Amyotrophic Lateral Sclerosis (ALS) Tissue Donation Program

• ClinicalTrials.gov Identifier: NCT00716131
• Recruitment Status: Completed
• First Posted: July 16, 2008
• Last Update Posted: January 6, 2017
• Sponsor: Drexel University College of Medicine
• Collaborator: MDA/ALS Center of Hope
• Information provided by (Responsible Party): Christine Barr, Drexel University

Study Description

Brief Summary:

Despite significant progress in the identification of mechanisms involved in motor neuron degeneration in Amyotrophic Lateral Sclerosis (ALS) and other motor system diseases, the actual pathogenesis and cause of these diseases remains unknown. Effective treatment of these diseases are dependent on the elucidation of their causes. The availability of diseased and control human tissues will be a critical resource for this research progress. . Samples of serum, spinal fluid, and urine from patients with motor system diseases can be used to study biochemical and genetic differences compared to tissues of neurologic disease controls and normal controls. Furthermore, the availability of autopsied CNS, PNS, as well as other tissues from patients with ALS or suspected ALS are useful for current and future research studies into the disease. Therefore, we propose to institute a Tissue Bank containing blood, urine, and cerebrospinal fluid donated from not only ALS and other motor neuron disease patients, but also those with other neurologic diseases and normals whose tissue can be used as controls. In addition there will be an autopsy band for post-mortem specimens of ALS and other motor neuron disease patients. Each specimen, whether from a living patient or autopsy will be de-identified and accompanied by a standard set of clinical information collected from the medical records in order that each specimen is characterized with the relevant clinical information to maximize the usefulness of the specimens.

Once established, this tissue bank will provide a resource in which a large number of samples will be readily available and expedite research by circumventing the delays in collecting specimens prospectively. These specimens will be used for research in the ALS Center of Hope at Drexel University College of Medicine and shared with any outside investigator with a valid IRB approved protocol.

Condition or disease
Amyotrophic Lateral Sclerosis; Neurodegenerative Disease; Motor Neuron Disease

Detailed Description:

Despite significant progress in the identification of mechanisms involved in motor neuron degeneration in Amyotrophic Lateral Sclerosis (ALS) and other motor system diseases, the actual pathogenesis and cause of these diseases remains unknown. Effective treatment of these diseases are dependent on the elucidation of their causes. The availability of diseased and control human tissues will be a critical resource for this research progress. . Samples of serum, spinal fluid, and urine from patients with motor system diseases can be used to study biochemical and genetic differences compared to tissues of neurologic disease controls and normal controls. Furthermore, the availability of autopsied CNS, PNS, as well as other tissues from patients with ALS or suspected ALS are useful for current and future research studies into the disease. Therefore, we propose to institute a Tissue Bank containing blood, urine, and cerebrospinal fluid donated from not only ALS and other motor neuron disease patients, but also those with other neurologic diseases and normals whose tissue can be used as controls. In addition there will be an autopsy band for post-mortem specimens of ALS and other motor neuron disease patients. Each specimen, whether from a living patient or autopsy will be de-identified and accompanied by a standard set of clinical information collected from the medical records in order that each specimen is characterized with the relevant clinical information to maximize the usefulness of the specimens.

Once established, this tissue bank will provide a resource in which a large number of samples will be readily available and expedite research by circumventing the delays in collecting specimens prospectively. These specimens will be used for research in the ALS Center of Hope at Drexel University College of Medicine and shared with any outside investigator with a valid IRB approved protocol.

Study Design

• Study Type: Observational
• Actual Enrollment: 205 participants
• Observational Model: Case-Control
• Time Perspective: Cross-Sectional
• Official Title: ALS Tissue Donation Program
• Study Start Date: April 2007
• Actual Primary Completion Date: November 2016
• Actual Study Completion Date: November 2016

Groups and Cohorts

Group/Cohort
ALS; Diagnosed with ALS or other motor system disorder including PLS, Bulbar Palsy or Motor neuropathy
Neuro; Diagnosed with other chronic neurologic illnesses (Alzheimers, multiple sclerosis, migraines, etc)
Healthy; Normal Controls
Autopsy

Outcome Measures

Primary Outcome Measures :

1. None Specified [ Time Frame: None Specified ]
   No primary outcome measure is specified for this study. Purpose of the study is to collect deidentified biological specimens which will be used to expedite other IRB approved studies.

Biospecimen Retention:   Samples With DNA

Blood (Serum and DNA) Urine (Serum and DNA) CSF Blood specimens and tissue specimens (from brain, spinal cord, gastrointestinal tract, skeletal muscle and peripheral nerve)

Eligibility Criteria

• Ages Eligible for Study: 18 Years and older (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: Yes
• Sampling Method: Non-Probability Sample

Study Population

ALS clinic patients at MDA/ALS Center of Hope, along with families and caregivers. Also those with ALS who have donated their bodies to research.

Criteria

Inclusion Criteria:

• Diagnosed with ALS or other motor system disorder including PLS, Bulbar Palsy or Motor neuropathy
• Diagnosed with other chronic neurologic illnesses (Alzheimers, multiple sclerosis, migraines, etc)
• Normal Controls
• In the case of spinal fluid collection, the patient will be undergoing a diagnostic lumbar puncture as part of the work-up

Exclusion Criteria:

• Any person with a non-neurologic chronic and poorly controlled systemic illness

Contacts and Locations

Locations

United States, Pennsylvania

MDA/ALS Center of Hope

Philadelphia, Pennsylvania, United States, 19107

Sponsors and Collaborators

Drexel University College of Medicine

MDA/ALS Center of Hope

Investigators

• Principal Investigator: Terry D Heiman-Patterson, MD; Drexel University College of Medicine

More Information

• Responsible Party: Christine Barr, Research Nurse Coordinator, Drexel University
• ClinicalTrials.gov Identifier: NCT00716131
• Other Study ID Numbers: Internal-16827
• First Posted: July 16, 2008
• Last Update Posted: January 6, 2017
• Last Verified: January 2017

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No
• Plan Description: The purpose of this protocol is to collect and bank tissue specimens.

Keywords provided by Christine Barr, Drexel University:

Amyotrophic Lateral Sclerosis; Cerebrospinal Fluid; Neurodegenerative Disease; Motor Neuron Disease; Autonomic Nervous System; Neurodegenerative Diseases; Movement Disorders

Additional relevant MeSH terms:

Motor Neuron Disease; Amyotrophic Lateral Sclerosis; Neurodegenerative Diseases; Sclerosis; Pathologic Processes; Nervous System Diseases; Neuromuscular Diseases; Spinal Cord Diseases; Central Nervous System Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Metabolic Diseases