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ALS Research
Overview
The Mass General NCTU was co-founded in 1994 by Dr. Merit Cudkowicz and Dr. Steven Greenberg to conduct clinical research in ALS and other neurological disorders.
Dr. Cudkowicz, Co- Director of the Mass General Neurology Clinical Trials Unit manages this large clinical trials research department dedicated to finding new treatments for people with ALS.
Our staff is also dedicated to finding new treatments for ALS. Our team leads and manages clinical trials at major medical institutions throughout the United States and Canada.
The Mass General NCTU can give interested individuals with ALS access to participate in clinical trials of medications and therapies designed to lead to better treatments.
Northeast ALS Consortium (NEALS)
Dr. Cudkowicz also co-founded and co-directs the Northeast ALS Consortium (NEALS), which coordinates a number of clinical trials with more than 74 research centers throughout the United States and Canada.
The NEALS Consortium was formed with the goal of bringing together leading ALS scientists and clinicians caring for ALS patients, to offer advice on the selection of the best therapies and direction of ALS research. Today there are 81 medical sites and more than 125 members.
The Mass General NCTU and NEALS are currently working on clinical trials of Ceftriaxone, ISIS 333611, KNS760704 and Arimoclomol.
In clinical trials involving ALS where the disease is inherited in families, Arimoclomol is also being looked at along with a chemical compound called Antisense Oligonucleotide. Studies looking at ALS biomarkers may help to predict and prevent familial ALS as these genetic studies explore the association between the family history of genetic make-up. The Mass General NCTU and NEALS are also planning studies on how exercise and nutrition affects the disease.
Merit Cudkowicz, MD, NCTU Co-Director
Massachusetts General Hospital
617-726-9122
mcudkowicz@partners.org
Swati Aggarwal, MD
Massachusetts General Hospital
617-724-3914
spaggarwal@partners.org
Nazem Atassi, MD
Massachusetts General Hospital
617-643-6114
natassi@partners.org
Anne-Marie Wills, MD
Massachusetts General Hospital
617-726-9122
awills@partners.org
Patricia Andres, MS, DPT
Massachusetts General Hospital
617-724-8995
pandres1@partners.org
Matt Bellanich, NP
617-726-8741
mbellanich@partners.org
Marianne Kearney
Director of Project Management
617-726-5893
mkearney4@Partners.org
Francine Murphy
Office/Program Manager
617-726-9122
fmurphy@partners.org
Darlene Pulley, RN
Research Nurse Manager
617-726-6190
dpulley@partners.org
Alex Sherman
Director of Systems
617-643-2935
avsherman@partners.org
Bryan Sweet
Director of Grants Management
617-724-3930
bsweet@partners.org
Hong Yu, MPH
Director of Data Management
617-726-5591
hyu2@partners.org
Active ALS Clinical Trials in the NCTU at Mass General
Arimoclomol in SOD1 Positive Familial ALS Clinical Trial
Principal Investigators
Michael Benatar
Emory University, Atlanta, GA
Merit Cudkowicz, MD
Massachusetts General Hospital
This is a randomized, placebo-controlled trial of Arimoclomol in people with SOD1 positive ALS. This is a unique study in which all participants will travel to one of two sites, Emory or the Massachusetts General Hospital, for a baseline and month 2 visit, and all subsequent visits will be made in the participant’s home by NEALS certified clinical evaluators and coordinators who are located near the participant’s home. The study is now open for enrolment.
Phase I Clinical Safety Trial of ISIS 333611 in SOD1 Familial ALS
Principal Investigators
Timothy Miller, MD
Washington University, St. Louis, MOMerit Cudkowicz, MD
Massachusetts General HospitalThis study will recruit between 20 and 40 people with familial ALS with SOD1 mutations associated with rapid progression. Escalating dosages of ISIS 333611 delivered intrathecally via an implanted pump will be tested for safety. Six NEALS clinical sites will participate in this study.
NEALS Data management, clinical trial project management, outcomes supervision, and study monitoring and performance are all components for this trial. The Electronic Data Capture and Clinical Trials Management Systems developed for the TREAT NEALS ALS Platform will be utilized for this study.
Dr. Miller is being mentored by Drs. Cudkowicz as part of the NEALS New Investigator Training program. The training program includes frequent communications and meetings with Drs. Cudkowicz regarding: protocol development, site selection and training, funding and budgeting, and resource utilization.
Enrolment is expected in the 4th quarter of 2009.
A Multi-Center Study for the Validation of ALS Biomarkers
Principal Investigator
Merit Cudkowicz, MD
Massachusetts General HospitalSix hundred volunteers, including 300 with ALS, 250 with other neuromuscular diseases and 50 healthy individuals, are participating in this study. Twenty one NEALS ALS Centers across the U.S. are participating. The principal aim for this study is to contribute samples of blood and cerebrospinal fluid to the NEALS ALS Sample Repository located at the Massachusetts General Hospital in Boston, MA. A protocol for sample collection, processing, shipment and storage has been established. The secondary aim of the study is to provide samples to Metabolon, Inc. to validate a diagnostic test for ALS.
All aspects of the NEALS infrastructure are utilized to support this trial. The Electronic Data Capture and Clinical Trials Management Systems developed at Mass General are an essential part of this trial and have contributed greatly to the speed of study start up. An electronic system to track and manage biological samples has been built and validated for this project. This can also be used to track biological samples collected in other NEALS clinical studies.
