Amyotrophic Lateral SclerosisBIO_ALS-01NCT00677768
Validation of Biomarkers in Amyotrophic Lateral Sclerosis (ALS)
An observational study of Amyotrophic Lateral Sclerosis and Lou Gehrig's Disease, run by Massachusetts General Hospital at 30 sites in 2 countries.
Trial summary
The purpose of this study is to collect 650 blood and 300 cerebrospinal fluid (CSF) samples from people with amyotrophic lateral sclerosis (ALS), pure lower or upper motor neuron diseases, as well as other neurodegenerative diseases and from people with no neurological disorder. Read more about this study
- Who is it for?
- Ages 30 to 80 yearsHealthy volunteers can take part
- Conditions the study is for
- Amyotrophic Lateral Sclerosis, Lou Gehrig's Disease, Primary Lateral Sclerosis
- How many people
- 475 participants
- Where
- 30 sitesin 2 countries
- Study dates
- Apr 2008to Nov 2015
- 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 30 to 80 years
- All sexes
- Healthy volunteers accepted
- 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
- Diagnosis of possible (excluding volunteers with UMN signs ONLY), probable, probable-laboratory supported, or definite ALS, either sporadic or familial according to revised El Escorial criteria
- Disease duration of less than or equal to two years from symptom onset
- Age 30-80 years at the time of disease onset
- Ability to provide informed consent
- Ability to comply with study procedures
- Medically safe to have lumbar puncture (lumbar puncture volunteers only)
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- Diagnosis of suspected ALS defined as presence of UMN or LMN signs alone and the diagnosis of Clinically Probably Laboratory-Supported ALS CANNOT be proven by evidence in clinical grounds in conjunction with electrodiagnostic, neurophysiologic, neuroimaging or clinically laboratory studies
- Disease duration of less than or equal to four years from symptom onset
- Age 30-80 years at time of disease onset
- Ability to provide informed consent
- Ability to comply with study procedures
- Medically safe to have lumbar puncture (lumbar puncture volunteers only)
- Diagnosis of one of the following:
- Pure Lower Motor Neuron Disease (LMND) mimics:
- Multi-focal motor neuropathy
- Autoimmune motor neuropathy
- Cervical or lumbosacral radiculopathies
- Peripheral mononeuropathies:
- Ulnar neuropathy
- Carpal tunnel syndrome/median neuropathy
- Peroneal neuropathy
- Sciatic neuropathy
- Spinal muscular atrophy
- Spinobulbar muscular atrophy (Kennedy's disease)
- Charcot Marie-Tooth Disease (CMT)
- Pure Upper Motor Neuron Disease (UMND) mimics:
- Cervical myelopathy
- Multiple sclerosis
- Hereditary spastic paraparesis
- Age 30-80 years
- Ability to provide informed consent
- Ability to comply with study procedures
- Medically safe to have lumbar puncture (lumbar puncture volunteers only)
Who cannot join
- Clinical evidence of chronic liver or renal failure
- Presence of a bleeding disorder, problems with CSF pressure, allergy to local anesthetics, or a topical or other skin infection at the LP site (lumbar puncture volunteers only)
- Use of any anti-platelet or anticoagulant drugs, such as plavix, aggrenox, ticlid, warfarin or coumadin (lumbar puncture volunteers only)
- Suspected ALS (PMND) Volunteers
- Clinical evidence of chronic liver or renal failure
- Genetically confirmed diagnosis of hereditary spastic paraparesis or spinal motor atrophy (SMA) disease
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- Presence of a bleeding disorder, problems with CSF pressure, allergy to local anesthetics, or a topical or other skin infection at the LP site (lumbar puncture volunteers only)
- Use of any anti-platelet or anticoagulant drugs, such as plavix, aggrenox, ticlid, warfarin or coumadin (lumbar puncture volunteers only)
- Neurological Disease Mimic Volunteers
- Diagnosis of suspected, possible, probable or definite ALS either sporadic or familial
- Presence of positive family history of ALS
- Clinical evidence of chronic renal or liver failure
- Presence of a bleeding disorder, problems with CSF pressure, allergy to local anesthetics, or a topical or other skin infection at the LP site (lumbar puncture volunteers only)
- Use of any anti-platelet or anticoagulant drugs, such as plavix, aggrenox, ticlid, warfarin or coumadin (lumbar puncture volunteers only)
- Healthy Control Volunteers Inclusion Criteria
- Absence of a known neurological disorder.
