Local Research & Clinical Trials

Clinical trials have proven to be the fastest and most reliable way of finding effective new therapies for ALS. The goal of clinical trials is to answer specific scientific questions to find better ways to prevent, detect or treat ALS or improve care for people with ALS.

What is a clinical trial?

A clinical trial is a research study that follows a specific set of rules (protocol). This research includes using newer, promising treatments and the best methods currently available. People with ALS and their family members and caregivers are essential partners in this research. Clinical trials are often held in hospitals, clinics or doctor's offices, under the direction of researchers. Some studies are held at multiple locations. The research team will explain where you need to go and how often you will need to follow up.
 

How do clinical trials help?

Taking part in a clinical trial may help you and may also help improve treatment for patients in the future. Being part of a clinical trial may give you early access to new drugs and treatments. When people participate in a clinical trial, it may help us find new treatment methods sooner.
 

What is it like to be on a clinical trial?

You will be given detailed information about the clinical trial by a member of the health care team, including why the trial is being done and what to expect. You and your family will have the opportunity to discuss your needs and have your questions answered.

It is important for you to follow the clinical trial protocol to have accurate results. The protocol will describe:

    • Exactly how the clinical trial will be done
    • What treatment is used
    • How the treatment is given
    • How often you will need follow up visits to evaluate the treatment


Who pays for a clinical trial?

Generally, there are no costs associated with participation in a clinical trial. You and your caregiver may also receive a stipend for your time. The stipend varies by trial so be sure to ask the Study Coordinator whether or not the trial you are participating in provides a stipend.


What if I decide I want to quit being on a clinical trial?

If you decide that you no longer want to take part in a clinical trial, you can stop at any time. If a person leaves a trial, they should talk to their doctor about other treatment options.


Can I participate in a clinical trial in one medical facility and attend an ALS multidisciplinary clinic at another? 

Yes. You do not have to attend clinic at the location where a clinical trial is taking place. This provides flexibility for you so that if you qualify for a trial in anothero location, you do not have to change where you are being seen for your multidisciplinary clinic visits - you can participate in both places - one location/provider for your multidisciplinary care and one location/provider for your clinical trial.


How do I find information on a clinical trial?

Sign up for the National ALS Registry, take the surveys and check the box that you would like to be notified of trials for which you may qualify.

    • There are additional ways you can obtain information on clinical trials. Talk with a member of your health care team if you are interested in participating in a clinical trial. If your situation matches with a clinical trial that is currently available, you may be eligible to take part.
    • ALS ATLAS study- SOD1 variant research


UC SAN DIEGO CLINICAL TRIALS

VITALITY ALS Study

  • Summary: The primary objective of this study is to assess the effect of tirasemtiv versus placebo on respiratory function in patients with ALS.
  • Length: The study lasts approximately 14 months
  • Eligibility: ALS diagnosis, familial or sporadic. Other criteria for eligibility will be assessed at a screening visit

CReATe Study

  • Summary: This study is aimed at identifying the link between phenotype (observable signs and symptoms) and genotype (your genetic information) for amyotrophic lateral sclerosis (ALS), as well as other related neurodegenerative diseases, including frontal temporal dementia (FTD), primary lateral sclerosis (PLS), hereditary spastic paraplegia (HSP), progressive muscular atrophy (PMA) and multisystem proteinopathy (MSP). It also will investigate potential biomarkers (measurable indicators) of the disease. With this information, it is hoped that therapeutic developments will progress.
  • Length: This is a longitudinal study that requires in-person visits for the first year. After the first year, there will be annual remote visits that can take place via phone. These remote visits will take place for as long as you are willing and physically able to complete them.
  • Eligibility: ALS or related-disorder diagnosis, familial or sporadic

C9ORF72 Wash U Study

  • Summary: The purpose of this study is to better understand a specific form of amyotrophic lateral sclerosis caused by a mutation (or abnormality) of the C9ORF72 gene. This mutation is the most common genetic cause of ALS and is present in 40% of patients with familial ALS and 5-10% of patients without a family history of ALS. We hope that the intense study of patients with the C9ORF72 mutation through behavioral assessments and bio specimen collection will help us develop treatments for this common form of ALS.
  • Length: This study will last approximately 4 years, with a total of 7 clinic visits.
  • Eligibility:
    • Known positive C9ORF72 ALS status upon enrollment, familial or sporadic
    • Caregivers can also participate