An Open-Label, Safety and Tolerability Study Evaluating KNS-760704 in Patients with Amyotrophic Lateral Sclerosis (ALS)
Principal Investigator
Merit Cudkowicz, MD
Massachusetts General HospitalSponsor Knopp Neuroscience, Inc.
This is an open-label, multi-center study designed to evaluate the safety, tolerability, and effects on functional outcome measures of oral administration of KNS760704 for up to 48 weeks in patients with ALS. Eligible subjects will have been actively participating in a previous study (KNS-760704-CL201).
Knopp Neuroscience, Inc., the Sponsor, has awarded a contract to Dr. Cudkowicz and the Mass General Neurology Clinical Trial unit for assistance with Project Management, Grant Management and Data Management. Knopp Neuroscience has asked Dr. Cudkowicz to also lead the planned phase 3 efficacy study of KNS760704.
Clinical Trial of Ceftriaxone in Subjects with ALS
Principal Investigator
Merit Cudkowicz, MD
Massachusetts General HospitalThe goals of this study are to evaluate the safety and effectiveness of ceftriaxone as a treatment for ALS, and to determine the safety and effectiveness of long-term use of the drug in people with ALS. A total of 600 eligible people with ALS will be enrolled in this multi-center research study. Participants will be randomly assigned to receive treatment with ceftriaxone (2/3 of participants) or placebo (1/3 of participants) for at least 12 months. The study consists of three stages. The first stage, which has completed enrollment, will look at whether ceftriaxone enters the cerebrospinal fluid (the fluid that surrounds the spinal cord, also called CSF) in amounts that are high enough to be of possible benefit. The second stage, which has also completed enrollment, will look at the safety and side effects of the study drug when taken daily for at least 20 weeks. The third stage, which began enrollment in May 2009, will determine whether the study drug prolong survival and slows decline in function due to ALS.
The first two stages are complete. We found that ceftriaxone levels in the CSF were sufficient to warrant proceeding to next stage. We also found that ceftriaxone was safe and a dosage of 4 grams/day was chosen for the stage 3 efficacy study. Approximately 57 sites in the United States and Canada will enroll the remaining 534 participants. The study is funded by the National Institute of Health.
Validation of a New Device to Measure Neuromuscular Disease Progression
Principal Investigator
Patricia Andres, MS, DPT
Massachusetts General HospitalThis study evaluates a new device that measures isometric strength called: Accurate Test of Limb Isometric Strength (ATLIS). This device was designed to be portable, quick, and easy to use, while generating accurate and reliable, interval level data. The purposes of this study are to validate the ATLIS device and to develop a normal databank. The specific aims are to: 1) determine the reliability of the ATLIS device by testing each participate two separate times.; 2) determine the validity of the device by comparing it to the Tufts Quantitative Neuromuscular Exam (TQNE); and 3) develop a normal databank containing tests from at least 500 adults, without physical disability, to be used to develop regression equations for converting data to a percentage of normal. In addition to MASS GENERAL, 4 other ALS clinical research centers around the country are participating in the normal data bank study. Aims one and two are complete and enrollment has started for aim 3. The study is supported by the MDA.
Trial of High fat/High Calorie Diet versus Optimal Nutrition in Amyotrophic Lateral Sclerosis
Principal Investigator
Anne-Marie Wills, MD
Massachusetts General HospitalThis is a phase II double-blind placebo-controlled clinical trial to study the safety, tolerability and feasibility of a high fat/high calorie diet versus high calorie diet versus normal diet. Secondary outcome measures include lipid levels, weight, body mass and composition and preliminary effects on disease progression, on the three diets. Twenty subjects from 5 ALS centers who are already receiving percutaneous nutrition will be randomized to each treatment arm and followed for four months. Energy needs for each subject will be calculated based on measured energy expenditure using indirect calorimetry and basal metabolic rate (BMR). The control diet will be treated with optimal calorie replacement while both intervention arms will be provided a high calorie diet 1.25 times their calculated energy needs. Subjects and the evaluating investigators will be blinded to treatment assignment. Primary outcome measures will be adverse events and compliance rates. Subjects will remain in the study 4 weeks after the end of the dietary intervention, allowing time to observe adverse events in respect to the primary endpoint of safety.
Neurology Clinical Trials NCTU
Charlestown Navy Yard, Building 149149 13th Street, 2nd Floor, Room 2274
Charlestown, MA 02129
Phone: 617-643-3980
Public Transportation Access: yes
Disabled Access: yes
E-mail Address
mghneuroclinicaltrialsunit@partners.org
Mailing Address
Neurology Clinical Trials Unit
Mailcode: CNY B149-2-2274
Massachusetts General Hospital
149 13th Street, 2nd Floor, Room 2274
Charlestown, MA 02129
Clinical Research Staff
Marianne Kearney
Clinical Research Program/Project Manager
Phone: 617-726-5893
E-mail: MKearney4@Partners.org
Francine Murphy
Office/Program Manager
617-726-9122
Darlene Pulley, RN
Research Nurse Manager
617-726-6190Patient Appointments & Clinical Office
ALS (Amyotrophic Lateral Sclerosis) Multidisciplinary Clinic
Phone: 617-724-3914
