Opportunities for People with ALS to Participate in Research
July 1, 2004
There are several ALS studies in process that are actively recruiting people with ALS for enrollment. Participation in a clinical trial is a tangible way that people with ALS can contribute to the scientific knowledge about ALS and help identify ALS treatments that are safe and effective for people living with ALS.
Please review the brief summaries below and visit ALSA’s web site for more information about these trials and other ALS studies. http://www.alsa.org/patient/drug.cfm?
Insulin-like growth factor-1 (IGF-1)
The objective of this trial is to determine whether IGF-1 (trade name MyotrophinTM) slows progression of weakness in amyotrophic lateral sclerosis (ALS). Three hundred patients with ALS will participate in a double blind, placebo-controlled two-year study. Half the patients will receive IGF-1 and the other half will receive a placebo. The drug will be administered twice a day subcutaneously. The primary endpoint will be the rate of change in manual muscle testing score (MMT). Secondary endpoints will include tracheostomy free survival and change in ALS Functional Rating Score (ALSFRS).
Enrollment began in the summer of 2003. Patients visit one of the participating centers every six months for testing and can take other medications as long as they are not enrolled in another study. This clinical trial is funded by the National Institute of Health’s National Institute of Neurological Diseases and Stroke (NINDS) and The ALS Association.
For more information on this study and the participating trial site nearest you, refer to the list of study site at http://www.alsa.org/patient/drug.cfm?id=52.
Minocycline
A NINDS-supported Phase III, 13-month clinical trial of minocycline began enrolling people living with ALS in November 2003. Minocycline has been tested and has been shown to protect motor neurons in scientific experiments.
This multi-center, randomized, double blind trial will compare minocycline to placebo for efficacy, tolerability and safety. Researchers hope to demonstrate that minocycline slows disease progression in people living with ALS.
At the study’s completion, the minocycline trial will be one of the largest investigator-initiated clinical trials conducted to date in ALS (400 patients); it will provide data not only on the effectiveness of specific outcome measures, but also on the course of ALS.
Each participant will have monthly outpatient evaluations for 13 months, measuring any changes in functions, such as strength, pulmonary function, survival and quality of life.
For more information on this study and the participating trial site nearest you, contact Jackie Montes by phone (212) 305-3632 or by e-mail ALSCenter@columbia.edu.
Neurodex – also known as AVP-923
Pseudobulbar affect manifests in inappropriate and uncontrollable emotional outbursts and can affect some ALS patients. Avanir Pharmaceuticals is conducting an open label trial of Neurodex for patients experiencing pseudobulbar affect, or PBA.
This open-label clinical trial is testing the combination of two well-known drugs, Dextromethorphan and Quinidine, for the treatment of PBA. Open-label trials such as this allow for each qualified participant to receive the active investigational therapy without using a placebo group. Only four visits to the study physician are required; the remaining follow-up is done by periodic phone interviews.
Prior research showed that use of Neurodex decreased the severity and frequency of pseudobulbar episodes and improved quality of life.
The open label study is a multi-center study assessing the long-term safety of Neurodex over one year. Possible side effects include nausea, sleepiness, fatigue, dizziness, euphoria, confusion, rash or diarrhea.
This study is being conducted at multiple sites throughout the U.S. If you think you have pseudobulbar affect and are interested in participating in this important clinical trial, please call (800) 669-0281, or visit www.pseudobulbar.com.
Biomarker study
Researchers who are working to identify biomarkers for ALS need more people with familial ALS to participate in this important study.
Biomarkers are substances in the blood and/or cerebral spinal fluid (CSF) that are present in people with a specific disease and are not present in anyone who does not have the disease. A small study identified proteins in the CSF of people who had ALS. More information about this initial study can be found on ALSA’s website:www.alsa.org/news/article.cfm?id=222
Currently, a second study is underway that is examining the blood and/or CSF of a larger number of people with ALS in order to, hopefully, replicate the findings from the initial research. There are now sufficient samples from people with sporadic ALS for this study; however, more samples from people with familialALSareneeded.
While this study is not a treatment trial and is not expected to change the course of ALS in the people who participate in the study, participants will be able to contribute to the growing knowledge about what causes ALS and how to benefit people who have the disease. Biomarkers hold important potential for ALS and, if this study can confirm the identity of one or more biomarkers for ALS, there would be many future benefits. Among the benefits could be a test for ALS, possibly a tool for physicians to make an earlier diagnosis of this disease, and a more objective way to measure the effects of drugs and other potential treatments.
If you have familial (inherited) ALS and would like more information in how to participate in the ALS biomarker study, please contact Kristyn Newhall at knewhall@partners.org or Dr. Merit Cudkowicz at mcudkowicz@partners.org. The investigators are located in Boston and Pittsburgh; arrangements can be made to collect samples from patients who live close to these sites but are unable to travel. This study is funded by The ALS Association.
Gene and Risk Factor Study
Northwestern University is collecting material for a Registry of Neurologic Diseases, particularly ALS and other motor neuron diseases, both sporadic and familial. Participation requires a one time blood sample from patients, as well as completion of several questionnaires about family history, environmental exposures, and release of medical records. These materials are being studied to identify genes and environmental factors that increase risk for developing ALS and related diseases in order to understand how ALS and related disorders occur so that eventually rational treatments or prevention can be devised. Participation does not require patient travel. For more information contact Nailah Siddique RN MSN at (312) 503-2712, e-mail nsiddique@northwestern.edu or Lisa Dellefave, MS CGC at (312) 503-0154, e-mail l-dellefave@northwestern.edu.
The ALS Association, July 1, 2004
