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

Tracking Information

• First Submitted Date: July 14, 2008
• First Posted Date: July 16, 2008
• Last Update Posted Date: January 6, 2017
• Study Start Date: April 2007
• Actual Primary Completion Date: November 2016 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: March 1, 2013)

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.

• Original Primary Outcome Measures: Not Provided
• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Amyotrophic Lateral Sclerosis (ALS) Tissue Donation Program
• Official Title: ALS Tissue Donation Program

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.

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 Type: Observational
• Study Design: Observational Model: Case-Control; Time Perspective: Cross-Sectional
• Target Follow-Up Duration: Not Provided

Biospecimen

Retention:   Samples With DNA

Description:

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)

• 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.

Condition

• Amyotrophic Lateral Sclerosis
• Neurodegenerative Disease
• Motor Neuron Disease

• Intervention: Not Provided

Study Groups/Cohorts

• 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

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: January 5, 2017): 205
• Original Estimated Enrollment (submitted: July 14, 2008): 300
• Actual Study Completion Date: November 2016
• Actual Primary Completion Date: November 2016 (Final data collection date for primary outcome measure)

Eligibility 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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: Yes
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT00716131
• Other Study ID Numbers: Internal-16827
• Has Data Monitoring Committee: No
• U.S. FDA-regulated Product: Not Provided

IPD Sharing Statement

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

• Current Responsible Party: Christine Barr, Drexel University
• Original Responsible Party: Terry Heiman-Patterson, MD, MDA/ALS Center of Hope
• Current Study Sponsor: Drexel University College of Medicine
• Original Study Sponsor: Drexel University
• Collaborators: MDA/ALS Center of Hope

Investigators

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

• PRS Account: Drexel University
• Verification Date: January 2017