A Multicenter Study for the Validation of ALS Biomarkers

Status: Enrolling till Dec. 2009

ClinicalTrials.gov identifier: NCT00677768

A key unmet clinical need in ALS both for clinical trials and early diagnosis is a biomarker. The Association has committed significant resources and helped forged industry-academic partnerships to meet this need. As part of a continuing collaboration with Metabolon, Massachusetts General Hospital and the University of Pittsburgh, novel biomarkers identified in pilot studies will be further verified in a large number of ALS samples. In order to achieve this we are seeking participation from healthy volunteers and people with ALS

For a detailed overview of the study and participating sites see below.

DISEASE CLASSIFICATION(S):

ALS, Multi-focal motor neuropathy, Autoimmune motor neuropathy, Cervical or lumbosacral radiculopathies, Ulnar neuropathy, Carpal tunnel syndrome/median neuropathy, Peroneal neuropathy, Sciatic neuropathy, Spinal muscular atrophy, Spinobulbar muscular atrophy (Kennedy's disease), Cervical myelopathy, Multiple sclerosis, Hereditary spastic paraparesis

NAME OF CLINICAL TRIAL/STUDY: 

A Multicenter Study for the Validation of ALS Biomarkers

PURPOSE AND RATIONALE: 

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

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.

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 DETAILS:

Volunteers will be enrolled into 1 of 4 groups.  Volunteers may have their blood drawn up to 3 times and may have cerebrospinal fluid (CSF) drawn once.  The researchers will collect information on your diagnosis, vital signs, medical history and medications, as well as contact you by telephone up to 3 times.

600 blood 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.

OPENING/CLOSING DATES: 

Study Start Date: April 2008

Will close to enrollment: December 2009

Estimated Study Completion Date: June 2011

TARGET NUMBER OF PARTICIPANTS: 600

RECRUITMENT STATUS: The study is currently open to recruitment.

ELIGIBILITY REQUIREMENTS:

1. 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)

2. 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)

3. 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 30-80 years
• Ability to provide informed consent
• Ability to comply with study procedures
• Medically safe to have lumbar puncture ( lumbar puncture volunteers only) Pure Upper Motor Neuron Disease (UMND) mimics: 

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
• 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)
• Requirement for artificial respiration

4. Healthy Control Volunteers

Enrollment to the Healthy Control Group is CLOSED 

LOCATION(S)/CONTACT(S):

NCTU Clinical Trials Information Line
Telephone:   1-877-458-0631  

California

University of California, Irvine
Orange, California 92868
Contact: Veronica Martin    
714-456-7760           
vero@uci.edu
Principal Investigator: Tahseen Mozaffar, MD

Florida

University of Miami
Miami, Florida 33136
Contact: Donald Koggan, MD        
305-243-7424          
dkoggan@med.miami.edu
Principal Investigator: Khema Sharma, MD

Georgia

Emory University
Atlanta, Georgia 30322
Contact: Meraida Polak, RN           
404-778-3807          
mpolak@emory.edu
Principal Investigator: Jonathan Glass, MD

Illinois

University of Chicago
Chicago, Illinois 60637
Contact: Liz Shaviers          
773-702-6221          
eshavier@neurology.bsd.uchicago.edu
Principal Investigator: Raymond Roos, MD

Maryland

Johns Hopkins University
Baltimore, Maryland 21205
Contact: Richard Kimball, RN, MSN          
410-955-8511          
rkimbal1@jhmi.edu
Principal Investigator: Nicholas Maragakis, MD

Massachusetts

Lahey Clinic
Burlington, Massachusetts 01805
Contact: Stephanie Scala    781-744-2950          
stephanie.a.scala@lahey.org
Principal Investigator: James Russell, DO

Massachusetts General Hospital
Boston, Massachusetts 02129
Contact: Matt Jaffa   
617-726-0563          
mjaffa@partners.org
Principal Investigator: Swati P. Aggarwal, MD

Missouri

Saint Louis University
Saint Louis, Missouri 63104
Contact: Susan Eller, RN     314-977-4867          
ellersc@slu.edu
Principal Investigator: Ghazala Hayat, MD

Washington University
Saint Louis, Missouri 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

New Hampshire

Dartmouth Hitchcock Medical Center
Lebanon, New Hampshire 03756
Contact: Brent Harris, MD   
603-650-0889          
bharris@dartmouth.edu
Principal Investigator: Jeffrey Allen Cohen, MD

New Jersey

Robert Wood Johnson/UMDNJ
New Brunswick, NJ 08901
Contact: Barbara Belsh, DPM, MS
732-235-7340
belshba@umdnj.edu 
Principal Investigator: Jerry Belsh, MD

New York

SUNY Upstate Medical University
Syracuse, New York 13210
Contact: Mary Lou Watson, RRT    
315-464-5004
watsonm@upstate.edu
Principal Investigator: Jeremy Shefner, MD

North Carolina

Duke University Medical Center
Durham, North Carolina 27705
Contact: Karen Grace, RN   919-668-2844          
karen.grace@duke.edu 
Principal Investigator: Richard Bedlack, MD

Wake Forest University
Winston-Salem, North Carolina 27157
Contact: Theresa Johnston-Crews, RN     
336-716-2323          
tjcrews@wfubmc.edu
Principal Investigator: James Caress, MD

Oregon

Providence ALS Clinic
Portland, Oregon 97213
Contact: Shiralynn Moore    
503-963-3128          
smoore@orclinic.com
Principal Investigator: Kimberly Goslin, MD

Pennsylvania

Drexel University College of Medicine
Philadelphia, Pennsylvania 19107
Contact: Christine Barr, RN
215-762-5186          
christine.barr@drexelmed.edu
Principal Investigator: Terry Heiman-Patterson, MD

Pennsylvania State University
Hershey, Pennsylvania 17033
Contact: Beth Stephens      
717-531-0003 ext. 283395             
hstephens1@psu.edu
Principal Investigator: Zachary Simmons, MD

University of Pittsburgh
Pittsburgh, Pennsylvania 15213
Contact: Danielle Rowlands, RN    
412-648-9053          
doerflerd@upmc.edu           
Principal Investigator: David Lacomis, MD

Utah

University of Utah
Salt Lake City, Utah 84132
Contact: Summer Davis      
801-588-9055          
summer.davis@hsc.utah.edu
Principal Investigator: Mark Bromberg, MD

FUNDING (Agencies, Pharmaceutical Companies, etc.):

ALS Association

IRB APPROVAL:

The NCTU Coordination Center IRB Approval: January 2, 2008.

Site IRB Approvals on ongoing basis.

 

Posted on October 17, 2008