Answer ALS: Individualized Initiative for ALS Discovery (AnswerALS)
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
| ClinicalTrials.gov Identifier: NCT02574390 |
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Recruitment Status :
Active, not recruiting
First Posted : October 12, 2015
Last Update Posted : February 5, 2019
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Sponsor:
Johns Hopkins University
Collaborators:
Massachusetts General Hospital
Emory University
Ohio State University
Washington University School of Medicine
Cedars-Sinai Medical Center
University of California, Irvine
Massachusetts Institute of Technology
New York Genome Center
Leandro P. Rizzuto Foundation
Texas Neurology
Northwestern University Les Turner ALS Center
Information provided by (Responsible Party):
Johns Hopkins University
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Brief Summary:
Creation of a large repository of induced pluripotent stem cells (iPSC), bio-fluid samples (blood and spinal fluid (optional)), and cell lines for ALS gene identification. This will be combined carefully with collected measures of the pattern of the symptoms people with ALS have and how these change over time. People with other motor neuron diseases and healthy controls will be included as comparisons
| Condition or disease |
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| Amyotrophic Lateral Sclerosis Primary Lateral Sclerosis Flail Arm ALS Progressive Muscular Atrophy Monomelic Amyotrophy Motor Neuron Disease Asymptomatic ALS Gene Carriers Healthy Controls |
Patients will have 5 study visits; screening, 3, 6, 9 and 12 months. There will be a one year post-participation follow-up period, during which they will receive an email or phone call interview once every 3 months. During the first year, samples will be collected, breathing, muscle strength, spasticity, general function and cognitive behavior will be assessed. Healthy controls will have 2 study visits during which blood samples will be collected and questionnaires given.
| Study Type : | Observational [Patient Registry] |
| Estimated Enrollment : | 1000 participants |
| Observational Model: | Case-Only |
| Time Perspective: | Prospective |
| Target Follow-Up Duration: | 1 Year |
| Official Title: | Answer ALS -Creation of a Large Bio-repository of iPS Cells, Cell Lines, and Bio-fluid Samples, Combined With Clinical Information to Rapidly Advance Therapeutics That Could Treat ALS |
| Study Start Date : | December 2015 |
| Actual Primary Completion Date : | January 2019 |
| Estimated Study Completion Date : | January 2020 |
Resource links provided by the National Library of Medicine
Genetics Home Reference related topics:
Amyotrophic lateral sclerosis
Primary Outcome Measures :
- ALS Functional Rating Scale-Revised (ALSFRS-R) [ Time Frame: once every 3 months for one year ]12 questions about patient's ability to function in certain activities of daily living. Each question is out of 4 with 4 being normal and 0 being completely impaired.
- ALS Cognitive Behavioral Scale (ALS-CBS) [ Time Frame: once every 3 months for one year ]short measure of cognition and behavior in patients with ALS. The cognitive portion consists of 8 tasks with a perfect score being 20. The behavioral portion measures changes in personality and behavior since the onset of ALS symptoms as well as mood, pseudobulbar affect and fatigue and is completed by a family member or caregiver. A normal score is 45.
- Slow Vital Capacity (SVC) [ Time Frame: once every 3 months for one year ]measurement of the maximum amount of air that can be exhaled following a deep breath.
- Strength Testing with Hand Held Dynamometer (HHD) [ Time Frame: once every 3 months for one year ]muscle strength testing performed on upper and lower limbs, ankles, wrists and fingers using a small hand held device. These measurements are followed over time and compared to measure decline.
Biospecimen Retention: Samples With DNA
plasma, serum, DNA, Cerebrospinal fluid, induced pluripotent stem cells,
Information from the National Library of Medicine
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| Ages Eligible for Study: | 18 Years to 100 Years (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | Yes |
| Sampling Method: | Non-Probability Sample |
Study Population
patients with ALS, Primary Lateral Sclerosis Flail Arm ALS Progressive Muscular Atrophy Monomelic Amyotrophy Motor Neuron Disease Asymptomatic ALS Gene Carriers
Criteria
Inclusion Criteria:
- Participants with familial or sporadic ALS diagnosed as possible, laboratory-supported probable, probable or definite according to the World Federation of Neurology (WFN) El Escorial criteria, Primary Lateral Sclerosis Flail Arm ALS, Progressive Muscular Atrophy, Monomelic Amyotrophy, Motor Neuron Disease, Asymptomatic ALS Gene Carriers
- Participants who are ages 18-100, inclusive.
Exclusion Criteria:
- Participants with Spinal-Bulbar Muscular Atrophy
- Known diagnosis of HIV/AIDS, Hepatitis B, or Hepatitis C.
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02574390
Locations
| United States, California | |
| Cedars-Sinai Medical Center | |
| Los Angeles, California, United States, 90048 | |
| United States, Georgia | |
| Emory University | |
| Atlanta, Georgia, United States, 30322 | |
| United States, Illinois | |
| Northwestern University Feinberg School of Medicine | |
| Chicago, Illinois, United States, 60611 | |
| United States, Maryland | |
| Johns Hopkins University | |
| Baltimore, Maryland, United States, 21205 | |
| United States, Massachusetts | |
| Massachusetts General Hospital | |
| Boston, Massachusetts, United States, 02114 | |
| United States, Missouri | |
| Washington University School of Medicine | |
| Saint Louis, Missouri, United States, 63110 | |
| United States, Ohio | |
| Ohio State University Wexner Medical Center | |
| Columbus, Ohio, United States, 43221 | |
| United States, Texas | |
| Texas Neurology | |
| Dallas, Texas, United States, 75214 | |
Sponsors and Collaborators
Johns Hopkins University
Massachusetts General Hospital
Emory University
Ohio State University
Washington University School of Medicine
Cedars-Sinai Medical Center
University of California, Irvine
Massachusetts Institute of Technology
New York Genome Center
Leandro P. Rizzuto Foundation
Texas Neurology
Northwestern University Les Turner ALS Center
Investigators
| Principal Investigator: | Nicholas J Maragakis, MD | Johns Hopkins University | |
| Principal Investigator: | James D Berry, MD | Massachusetts Generel Hospital |
Additional Information:
| Responsible Party: | Johns Hopkins University |
| ClinicalTrials.gov Identifier: | NCT02574390 History of Changes |
| Other Study ID Numbers: |
IRB00082277 |
| First Posted: | October 12, 2015 Key Record Dates |
| Last Update Posted: | February 5, 2019 |
| Last Verified: | February 2019 |
Keywords provided by Johns Hopkins University:
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ALS stem cell biomarker disease progression |
Additional relevant MeSH terms:
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Sclerosis Atrophy Motor Neuron Disease Amyotrophic Lateral Sclerosis Muscular Atrophy Muscular Atrophy, Spinal Pathologic Processes Pathological Conditions, Anatomical Neurodegenerative Diseases Nervous System Diseases |
Neuromuscular Diseases Spinal Cord Diseases Central Nervous System Diseases TDP-43 Proteinopathies Proteostasis Deficiencies Metabolic Diseases Neuromuscular Manifestations Neurologic Manifestations Signs and Symptoms |