or any other approved AED. • Open label • Primary outcome: Change in Child Behavior Checklist (CBCL) Total Problems Score Sabril® (Vigabatrin) Vision Study • Structure and function of the retina in adult patients treated with Vigabatrin • Ages 18 and older • Complex partial epilepsy • 2 or more seizures/month • At least 1 concomitant AED • No prior exposure to Vigabatrin • Prospective, open label HOBSCOTCH • HOme Based Self-management and COgnitive Training CHanges Lives • Ages 18 -55, confirmed diagnosis of epilepsy, controlled or uncontrolled • Self-reported memory complaints and low quality of life • Intervention 1: problem solving and compensatory skills for cognitive dysfunction, taught in 8 weekly sessions • Intervention 2: Addition of BrainAge© program (NintendoDS) for 5 weeks Stroke/ TIA Clinical Studies now enrolling • Acute Stroke – DIAS4 – Multicenter acute stroke study involving desmoteplase in ischemic strokes – POINT Trial – Multi-center NIH sponsored trial of aspirin with or without Plavix in acute (less than 12 hours) minor stroke or TIA • Secondary Stroke Prevention – IRIS Trial – Multi-center NIH-sponsored of pioglitazone or placebo in adult ischemic stroke patients who do not have diabetes and are screened for insulin resistance. DIAS-4 – Desmoteplase is the recombinant form of the saliva found in the vampire bat, desmodus rotundus – Desmoteplase or placebo IV 1 min bolus – 3-9 hr window – 18-85y – MCA, ACA, PCA stenosis or occlusion on CTA or MRA – 1 hr between CT and Rx – mRS at 90days primary outcome measure “FLUFFY” Our stroke team’s mascot IRIS Insulin Resistance Intervention after Stroke Trial NIH U01 NS044876 Primary Aim: To determine if reduction of insulin resistance with pioglitazone, compared with placebo, will reduce the risk for stroke or myocardial infarction (MI) among nondiabetic patients with a recent ischemic stroke or transient ischemic attack (TIA) and insulin resistance. •Insulin resistance is associated with ↑ risk for atherosclerosis & clinical vascular disease. •The IRIS trial is based on the hypothesis that improving insulin resistance will reduce progression of atherosclerosis and prevent recurrent stroke and MI. •If successful, IRIS will introduce a new approach to stroke treatment. Eligibility: Recent TIA or Ischemic Stroke Non-diabetic Insulin resistant No congestive heart failure Enrollment: Expected total = 3,936 Expected at DHMC = 40 Enrolled to date = 38 Exited at 5 years = 3 Movement Disorders trial now enrolling – FS-ZONE FS-Zone • NIH-sponsored trial of 2 doses of pioglitazone or placebo in Parkinson’s Disease • Age 30 years or older, with idiopathic PD of less than 5 years duration • Inclusion - stable dosage of Rasagiline 1mg/day or Selegiline 10 mg/day for 2 to 8 months. • Exclusion – exposure to dopaminergic PD therapy or amantadine • Primary outcome: change in total UPDRS score. Handwriting analysis in PD - Stephen Lee • Observational study to characterize early neurophysiological changes in PD • NSF supported • Collaboration between • James Leiter MD, Joe Erlichman PhD • Norconnect, Inc. • EMG/EEG correlates during handwriting tasks. ~ 1.5 hour long session • Looking for patients who have not been on medications yet, or their unaffected spouse or friend. Multiple Sclerosis trials now enrolling Relapsing Remitting – FTY 2403 (Passages) Secondary Progressive – ASCEND Coming soon… Study validating questionnaires used as a tool in clinical studies PASSAGE • Safety study in patients with RRMS newly treated with Fingolimod or other approved diseasemodifying therapies • Long term, participants will be followed for 5 years • Participants will start fingolimod at time of study entry, or must have started another approved therapy within a maximum of 6 months prior to entry • Exclusion – previous or current treatment with a cytotoxic agent or natalizumab ASCEND Trial Objective: To investigate whether treatment with natalizumab slows the accumulation of disability not related to relapses in subjects with SPMS Inclusion Criteria: • Between 18-58 years old • Onset of SPMS at least 2 years prior to enrollment • EDSS score 3.0 – 6.5 inclusive • Disease progression independent of clinical relapses over the past year ALS Clinical Research Upcoming study – November 2012 CY4026 – “BENEFIT-ALS” an oral medication being investigated as a potential new therapy for the improvement of muscle weakness and muscle fatigue • Primary endpoint is the change from baseline to the average of the ALSFRS-R total score at specific time points • Participation is ~ 20 weeks with one week of openlabel period • Must have diminished, but measurable max voluntary hand grip and able to swallow medication • Upright slow Vital Capacity > 60% of predicted for age, height and sex. Thank You! For more info: email NeurologyResearch@hitchcock.org