- Age 30 - 80 years.
- Ability to provide informed consent.
- Ability to comply with study procedures.
- Medically safe to have lumbar puncture.
- History of ALS, myopathy, neuropathy, ALS mimic disorder or other neurodegenerative disease.
- Presence of positive family history of ALS.
- Clinical evidence of chronic liver or renal failure.
- Presence of bleeding disorder, problems with CSF pressure, allergy to local anesthetics, or a topical or other skin infection at the LP site (LP research volunteers only).
- Research participant must not be taking anti-platelet or anticoagulant drugs, such as plavix, aggrenox, ticlid, warfarin or coumadin (LP research volunteers only).
Trial locations and contacts
30 sites in 2 countries.
30 sites in 2 countries
United States29 sites
Upstate Clinical Research, LLC
Albany, New York, 12205
Emory University
Atlanta, Georgia, 30322
Johns Hopkins University
Baltimore, Maryland, 21205
Massachusetts General Hospital
Boston, Massachusetts, 02129
Lahey Clinic
Burlington, Massachusetts, 01805
Carolinas Health Care
Charlotte, North Carolina, 28207
University of Chicago
Chicago, Illinois, 60637
OSU Medical Center
Columbus, Ohio, 43210
Duke University Medical Center
Durham, North Carolina, 27705
Saint Mary's Healthcare
Grand Rapids, Michigan, 49503
Pennsylvania State University
Hershey, Pennsylvania, 17033
Methodist Neurological Institute
Houston, Texas, 77030
Mayo Clinic Neurology
Jacksonville, Florida, 32224
Dartmouth Hitchcock Medical Center
Lebanon, New Hampshire, 03756
University of Miami
Miami, Florida, 33136
Hennepin County Medical Center
Minneapolis, Minnesota, 55404
Robert Wood Johnson/UMDNJ
New Brunswick, New Jersey, 08901
Beth Israel Medical Center, PACC
New York, New York, 10003
University of California Irvine
Orange, California, 92868
Drexel University College of Medicine
Philadelphia, Pennsylvania, 19107
Phoenix Neurological Associates, Ltd.
Phoenix, Arizona, 85018
University of Pittsburgh
Pittsburgh, Pennsylvania, 15213
Providence ALS Clinic
Portland, Oregon, 97213
Oregon Health & Science University
Portland, Oregon, 97233
University of Utah
Salt Lake City, Utah, 84132
Saint Louis University
St Louis, Missouri, 63104
Washington University
St Louis, Missouri, 63110
SUNY Upstate Medical University
Syracuse, New York, 13210
Wake Forest University
Winston-Salem, North Carolina, 27157
About this study
Official title: A Multicenter Study for the Validation of ALS Biomarkers
The purpose of this study is to collect 650 blood and 300 cerebrospinal fluid (CSF) samples from people with amyotrophic lateral sclerosis (ALS), pure lower or upper motor neuron diseases, as well as other neurodegenerative diseases and from people with no neurological disorder. 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 diagnose ALS and monitor disease progression.
Additionally, up to 600 blood samples will be collected for a sub-study for DNA analysis. Studying components of the blood, such as DNA, may help us understand what happens when genes function abnormally and how it might be related to disease.
Read the detailed description
Researchers tested what changes happen in volunteers with ALS that can be seen in the blood and what changes are unique to ALS and are different from those found in healthy volunteers and volunteers with neurological diseases other than ALS. These changes are called biomarkers. Biomarkers for ALS have been found in blood collected in earlier phases of this study. Biomarkers are non-genetic elements in your blood that may help to make diagnosing ALS easier. In the next phase, comparison of these changes in the blood of volunteers with ALS and without ALS will be used to confirm these biomarkers and to develop a tool to diagnose and monitor progression of ALS.
