New NEALS
October 2012                                                   Issue 5
ALS Clinical Research Update 

 

People with ALS and caregivers are important to the clinical research process. NEALS is committed to providing accurate, up-to-date information for all stakeholders in this process.    

News and Announcements
 

Cytokinetics Phase IIB Trial of Tirasemtiv (Formerly CK-2017357) to Begin in Late 2012 

Sponsored by Cytokinetics, Tirasemtiv (formerly CK-2017357) will be evaluated for safety, tolerability, and preliminary efficacy in approximately 400 people with ALS in an upcoming randomized, double-blind, placebo-controlled trial phase IIB. Participants in the trial will be randomly assigned to one of two groups. Those in the treatment group will receive twice-daily doses of tirasemtiv for three months, while those in the control group will receive the placebo. Participants who are randomized to receive tirasemtiv and who are taking rilutek at the time of enrollment will take riluzole at a daily dose of 50 milligrams for the duration of the study (3 months). Outcome measures are changes in motor function using the ALS Functional Rating Scale-Revised (ALSFRS-R) and measurements of pulmonary, respiratory and skeletal muscle function.

  

This is the fourth phase II trial of tirasemtiv. The initial trials were small safety and dose-finding studies; this is the first trial to systematically test whether tirasemtiv is effective in preserving function in patients with ALS.  Research sites will be posted to the NEALS website when available.

  

The NeuRx� Diaphragm Pacing System™ (DPS) in Participants with ALS Trial
Enrollment for this trial is not yet open.  People with ALS over the age of 21 years with some degree of breathing muscle weakness are eligible for participation in this multi-center, randomized controlled study.  People with ALS eligible to participate will have a 2/3 chance of being randomized to receive the DPS device by surgical implantation during a brief hospital stay.  Those people with ALS not randomized to receive DPS will still undergo the same intensive testing of breathing muscle function which may result in earlier detection of breathing symptoms.  Those patients with ALS participating in the research study will have approximately six study visits and otherwise will receive monthly phone calls for the duration of his or her study participation.  The average duration of study participation is estimated to be 18 months.  When site information is available, it will be posted to the NEALS website.

NeuralStem Stem Cell Trial has been Completed 
Neuralstem's Phase I trial, designed to determine safety of human spinal cord stem cells transplantation into ALS patients, has been completed.  Published interim results from the first 12 patients showed that spinal cord stem cells can be delivered safely into the lumbar region of the spine, or lower back.  Patients 13-15 received injections into the cervical (neck) spinal cord, and the final 3 patients received injections in both the lumbar (lower back) and cervical (neck) regions of the spinal cord.  Final safety results  are expected early next year.  The next step is to increase the dose of cells into the cervical spinal cord and determine if this intervention will slow or stop the progression of disease.


The Phase II Clinical Trial of Gilenya
The ALS Therapy Development Institute has made substantial progress toward launching a Phase II clinical trial of Novartis' MS-drug Gilenya� in ALS patients.  This includes preparing an application to the FDA as well as separately securing funding for the trial since announcing its intent to run this trial in February 2012.

ALS Association/NEALS PALS Webinars Series
NEALS offers live, online seminars (or "webinars") for people with ALS and other motor neuron diseases.  Experts explain the scientific rational and design of current ALS clinical research. Our webinars also offer participants the opportunity to ask questions directly to the experts running trials. Below is the information for our upcoming
ALS Association/NEALS Patient Webinar Schedule:

ALS Clinical Trials: Expediting Therapy Development 
Friday November 30, 2012 3pm-4pm ET
Merit Cudkowicz, MD, MSc
(Massachusetts General Hospital)
Registration information to come!

US Brainstorm Trial Statement   
We have had many inquires regarding the Brainstorm Stem Cell Therapeutics Clinical Trial. We are working with Brainstorm on a planned Phase II study in the US. The current trial is taking place at the Hadassah Medical Organization in Israel.  We hope to begin the trial in the first half of 2013.  We will continue to follow the progress of the study in Israel and will keep the NEALS website current on all new trials and start dates.  Thank you for your interest. 
Enrolling ALS Clinical Trials 

Phase II Trial

Phase II High Fat/High Calorie Trial
A phase II nutritional diet trial looking at safety, tolerability, and preliminary efficacy.  The trial is investigating the ideal amount and type of calories to be consumed by ALS patients daily.  Participants must be currently tolerating tube feedings through the use of either a g-tube or j-tube.  Participants will be randomly assigned to consume one of three diets: high fat and high calorie, high calorie, or normal diet.  The trial consists of five in-person clinic visits and four telephone visits over five months: four months of tube feeds with an additional month for follow-up.  This trial is currently enrolling at 12 sites throughout the United States.

Familial ALS Trial  
Arimoclomol in SOD1 Positive Familial ALS
An amplifier of heat-shock protein, Arimoclomol
is being investigated in a phase II/III randomized, double-blind, placebo-controlled trial in patients with SOD1 positive familial ALS. Researchers are assessing safety, tolerability, and efficacy of Arimoclomol and hypothesize that it will slow disease progression.  This trial is currently enrolling 80 participants at two sites: the University of Miami and Massachusetts General Hospital.   

