ONGOING%20CLINICAL%20TRIALS

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ONGOING CLINICAL TRIALS

• University of Vermont
• Presented by Rup Tandan, MD, FRCP

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TRIAL OF HIGH FAT / HIGH CALORIE DIET IN ALS

• PI Rup Tandan, MD
• Supported By Muscular Dystrophy Association
• Objectives
• To compare the safety and tolerability in three
  subject groups randomized to high fat/high
  calorie, high calorie or control diets
• To measure biomarkers of body composition and
  lipid metabolism before and during diet
  intervention
• To examine preliminary effects of the three diets
  on measures of disease progression

Enrollment closes 10/31/12
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ALS RESEARCH GROUPDNA BANKING STUDY

• PI Rup Tandan, MD
• Supported By The National Institutes of Health
  (NIH),
• the Muscular Dystrophy Association, and the
• Amyotrophic Lateral Sclerosis Association (ALSA)
• Objectives To look at genetic factors that may
  contribute to the development of ALS and which
  may improve the genetic diagnosis of ALS and
  related Motor Neuron Diseases.

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A MULTI-CENTER, SINGLE BLIND, RANDOMIZED STUDY
COMPARING THYMECTOMY TO NON THYMECTOMY IN
NON-THYMOMATOUS MYASTHENIA GRAVIS (MG) PATIENTS
RECEIVING PREDNISONE

• PI Rup Tandan, MD
• Supported By The National Institutes of
  Neurological Disorders Stroke (NINDS)
• Objectives
• The main purpose of this research is to determine
  whether thymectomy has a positive influence on
  the management of patients with myasthenia gravis
  who are taking prednisone and who do not have a
  thymoma. The project's ancillary Biomarkers
  study will evaluate genetic factors and gene and
  protein expression in the blood and thymus (in
  patients undergoing thymectomy) which may
  influence MG.

Enrollment closes in November 2012
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CONTACT INFORMATION for Dr. Tandans Studies

• Rup Tandan, MD - Site PI
• [phone]
• Rup.Tandan_at_vtmednet.org
• Study Coordinator for Nutrition Study and
  Thymectomy Trial
• [phone]
• Shannon.Lucy_at_med.uvm.edu

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A MULTICENTER, DOUBLE-BLIND, PARALLEL GROUP,
PLACEBO CONTROLLED STUDY OF CREATINE IN SUBJECTS
WITH TREATED PARKINSONS DISEASE (PD).LONG-TERM
STUDY 1 (LS-1)

• PI Robert Hamill, MD
• Supported By NIH, National Institute of
  Neurological Disorders and Stroke (NINDS)
• Objective
• To determine if there is a slowing of clinical
  decline in PD patients defined by a combination
  of cognitive, physical, and quality of life
  measures.
•  

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NET-PD LS-1

• 1,720 subjects from approximately 52 US and
  Canadian sites will be equally randomized to the
  study arms.
•  
• Main Inclusion Criteria
• Subject is willing and able to give informed
  consent and commit to long-term follow-up
• PD within 5 years of diagnosis
• Treated/responsive to dopaminergic therapy
  (dopamine agonists or levodopa) for at least 90
  days, but not more than 2 years
• Enrollment was closed as of May 2011.
• There are 27 active patients at our site.

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A MULTICENTER, DOUBLE-BLIND, PLACEBO CONTROLLED,
PHASE II STUDY OF PIOGLITAZONE IN EARLY
PARKINSONS DISEASE (PD).(FS-ZONE)

• PI James Boyd, MD
• Supported By NIH, National Institute of
  Neurological Disorders and Stroke (NINDS)
• Objective
• To assess the impact of pioglitazone on the
  progression of PD in order to determine whether
  it is futile to proceed with further study of
  this agent. The secondary objectives of the study
  are to collect additional efficacy and
  safety/tolerability data to be used in planning a
  subsequent Phase III trial of pioglitazone in
  early, treated PD.

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NET-PD FS-ZONE

• Approximately 216 subjects from approximately 43
  sites in the US will be enrolled in this study to
  assess the impact of two doses of pioglitazone on
  the clinical decline of PD.
• Main Inclusion Criteria
• Subject is willing and able to give informed
  consent and commit to 44 weeks of follow-up
• Subjects who are on stable dose of rasagiline 1
  mg/day or selegiline 10 mg/day for at least 8
  weeks but no more than 8 months

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CONTACT INFORMATION

• Robert Hamill, MD - Site PI
• [phone]
• Robert.Hamill_at_vtmednet.org
• Study Coordinator
• [phone]
• Emily.houston_at_uvm.edu

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EPILEPSY CLINICAL TRIALSCLOSED TO FURTHER
ENROLLMENT

• An Open-Label Extension Phase of the
  Double-blind, Placebo-controlled,
  Dose-escalation, Parallel-group Study of E2007
  (perampanel) as an Adjunctive Therapy in Patients
  with Refractory Partial Seizures
• An open-label, multinational, multicenter,
  follow-up study to evaluate the long-term safety
  and efficacy of brivaracetam, used at a flexible
  dose up to a maximum of 150 mg/day, in subjects
  aged 16 years or older suffering from epilepsy.

