Amyotrophic Lateral SclerosisNCT01495390
A Longitudinal Study of Amyotrophic Lateral Sclerosis (ALS) Biomarkers
An observational study of Amyotrophic Lateral Sclerosis and Nervous System Diseases, run by Massachusetts General Hospital at 6 sites in United States.
Trial summary
The purpose of this study is to collect biofluid samples for the banking and usage in ALS research. Through comparison of these samples, the researchers hope to learn more about the underlying cause of ALS, as well as find unique biological markers, which could be used to develop new therapies. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyotrophic Lateral Sclerosis, Nervous System Diseases, Sclerosis
- How many people
- 139 participants
- Where
- 6 sitesUnited States
- Study dates
- Nov 2011to 31 Dec 2016
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Volunteers will be invited to participate in this study by their neurologists either in clinic or at a regular scheduled appointment visit
Who may be able to join
- Age 18 or older
- Diagnosis of suspected, possible, probable or definite ALS according to El Escorial Criteria
- Vital capacity (VC) at least 50 percent predicted
- Able to undergo multiple lumbar punctures
Who cannot join
- Abnormal CSF pressure or intracranial/intraspinal tumors
- Use of anticoagulant medication that cannot be safely withheld
- Bleeding disorders
- This is a partial listing.
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
United States6 sites
Emory University
Atlanta, Georgia, 30322
Massachusetts General Hospital
Boston, Massachusetts, 02114
Mayo Clinic Jacksonville
Jacksonville, Florida, 32224
Barrow Neurological Institute
Phoenix, Arizona, 85013
University of Pittsburgh
Pittsburgh, Pennsylvania, 15213
University of Massachusetts Medical Center
Worcester, Massachusetts, 01655
About this study
Official title: A Multicenter Study for the Discovery and Validation of ALS Biomarkers
The purpose of this study is to collect biofluid samples for the banking and usage in ALS research. Through comparison of these samples, the researchers hope to learn more about the underlying cause of ALS, as well as find unique biological markers, which could be used to develop new therapies.
Read the detailed description
The purpose of the research study is to collect blood samples and cerebrospinal fluid (CSF) from people with amyotrophic lateral sclerosis (ALS). These samples will be collected approximately every 4 months.
- Conditions
- Amyotrophic Lateral Sclerosis, Nervous System Diseases, Sclerosis, Motor Neuron Disease, Spinal Cord Diseases, Central Nervous System Diseases
- Keywords
- Biomarkers, Biological Markers, Surrogate Markers, Clinical Markers, Plasma, Cerebrospinal Fluid, DNA, Serum, RNA
- Medical subject headings
- Amyotrophic Lateral Sclerosis, Nervous System Diseases, Sclerosis, Motor Neuron Disease, Spinal Cord Diseases, Central Nervous System Diseases, Neurodegenerative Diseases, Neuromuscular Diseases
What is being measured
The primary outcome is the main question the study is designed to answer.
ALS Functional Rating Scale
The ALSFRS-R is a quickly administered (5 min) ordinal rating scale used to determine a subject's assessment of their capability and independence in 12 functional activities. There are 12 questions, graded by the subject 0-4 (4 is normal). Score of 0 (worst) to 48 (best). Reflects speech and swallowing, fine motor skills, large motor skills, and breathing.
Measured: Approximately every 4 months
Vital Capacity (VC)
The vital capacity (VC) (percent of predicted normal) was determined using the slow VC method. Vital Capacity is the maximum amount of air a person can expel from the lungs after a maximum inhalation. A subject's VC depends on their age, sex and height. The value is recorded as a percent of predicted normal.
Measured: Approximately every 4 months
Hand Held Dynamometry (HHD)
Hand Held Dynamometry (HHD) will be used as a quantitative measure of muscle strength for this study. Six proximal muscle groups will be examined bilaterally in both upper and lower extremities (shoulder flexion, elbow flexion, elbow extension, hip flexion, knee flexion, and knee extension), all of which have been validated against maximum voluntary isometric contraction (MVIC) testing.
Measured: Approximately every 4 months
Ashworth Spasticity Scale
This is a standard measure for spasticity, has been used in a previous ALS clinical trials, and is applied in the current trial to evaluate the progression of spasticity due to upper motor neuron dysfunction in ALS.
Measured: Approximately every 4 months
Fronto-Temporal Dementia (FTD) Assessment
The FTD Screening Assessment is a quickly administered scale used to evaluate memory, executive functions, and language. It is aimed at determining the presence of subtle dysfunction of these domains of cognition and behavior that may portend the onset of FTD or FTD-like symptoms.
Measured: Approximately every 4 months
ALS Cognitive Behavioral Screen (ALS CBS)
The ALS Cognitive Behavioral Screen (ALS-CBS™) is a short measure of cognition and behavior in patients with Amyotrophic Lateral Sclerosis (ALS). The cognitive section includes commonly used elements of standard testing batteries, consisting of 8 tasks, with a possible total score of 20. It can be administered by a physician or other clinical care staff and takes approximately 5 minutes to complete.
The behavioral section (ALS Caregiver Behavioral Questionnaire) is composed of questions sensitive to organic brain changes. It consists of a set of questions that compare changes in personality and behavior since the onset of ALS, as well as yes/no questions about mood, pseudobulbar affect, and fatigue. It is completed by a caregiver, family member or other informant during the same time that the patient completes the cognitive portion. The questionnaire typically takes about 2 minutes to complete.