- Conditions
- Amyotrophic Lateral Sclerosis, Lou Gehrig's Disease, Primary Lateral Sclerosis, Nervous System Diseases, Hereditary Spastic Paraparesis
- Being tested
- No intervention
- Keywords
- Biomarkers, Plasma, Cerebrospinal Fluid, DNA, Serum
- Medical subject headings
- Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Nervous System Diseases, Spastic Paraplegia, Hereditary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
No interventionOther
Sample collection
| Study group | Role | What they receive |
|---|---|---|
| Early ALS | Not described Receives: Other: No intervention | |
| Suspected ALS | Not described Receives: Other: No intervention | |
| Disease Mimics of ALS | Not described Receives: Other: No intervention | |
| Healthy Controls | Not described Receives: Other: No intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
ALS Functional Rating Scale (ALSFRS-R)
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: Every 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 May 2008 (estimated)
- Study startApr 2008
- Primary completionNov 2015
- Study completionNov 2015
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-control
- Time perspective
- Prospective
- Enrolment
- 475 participants (actual)
Sponsor and investigators
- Lead sponsor
- Massachusetts General Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- ALS Association, ALS Therapy Alliance, National Institutes of Health (NIH), National Institute of Neurological Disorders and Stroke (NINDS)
- 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
- NCT00677768
- Sponsor's protocol ID
- BIO_ALS-01
- Other IDs
- 5RC1NS068179-02
- Acronym
- BIO_ALS-01
- Status last verified
- Jun 2016
- First submitted
- 9 May 2008
- Last updated
- 3 Jun 2016
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Jun 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter Study for the Validation of ALS Biomarkers
- ClinicalTrials.gov ID
- NCT00677768
- Acronym / sponsor ID
- BIO_ALS-01 / BIO_ALS-01
- Conditions
- Amyotrophic Lateral Sclerosis, Lou Gehrig's Disease, Primary Lateral Sclerosis, Nervous System Diseases, Hereditary Spastic Paraparesis
- Keywords
- Biomarkers, Plasma, Cerebrospinal Fluid, DNA, Serum
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 475 participants (actual)
- Lead sponsor
- Massachusetts General HospitalUniversity, hospital or other
- Collaborators
- ALS Association, ALS Therapy Alliance, National Institutes of Health (NIH), National Institute of Neurological Disorders and Stroke (NINDS)
- Study start
- Apr 2008
- Primary completion
- Nov 2015 (actual)
- Study completion
- Nov 2015 (actual)
- First posted
- 14 May 2008 (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 650 blood and 300 cerebrospinal fluid (CSF) samples from people with amyotrophic lateral sclerosis (ALS), pure lower or upper motor neuron diseases, as well as other neurodegenerative diseases and from people with no neurological disorder. 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 diagnose ALS and monitor disease progression.
Additionally, up to 600 blood samples will be collected for a sub-study for DNA analysis. Studying components of the blood, such as DNA, may help us understand what happens when genes function abnormally and how it might be related to disease.
Detailed description
Researchers tested what changes happen in volunteers with ALS that can be seen in the blood and what changes are unique to ALS and are different from those found in healthy volunteers and volunteers with neurological diseases other than ALS. These changes are called biomarkers. Biomarkers for ALS have been found in blood collected in earlier phases of this study. Biomarkers are non-genetic elements in your blood that may help to make diagnosing ALS easier. In the next phase, comparison of these changes in the blood of volunteers with ALS and without ALS will be used to confirm these biomarkers and to develop a tool to diagnose and monitor progression of ALS.
Study design
- Observational model
- Case-control
- Time perspective
- Prospective
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Early ALS | – | Other: No intervention |
| Suspected ALS | – | Other: No intervention |
| Disease Mimics of ALS | – | Other: No intervention |
| Healthy Controls | – | Other: No intervention |
Who may qualify
- Ages
- 30 to 80 years
- Sex
- All
- Healthy volunteers
- 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 (33 listed)
Key exclusion criteria (25 listed)
- Suspected ALS (PMND) Volunteers
- Neurological Disease Mimic Volunteers
- Healthy Control Volunteers Inclusion Criteria
- History of ALS, myopathy, neuropathy, ALS mimic disorder or other neurodegenerative disease.
- Presence of positive family history of ALS.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| No interventionSample collection | Other | Disease Mimics of ALS, Early ALS, Healthy Controls, Suspected ALS |
Primary outcome
ALS Functional Rating Scale (ALSFRS-R)
Time frame: Every 6 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.
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 3 Jun 2016.
- ALS Association WebsiteLink provided by the sponsor.
- NEALS ALS ConsortiumLink provided by the sponsor.
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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
30 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Amyotrophic Lateral Sclerosis and Lou Gehrig's Disease, run by Massachusetts General Hospital at 30 sites in 2 countries.
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 33 inclusion and 25 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 3 Jun 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 30 sites in United States, Canada. 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 (ALSFRS-R) (time frame: Every 6 months). 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 3 Jun 2016.