ONGOING CLINICAL TRIALS
University of Vermont
Presented by: Rup Tandan, MD, FRCP
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
ALS RESEARCH GROUP
DNA 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.
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
CONTACT INFORMATION for
Dr. Tandan’s Studies
Rup Tandan, MD - Site PI
(802) 847-4589
Rup.Tandan@vtmednet.org
Study Coordinator for Nutrition Study and Thymectomy
Trial
(802) 656-4582
Shannon.Lucy@med.uvm.edu
A MULTICENTER, DOUBLE-BLIND, PARALLEL GROUP,
PLACEBO CONTROLLED STUDY OF CREATINE IN
SUBJECTS WITH TREATED PARKINSON’S 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.
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.
A MULTICENTER, DOUBLE-BLIND, PLACEBO
CONTROLLED, PHASE II STUDY OF PIOGLITAZONE IN
EARLY PARKINSON’S 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.
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
CONTACT INFORMATION
Robert Hamill, MD - Site PI
(802) 847-4589
Robert.Hamill@vtmednet.org
Study Coordinator:
(802) 656-3878
Emily.houston@uvm.edu
EPILEPSY CLINICAL TRIALS
CLOSED 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.
THE MS CENTER
OF 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
ONGOING CLINICAL TRIALS IN MS
CLOSED 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 β-1a) in patients
with relapsing-remitting MS.
ADDITIONAL CLINICAL TRIALS IN MS
CLOSED 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, placebocontrolled, parallel-group study to evaluate efficacy and safety of
teriflunomide in patients with a first clinical episode suggestive of
MS.
ONGOING CLINICAL TRIALS IN MS
OPEN 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
STROKE/NEUROCRITICAL CARE
IRIS
(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
IRIS STUDY Contact Information
Study Coordinator:
Catherine.Gregory@med.uvm.edu
802-656-8993
Principal Investigator:
mgorman1@uvm.edu
ABBOTT PARKINSON’S STUDY
PI: James Boyd, MD
 Study of levodopa/carbidopa intestinal gel vs. oral
levodopa/carbidopa
 For pts with advanced Parkinson’s, 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 Parkinson’s
are not eligible
ABBOTT PARKINSON’S 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.
ABBOTT PARKINSON’S STUDY
Contact Information
Study Coordinator:
Emily.Houston@uvm.edu
802-656-3878
Principal Investigator:
James.Boyd@uvm.edu