Measured: Approximately every 4 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Dec 2011 (estimated)
- Study startNov 2011
- Primary completion31 Dec 2016
- Study completion31 Dec 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- More trials run by Massachusetts General HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 139 participants (actual)
Sponsor and investigators
- Lead sponsor
- Massachusetts General Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- ALS Association, ALS Finding a Cure
- Responsible party
- Principal investigator, James D. Berry MD, Massachusetts General Hospital
- Investigators
- James D. Berry, MD, MPH, Principal investigator, Massachusetts General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01495390
- Sponsor's protocol ID
- BIO-ALS-02
- Status last verified
- May 2017
- First submitted
- 16 Dec 2011
- Last updated
- 30 May 2017
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 30 May 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter Study for the Discovery and Validation of ALS Biomarkers
- ClinicalTrials.gov ID
- NCT01495390
- Acronym / sponsor ID
- BIO-ALS-02
- Conditions
- Amyotrophic Lateral Sclerosis, Nervous System Diseases, Sclerosis, Motor Neuron Disease, Spinal Cord Diseases, Central Nervous System Diseases
- Keywords
- Biomarkers, Biological Markers, Surrogate Markers, Clinical Markers, Plasma, Cerebrospinal Fluid, DNA, Serum
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 139 participants (actual)
- Lead sponsor
- Massachusetts General HospitalUniversity, hospital or other
- Collaborators
- ALS Association, ALS Finding a Cure
- Study start
- Nov 2011
- Primary completion
- 31 Dec 2016 (actual)
- Study completion
- 31 Dec 2016 (actual)
- First posted
- 20 Dec 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to collect biofluid samples for the banking and usage in ALS research. Through comparison of these samples, the researchers hope to learn more about the underlying cause of ALS, as well as find unique biological markers, which could be used to develop new therapies.
Detailed description
The purpose of the research study is to collect blood samples and cerebrospinal fluid (CSF) from people with amyotrophic lateral sclerosis (ALS). These samples will be collected approximately every 4 months.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Volunteers will be invited to participate in this study by their neurologists either in clinic or at a regular scheduled appointment visit
Key inclusion criteria (4 listed)
- Age 18 or older
- Diagnosis of suspected, possible, probable or definite ALS according to El Escorial Criteria
- Vital capacity (VC) at least 50 percent predicted
- Able to undergo multiple lumbar punctures
Key exclusion criteria (4 listed)
- Abnormal CSF pressure or intracranial/intraspinal tumors
- Use of anticoagulant medication that cannot be safely withheld
- Bleeding disorders
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
ALS Functional Rating Scale
Time frame: Approximately every 4 months
The ALSFRS-R is a quickly administered (5 min) ordinal rating scale used to determine a subject's assessment of their capability and independence in 12 functional activities. There are 12 questions, graded by the subject 0-4 (4 is normal). Score of 0 (worst) to 48 (best). Reflects speech and swallowing, fine motor skills, large motor skills, and breathing.
Vital Capacity (VC)
Time frame: Approximately every 4 months
The vital capacity (VC) (percent of predicted normal) was determined using the slow VC method. Vital Capacity is the maximum amount of air a person can expel from the lungs after a maximum inhalation. A subject's VC depends on their age, sex and height. The value is recorded as a percent of predicted normal.
Hand Held Dynamometry (HHD)
Time frame: Approximately every 4 months
Hand Held Dynamometry (HHD) will be used as a quantitative measure of muscle strength for this study. Six proximal muscle groups will be examined bilaterally in both upper and lower extremities (shoulder flexion, elbow flexion, elbow extension, hip flexion, knee flexion, and knee extension), all of which have been validated against maximum voluntary isometric contraction (MVIC) testing.
Ashworth Spasticity Scale
Time frame: Approximately every 4 months
This is a standard measure for spasticity, has been used in a previous ALS clinical trials, and is applied in the current trial to evaluate the progression of spasticity due to upper motor neuron dysfunction in ALS.
Fronto-Temporal Dementia (FTD) Assessment
Time frame: Approximately every 4 months
The FTD Screening Assessment is a quickly administered scale used to evaluate memory, executive functions, and language. It is aimed at determining the presence of subtle dysfunction of these domains of cognition and behavior that may portend the onset of FTD or FTD-like symptoms.
ALS Cognitive Behavioral Screen (ALS CBS)
Time frame: Approximately every 4 months
The ALS Cognitive Behavioral Screen (ALS-CBS™) is a short measure of cognition and behavior in patients with Amyotrophic Lateral Sclerosis (ALS). The cognitive section includes commonly used elements of standard testing batteries, consisting of 8 tasks, with a possible total score of 20. It can be administered by a physician or other clinical care staff and takes approximately 5 minutes to complete. The behavioral section (ALS Caregiver Behavioral Questionnaire) is composed of questions sensitive to organic brain changes. It consists of a set of questions that compare changes in personality and behavior since the onset of ALS, as well as yes/no questions about mood, pseudobulbar affect, and fatigue. It is completed by a caregiver, family member or other informant during the same time that the patient completes the cognitive portion. The questionnaire typically takes about 2 minutes to complete.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 May 2017.
- Northeast ALS Consortium WebsiteLink provided by the sponsor.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
James D. Berry, MD, MPH principal investigator
Massachusetts General Hospital
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Amyotrophic Lateral Sclerosis and Nervous System Diseases, run by Massachusetts General Hospital at 6 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 30 May 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
ALS Functional Rating Scale (time frame: Approximately every 4 months). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 May 2017.