Validation of Biomarkers in Amyotrophic Lateral Sclerosis (ALS) - Full Text View

Sponsors and Collaborators:	Massachusetts General Hospital ALS Association
Information provided by:	Massachusetts General Hospital
ClinicalTrials.gov Identifier:	NCT00677768

Purpose

The purpose of this study is to collect 600 blood samples from healthy subjects, and both blood and 210 cerebrospinal fluid (CSF) samples from people with amyotrophic lateral sclerosis (ALS), pure lower or upper motor neuron diseases, as well as other neurodegenerative diseases. 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.

Condition	Intervention
Amyotrophic Lateral Sclerosis Lou Gehrig's Disease	Other: No intervention

Genetics Home Reference related topics: amyotrophic lateral sclerosis

MedlinePlus related topics: Amyotrophic Lateral Sclerosis

U.S. FDA Resources

Study Type:	Observational
Study Design:	Case Control, Prospective
Official Title:	A Multicenter Study for the Validation of ALS Biomarkers

Further study details as provided by Massachusetts General Hospital:

Biospecimen Retention: Samples Without DNA

Biospecimen Description:

600 plasma samples will be collected from four groups: ALS volunteers diagnosed with ALS, volunteers with pure lower or pure upper motor neuron diseases, volunteers with other neurological diseases and healthy control volunteers.

210 cerebrospinal fluid (CSF) samples will be collected from a group of volunteers with in the ALS, pure lower or pure upper motor neuron diseases, and other neurological diseases.

Estimated Enrollment:	600
Study Start Date:	April 2008
Estimated Study Completion Date:	June 2011
Estimated Primary Completion Date:	June 2011 (Final data collection date for primary outcome measure)

Groups/Cohorts	Assigned Interventions
1 Those volunteers with ALS.	Other: No intervention Sample collection
2 Those volunteers with a pure lower or pure upper motor neuron disease.	Other: No intervention Sample collection
3 Those volunteers with other neurological diseases (masquerades of ALS).	Other: No intervention Sample collection
4 Healthy control volunteers.	Other: No intervention Sample collection

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.

Eligibility

Ages Eligible for Study:	40 Years to 70 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	Yes
Sampling Method:	Non-Probability Sample

Study Population

Volunteers will be invited to participate in this study by their neurologists either in clinic or at a regular scheduled appointment visit.

Criteria

ALS Volunteers

Inclusion Criteria:
- Diagnosis of possible, probable, probable-laboratory supported, or definite ALS, either sporadic or familial according to modified El Escorial criteria
- Disease duration of less than or equal to two years from symptom onset
- Age 40-70 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)
- Absence of any of the exclusion criteria
Exclusion Criteria:
- Clinical evidence of chronic liver or renal failure
- Requirement for artificial respiration
- 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

Inclusion Criteria:
- Diagnosis of suspected ALS according to modified El Escorial criteria
- Disease duration of less than or equal to two years from symptom onset
- Age 40-70 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)
Exclusion Criteria:
- Clinical evidence of chronic liver or renal failure
- Genetically confirmed diagnosis of hereditary spastic paraparesis or spinal motor atrophy (SMA) disease
- Requirement for artificial respiration
- 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

Inclusion Criteria:

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)
Pure Upper Motor Neuron Disease (UMND) mimics:
- Cervical myelopathy
- Multiple sclerosis
- Hereditary spastic paraparesis
- Age 40-70 years
- Ability to provide informed consent
- Ability to comply with study procedures
- Medically safe to have lumbar puncture (lumbar puncture volunteers only)
Exclusion Criteria:
- Diagnosis of suspected, possible, probable or definite ALS either sporadic or familial according to modified El Escorial criteria
- Presence of positive family history of ALS
- Clinical evidence of chronic renal or liver failure
- Presence of more than one neurological disease
- Requirement for artificial respiration
- 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 40-70 years
Ability to provide informed consent
Ability to comply with study procedures

Exclusion Criteria:

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00677768

Contacts

Contact: Clinical Trials Information Line

1-877-458-0631

Locations

United States, California
University of California Irvine	Recruiting
Orange, California, United States, 92868
Contact: Veronica Martin 714-456-7760 vero@uci.edu
Principal Investigator: Tahseen Mozaffar, MD
United States, Florida
University of Miami	Recruiting
Miami, Florida, United States, 33136
Contact: Donald Koggan, MD 305-243-7424 dkoggan@med.miami.edu
Principal Investigator: Khema Sharma, MD
United States, Georgia
Emory University	Recruiting
Atlanta, Georgia, United States, 30322
Contact: Meraida Polak, RN 404-778-3807 mpolak@emory.edu
Contact: Crystal Richards 404-727-3813 cricha6@emory.edu
Principal Investigator: Jonathan Glass, MD
United States, Illinois
University of Chicago	Recruiting
Chicago, Illinois, United States, 60637
Contact: Liz Shaviers 773-702-6221 eshavier@neurology.bsd.uchicago.edu
Principal Investigator: Raymond Roos, MD
United States, Maryland
Johns Hopkins University	Recruiting
Baltimore, Maryland, United States, 21205
Contact: Richard Kimball, RN 410-955-8511 rkimbal1@jhmi.edu
Principal Investigator: Nicholas Maragakis, MD
United States, Massachusetts
Massachusetts General Hospital	Recruiting
Boston, Massachusetts, United States, 02129
Contact: Matt Jaffa 617-726-0563 mjaffa@partners.org
Principal Investigator: Swati P Aggarwal, MD
Lahey Clinic	Recruiting
Burlington, Massachusetts, United States, 01805
Contact: Stephanie Scala 781-744-2950 stephanie.a.scala@lahey.org
Principal Investigator: James Russell, DO
United States, Missouri
Washington University	Recruiting
Saint Louis, Missouri, United States, 63110
Contact: Charlie Wulf 314-362-6980 wulfc@neuro.wustl.edu
Contact: Julaine Florence, DPT 314-362-6983 florencej@neuro.wustl.edu
Principal Investigator: Alan Pestronk, MD
Saint Louis University	Recruiting
Saint Louis, Missouri, United States, 63104
Contact: Susan Eller, RN 314-977-4867 susan.eller@tenethealth.edu
Principal Investigator: Ghazala Hayat, MD
United States, New Hampshire
Dartmouth Hitchcock Medical Center	Recruiting
Lebanon, New Hampshire, United States, 03756
Contact: Brent Harris, MD 603-650-0889 bharris@dartmouth.edu
Principal Investigator: Jeffrey Allen Cohen, MD
United States, New Jersey
Robert Wood Johnson/UMDNJ	Not yet recruiting
New Brunswick, New Jersey, United States, 08901
Contact: Barbara Belsh, DPM, MS 732-235-7340 belshba@umdnj.edu
Principal Investigator: Jerry Belsh, MD
United States, New York
SUNY Upstate Medical University	Recruiting
Syracuse, New York, United States, 13210
Contact: Mary Lou Watson, RRT 315-464-5004 watsonm@upstate.edu
Principal Investigator: Jeremy Shefner, MD
Upstate Clinical Research, LLC	Not yet recruiting
Albany, New York, United States, 12205
Contact: Suzanne A. Alterman, RN, BSN 518-533-1546 salterman@upstateneurology.com
Principal Investigator: James Wymer, MD, PhD
United States, North Carolina
Duke University Medical Center	Recruiting
Durham, North Carolina, United States, 27705
Contact: Karen Grace, RN 919-668-2844 karen.grace@duke.edu
Principal Investigator: Richard Bedlack, MD
Wake Forest University	Recruiting
Winston-Salem, North Carolina, United States, 27157
Contact: Theresa Johnston-Crews, RN 336-716-2323 tjcrews@wfubmc.edu
Contact: Mozhdeh Marandi, MD 336-713-8577 mmarandi@wfubmc.edu
Principal Investigator: James Caress, MD
United States, Oregon
Providence ALS Clinic	Recruiting
Portland, Oregon, United States, 97213
Contact: Shiralynn Moore 503-963-3128 smoore@orclinic.com
Principal Investigator: Kimberly Goslin, MD
United States, Pennsylvania
Drexel University College of Medicine	Recruiting
Philadelphia, Pennsylvania, United States, 19107
Contact: Christine Barr, RN 215-762-5186 christine.barr@drexelmed.edu
Principal Investigator: Terry Heiman-Patterson, MD
Pennsylvania State University	Recruiting
Hershey, Pennsylvania, United States, 17033
Contact: Beth Stephens 717-531-0003 ext 283395 hstephens1@psu.edu
Contact: Allyson Brothers 717-531-0003 ext 287177 abrothers@psu.edu
Principal Investigator: Zachary Simmons, MD
University of Pittsburgh	Recruiting
Pittsburgh, Pennsylvania, United States, 15213
Contact: Danielle Rowlands, RN 412-648-9053 doerflerd@upmc.edu
Principal Investigator: David Lacomis, MD
United States, Utah
University of Utah	Not yet recruiting
Salt Lake City, Utah, United States, 84132
Contact: Summer Davis 801-588-9055 summer.davis@hsc.utah.edu
Principal Investigator: Mark Bromberg, MD

Sponsors and Collaborators

Massachusetts General Hospital

ALS Association

Investigators

Principal Investigator:	Merit E Cudkowicz, MD, MSc	Massachusetts General Hospital
Principal Investigator:	Swati P Aggarwal, MD	Massachusetts General Hospital

More Information

ALS Association Website This link exits the ClinicalTrials.gov site

Responsible Party:	Massachusetts General Hospital ( Merit Cudkowicz, MD, MSc )
Study ID Numbers:	BIO_ALS-01
Study First Received:	May 9, 2008
Last Updated:	January 7, 2009
ClinicalTrials.gov Identifier:	NCT00677768
Health Authority:	United States: Institutional Review Board

Keywords provided by Massachusetts General Hospital:

Biomarkers
Plasma
Cerebrospinal Fluid

Study placed in the following topic categories:

Amyotrophic lateral sclerosis
Neuromuscular Diseases
Spinal Cord Diseases
Amyotrophic Lateral Sclerosis
Central Nervous System Diseases
Lou Gehrig's disease

Sclerosis
Degenerative motor system disease
Neurodegenerative Diseases
Motor neuron disease
Motor Neuron Disease

Additional relevant MeSH terms:

Pathologic Processes
Nervous System Diseases

ClinicalTrials.gov processed this record on January 14, 2009