Observational Studies
A Multicenter Study for the Discovery and Validation of Biomarkers in ALS

This biomarkers study is recruiting participants at NEALS sites in Boston MA, Worcester MA, Pittsburgh PA, Atlanta GA, Jacksonville FL, and Phoenix AZ.  Blood samples and cerebrospinal fluid (CSF) from patients with ALS will be collected. Through comparison of these samples, the researchers will learn more about the underlying cause of ALS, as well as find unique biological markers, which could be used to diagnose ALS more quickly and develop new therapies.  

Interventional Trial

Resistance and Endurance Exercise in ALS

How can exercise be combined with other treatments? Standard of care has been considered to be stretching and range of motion exercises; there has been little, if any, research into other forms of exercise in ALS patients.  In this pilot study, 60 participants with ALS will be assigned to treatment in 1 of 3 arms:  resistance exercise, endurance exercise, or stretching and range of motion exercise.   Researchers are looking to learn about which is safe and tolerable for patients with ALS.  The trial will enroll at four locations: Johns Hopkins University, Massachusetts General Hospital, Carolinas Medical Center, and Washington University in St. Louis, Missouri.

For a full list of enrolling trials, go here
Enrollment Closures  

Selection Trial of High Dosage Creatine and Two Dosages of Tamoxifen

The Phase II Selection Trial of High Dosage Creatine and Two Dosages of Tamoxifen has been closed to enrollment. Sixty subjects have been enrolled at 9 NEALS sites across the US.  Each participant will take one active study drug (creatine 30gm, tamoxifen 40mg, or tamoxifen 80mg) and one placebo for 38 weeks.  This trial has a unique design which allows selecting the most promising treatment to be tested in a future larger trial.  Most participants have completed their study participation and the last participant visit will take place in December 2013, with trial results in expected in spring 2013.

For more information on the Selection Trial, click here. Questions can be directed to Julie Berkley at [email protected] or 617-643-7429.
Primary Lateral Sclerosis Research  

The NEALS Upper Motor Neuron Task Force has Recently Launched the UMN Pilot Registry
The goal of this Registry is to identify cases of Primary Lateral Sclerosis (PLS) and Early PLS within the NEALS Network. This project will conduct a retrospective chart review of cases of UMN disease seen at NEALS clinics. The UMN Pilot Registry will establish the foundation for future PLS natural history and biomarkers studies, and PLS clinical trials. Several NEALS sites have joined the registry and are working with their Institutional Review Boards (IRB) to collect data.

PLS COSMOS Study Investigates Oxidative Stress in People with PLS

A research project will investigate patients with clinically definite Primary Lateral Sclerosis (PLS), that is, the disease has been present for more than 5 years and less than 15 years, and there is no evidence of lower motor dysfunction (no EMG abnormalities).  The goal is to uncover the disease mechanisms and determine if oxidative stress (excessive  
free radicals) is associated with the disease and its progression.  Researchers will study biomarkers of oxidative stress and oxidative stress from environmental, occupational, lifestyle, diet and psychological factors.  This knowledge can be used to improve understanding of not only PLS but ALS as well.  The grant is funded by the Spastic Paraplegia Foundation, involving the National Institute of Neurological Diseases and Stroke, Western University, London, Ontario, University of Kansas, University of Texas Southwestern, and Columbia University.  This is a counterpart of the NIH-funded ALS COSMOS study (no longer enrolling), which allows us to study ALS in a very similar way.  Enrollment will continue through at least 3/31/2013 (and could be extended further).
NEALS is Looking Forward to the ALS Clinical Research Learning Institute  

The Clinical Research Learning Institute, sponsored by the ALS Association's TREAT ALS initiative, is an intensive program dedicated to educate 10 patient-caregiver pairs on clinical research and to empower them to be more effective research advocates...  Read more on the NEALS blog.
The Exercise Trial in ALS Webinar has been Archived

If you missed Dr. Nicholas Margakis' "Resistance and Endurance Exercise in ALS Webinar" presentation, or if you would like to listen again, it has been archived for viewing.

Watch it here.


To view the archive of all other ALS Association/NEALS PALS Webinars, please go here.
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In This Issue
News and Announcements
Enrolling ALS Trials
Enrollment Closures
Primary Lateral Sclerosis Research
Clinical Research Learning Institute
Exercise Trial Webinar
Researcher Highlight
Trial Expert
Researcher Highlight
Timothy Miller, MD
 Timothy Miller, MD, PhD, serves as Assistant Professor of Neurology at Washington University in St. Louis; Faculty Member of the Hope Center for Neurological Disorders; and Director of the Christopher Wells Hobler Laboratory for ALS Research.

Timothy Miller's, MD, PhD interest in exploring neurodegenerative disease began as a graduate student studying the basic mechanisms of cell death. During his residency and fellowship under the direction and guidance of...
to read more.

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ALS Association/ NEALS Trial Expert 

 

Devon Punch is the "ALS Association/NEALS Clinical Trial Expert." She is available to help people with motor neuron disease and their caregivers navigate the NEALS website and answer questions about clinical research during normal business hours eastern standard time.

 ALS Trial Expert
(877) 458-0631
[email protected]
Sincerely,
The Northeast ALS Consortium
ALS Trial Expert
(877) 458-0631