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THE MS CENTEROF NORTHERN NEW ENGLAND AT
UVM/FLETCHER ALLEN

• Angela Applebee, MD Director
• Andrew Solomon, MD
• Sandra McGrath, RN FNP
• Patty Krusinski, CCRC, Study Coordinator
• AnneMarie Savage, RN, Research Nurse
• Jane Low, MPA, Study Coordinator

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ONGOING CLINICAL TRIALS IN MSCLOSED TO FURTHER
ENROLLMENT

• Novartis 2306
• A double-blind, randomized, multicenter,
  placebo-controlled, parallel-group study
  evaluating safety and efficacy of 0.5mg FTY720
  (fingolimod) in patients with PPMS
• Novartis 2309
• A DB, PC RCT to examine safety and efficacy
  of 2 doses of FTY720 (fingolimod) capsules in
  RRMS over 24 months.
• Biogen Idec Daclizumab Study in
  Relapsing-Remitting MS
• To determine the efficacy and safety of
  daclizumab high yield process (DAC HYP) versus
  Avonex (Interferon ß-1a) in patients with
  relapsing-remitting MS.

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ADDITIONAL CLINICAL TRIALS IN MSCLOSED TO
FURTHER ENROLLMENT (Continued)

• Extension Study AC-058B202
• Extension study to AC-058B201 to study long-term
  safety, tolerability and efficacy of 3 doses of
  ACT-128800 in RRMS
• EFC6260 Sanofi-Aventis
• International, multi-center, randomized,
  double-blind, placebo-controlled, parallel-group
  study to evaluate efficacy and safety of
  teriflunomide in patients with a first clinical
  episode suggestive of MS.

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ONGOING CLINICAL TRIALS IN MSOPEN TO ENROLLMENT

• Stratify 2 Biogen
• JCV Anitbody program in patients with RRMS
  receiving or considering treatment with Tysabri
• Genentech/Roche Ocrelizumab study in Relapsing MS
  
• A multicenter, randomized trial to evaluate the
  safety and efficacy of ocrelizumab in comparision
  to interferon beta-1a (Rebif) in patients with
  relapsing MS
• Opexa Therapeutics, Inc. In Secondary Progressive
  MS
• A multicenter, double-blind trial to evaluate the
  safety and effectiveness of an investigational
  T-cell product when compared to placebo in
  patients with secondary progressive MS

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STROKE/NEUROCRITICAL CAREIRIS

• (Insulin Resistance Intervention after Stroke)
• PI Mark Gorman, MD
• NINDS-funded multicenter randomized clinical
  secondary prevention trial
• Looks at reduction of recurrent stroke, MI and
  death in insulin resistant stroke/TIA patients
• Intervention is pioglitazone 45 mg (insulin
  sensitizer/PPAR-? agonist) vs. placebo
• Follow-up 3-5 years

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IRIS STUDY Contact Information

• Study Coordinator
• Catherine.Gregory_at_med.uvm.edu
• 802-656-8993
• Principal Investigator
• mgorman1_at_uvm.edu

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ABBOTT PARKINSONS STUDY

• PI James Boyd, MD
• Study of levodopa/carbidopa intestinal gel vs.
  oral levodopa/carbidopa
• For pts with advanced Parkinsons, who
  experience motor fluctuations and 3 or more hours
  of off time daily, despite optimized levodopa
  treatment. Closed to enrollment in July 2011.
• Pts who have had DBS or other surgery for
  Parkinsons are not eligible

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ABBOTT PARKINSONS STUDY (Continued)

• Levodopa/carbidopa intestinal gel is an approved
  therapy in Europe known as Duodopa. Under testing
  in U.S. for FDA approval.
• Medication delivered through a PEG with jejunal
  extension tube, by continuous infusion pump pts
  wear 16 hrs per day.
• Intended to stabilize dopamine levels and
  minimize motor fluctuations.

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ABBOTT PARKINSONS STUDY Contact Information

• Study Coordinator
• Emily.Houston_at_uvm.edu
• 802-656-3878
• Principal Investigator
• James.Boyd_at_uvm.edu