Amyotrophic Lateral SclerosisAnswerALSNCT02574390
Answer ALS: Individualized Initiative for ALS Discovery
An observational study of Amyotrophic Lateral Sclerosis and Primary Lateral Sclerosis, run by Johns Hopkins University at 8 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 100 yearsHealthy volunteers can take part
- Conditions the study is for
- Amyotrophic Lateral Sclerosis, Primary Lateral Sclerosis, Flail Arm ALS
- How many people
- 1,049 participants
- Where
- 8 sitesUnited States
- Study dates
- Dec 2015to 1 Jan 2020
- How long will I take part?
- 1 Year
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 100 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Study population: patients with ALS, Primary Lateral Sclerosis Flail Arm ALS Progressive Muscular Atrophy Monomelic Amyotrophy Motor Neuron Disease Asymptomatic ALS Gene Carriers
Who may be able to join
- 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.
Who cannot join
- Participants with Spinal-Bulbar Muscular Atrophy
- Known diagnosis of HIV/AIDS, Hepatitis B, or Hepatitis C.
Trial locations and contacts
8 sites in 1 country.
8 sites in 1 country
United States8 sites
Emory University
Atlanta, Georgia, 30322
Johns Hopkins University
Baltimore, Maryland, 21205
Massachusetts General Hospital
Boston, Massachusetts, 02114
Northwestern University Feinberg School of Medicine
Chicago, Illinois, 60611
Ohio State University Wexner Medical Center
Columbus, Ohio, 43221
Texas Neurology
Dallas, Texas, 75214
Cedars-Sinai Medical Center
Los Angeles, California, 90048
Washington University School of Medicine
St Louis, Missouri, 63110
About this study
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
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
Read the detailed description
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.
- Conditions
- Amyotrophic Lateral Sclerosis, Primary Lateral Sclerosis, Flail Arm ALS, Progressive Muscular Atrophy, Monomelic Amyotrophy, Motor Neuron Disease
- Keywords
- ALS, stem cell, biomarker, disease progression
- Medical subject headings
- Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Muscular Atrophy, Spinal, Amyotrophy, monomelic, Disease Progression
What is being measured
The primary outcome is the main question the study is designed to answer.
ALS Functional Rating Scale-Revised (ALSFRS-R)
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.
Measured: once every 3 months for one year
ALS Cognitive Behavioral Scale (ALS-CBS)
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.
Measured: once every 3 months for one year
Slow Vital Capacity (SVC)
measurement of the maximum amount of air that can be exhaled following a deep breath.
Measured: once every 3 months for one year
Strength Testing with Hand Held Dynamometer (HHD)
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.
Measured: once every 3 months for one year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Oct 2015 (estimated)
- Study startDec 2015
- Primary completion31 Dec 2019
- Study completion1 Jan 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- More trials run by Johns Hopkins UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 1,049 participants (actual)
- Target duration
- 1 Year
Sponsor and investigators
- Lead sponsor
- Johns Hopkins University
- Sponsor type
- University, hospital or other
- 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
- Responsible party
- Sponsor
- Investigators
- Nicholas J Maragakis, MD, Principal investigator, Johns Hopkins UniversityJames D Berry, MD, Principal investigator, Massachusetts Generel Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02574390
- Sponsor's protocol ID
- IRB00082277
- Acronym
- AnswerALS
- Status last verified
- Jan 2020
- First submitted
- 9 Oct 2015
- Last updated
- 13 Jan 2020
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Jan 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT02574390
- Acronym / sponsor ID
- AnswerALS / IRB00082277
- Conditions
- Amyotrophic Lateral Sclerosis, Primary Lateral Sclerosis, Flail Arm ALS, Progressive Muscular Atrophy, Monomelic Amyotrophy, Motor Neuron Disease
- Keywords
- ALS, stem cell, biomarker, disease progression
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 1,049 participants (actual)
- Lead sponsor
- Johns Hopkins UniversityUniversity, hospital or other
- 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
- Study start
- Dec 2015 (actual)
- Primary completion
- 31 Dec 2019 (actual)
- Study completion
- 1 Jan 2020 (actual)
- First posted
- 12 Oct 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Detailed description
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 design
- Observational model
- Case only
- Time perspective
- Prospective
- Target follow-up
- 1 Year
Who may qualify
- Ages
- 18 to 100 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- Study population
- patients with ALS, Primary Lateral Sclerosis Flail Arm ALS Progressive Muscular Atrophy Monomelic Amyotrophy Motor Neuron Disease Asymptomatic ALS Gene Carriers
Key inclusion criteria (2 listed)
- 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.
Key exclusion criteria (2 listed)
- Participants with Spinal-Bulbar Muscular Atrophy
- Known diagnosis of HIV/AIDS, Hepatitis B, or Hepatitis C.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Jan 2020.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Nicholas J Maragakis, MD principal investigator
Johns Hopkins University
James D Berry, MD principal investigator
Massachusetts Generel Hospital
8 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Amyotrophic Lateral Sclerosis and Primary Lateral Sclerosis, run by Johns Hopkins University at 8 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 13 Jan 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
ALS Functional Rating Scale-Revised (ALSFRS-R) (time frame: once every 3 months for one year). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Jan 2020; AzTrials last refreshed its copy on 27 Oct 2026.