Amyotrophic Lateral SclerosisStARLiTENCT03706391
Study of ALS Reversals 4: LifeTime Exposures
An observational study of Amyotrophic Lateral Sclerosis and Progressive Muscular Atrophy, run by Duke University at 1 site in United States.
Trial summary
Hypothesis: There exists patients who have met ALS or PMA diagnostic criteria and subsequently experienced robust and sustained improvement, i.e. a "reversal." Thirty-eight of these patients were identified in the prior Duke University study, Documentation of Known ALS Reversals (St.A.R. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Amyotrophic Lateral Sclerosis, Progressive Muscular Atrophy, Motor Neuron Disease
- How many people
- 25 participants
- Where
- 1 siteUnited States
- Study dates
- 26 Sep 2018to 1 Jun 2020
- How long will I take part?
- 1 Day
- 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 Any age
- All sexes
- Child, Adult, Older adult
Study population: Participants will be recruited from the cohort of subjects (38 to-date) who participated in the Documentation of Known ALS Reversals (Duke IRB Pro00076395) protocol and were subsequently thought to have an ALS Reversal.
Who may be able to join
- Prior participation in Documentation of Known ALS Reversals (Duke IRB Pro00076395)
- Confirmation of ALS or PMA (primary muscular atrophy) diagnosis through medical record review (previously documented in Documentation of Known ALS Reversals protocol)
- Sustained, robust improvement on at least one objective ALS outcomes measure (ex. ALSFRS-R, FVC, strength testing, EMG) (previously documented in Documentation of Known ALS Reversals protocol)
- Able to understand English
Who cannot join
- History of cognitive impairment severe enough to preclude informed consent, reported by patient on direct questioning or as suspected by research personnel from Documentation of Known ALS Reversals (Duke IRB Pro00076395) study data
Trial locations and contacts
1 site in 1 country.
United States1 site
Duke ALS Clinic / DUSOM Dept of Neurology / DUHS
Durham, North Carolina, 27705
About this study
Hypothesis: There exists patients who have met ALS or PMA diagnostic criteria and subsequently experienced robust and sustained improvement, i.e. a "reversal." Thirty-eight of these patients were identified in the prior Duke University study, Documentation of Known ALS Reversals (St.A.R. Protocol 1, Duke IRB Pro00076395). The investigators hypothesize these patients have had different environmental exposures than patients with typically progressive ALS. Identification of specific environmental influences may point to exposures which are protective or exposure that lead to the development of a rare and novel reversible ALS-like disease.
Objective: This study seeks to identify environmental exposures associated with ALS reversals.
Read the detailed description
Amyotrophic Lateral Sclerosis (ALS) is a devastating motor neuron disease that typically causes rapidly progressive muscle weakness, disability and premature death. In spite of a large number of attempted ALS trials, there are no significant disease-modifying therapies for this condition to-date.
There exists a small group of patients who meet diagnostic criteria for ALS or progressive muscular atrophy (PMA), progress for a period of time, and then significantly improve. Some of these "ALS reversals" even make a complete recovery back to normal neurological function. The investigators have independently verified 38 of these cases so far through review of medical records and peer-reviewed literature. These patients are different in their demographics and disease characteristics as compared to patients with more typically progressive ALS.
One possible explanation for these cases is that these patients have had protective environmental exposures. Another possible explanation is that these patients have had unique environmental exposures that led to a reversible form of ALS. Study of these selected reversal patients may yield valuable clues to environmental mechanisms of ALS resistance.
This is a pilot case-control study attempting to discover environmental exposure correlates to ALS reversals. The investigators will recruit and enroll ALS reversal "cases" to fill out an online survey form about their life. Topics include demographics, employment history, military service, substance use, physical activity, family medical history, disease progression, residential history, occupational exposures, home exposures, hobby exposures, hormonal and reproductive history (female identifying subjects only), caffeine, head and neck injuries, electrical shocks, health insurance, subjective perception of the etiology of ALS, and clinical features of disease. "Control" participant data will come from a pre-existing database.
- Keywords
- amyotrophic lateral sclerosis, progressive muscular atrophy, motor neuron disease, ALS Reversal, PMA Reversal
- Medical subject headings
- Amyotrophic Lateral Sclerosis, Muscular Atrophy, Spinal, Motor Neuron Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| ALS and PMA Reversals | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
Life Time Environmental Exposures
Case-control analysis of differences in environmental exposures over life time of subject. Environmental exposures will be measured with the National ALS Registry survey. Cases are "ALS Reversals." Controls are typically progressive patients with ALS.
Measured: 2 hours to fill out survey
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Oct 2018
- Study start26 Sep 2018
- Primary completion1 Jun 2020
- Study completion1 Jun 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- More trials run by Duke 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-control
- Time perspective
- Cross-sectional
- Enrolment
- 25 participants (actual)
- Target duration
- 1 Day
Sponsor and investigators
- Lead sponsor
- Duke University
- Sponsor type
- University, hospital or other
- Collaborators
- National ALS Registry, Centers for Disease Control and Prevention, Agency for Toxic Substances and Disease Registry
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03706391
- Sponsor's protocol ID
- Pro00100219
- Acronym
- StARLiTE
- Status last verified
- Jul 2020
- First submitted
- 11 Oct 2018
- Last updated
- 20 Jan 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
ALSUntangled Group. ALSUntangled No. 12: Dean Kraft, Energy Healer. Amyotroph Lateral Scler. 2011 Sep;12(5):389-91. doi: 10.3109/17482968.2011.609309. No abstract available.
PubMed 21981685Bedlack RS, Vaughan T, Wicks P, Heywood J, Sinani E, Selsov R, Macklin EA, Schoenfeld D, Cudkowicz M, Sherman A. How common are ALS plateaus and reversals? Neurology. 2016 Mar 1;86(9):808-12. doi: 10.1212/WNL.0000000000002251. Epub 2015 Dec 9.
PubMed 26658909http://www.wsj.com/articles/the-mystery-of-als-patients-who-see-improvement-1465845332
Harrison D, Mehta P, van Es MA, Stommel E, Drory VE, Nefussy B, van den Berg LH, Crayle J, Bedlack R; Pooled Resource Open-Access ALS Clinical Trials Consortium. "ALS reversals": demographics, disease characteristics, treatments, and co-morbidities. Amyotroph Lateral Scler Frontotemporal Degener. 2018 Nov;19(7-8):495-499. doi: 10.1080/21678421.2018.1457059. Epub 2018 Apr 2.
PubMed 29607695Crayle J and Bedlack R. Unpublished data. 2018.
Study at a glance
From the registry record, last updated by the sponsor on 20 Jan 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study of ALS Reversals 4: LifeTime Exposures
- ClinicalTrials.gov ID
- NCT03706391
- Acronym / sponsor ID
- StARLiTE / Pro00100219
- Keywords
- amyotrophic lateral sclerosis, progressive muscular atrophy, motor neuron disease, ALS Reversal, PMA Reversal
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 25 participants (actual)
- Lead sponsor
- Duke UniversityUniversity, hospital or other
- Collaborators
- National ALS Registry, Centers for Disease Control and Prevention, Agency for Toxic Substances and Disease Registry
- Study start
- 26 Sep 2018 (actual)
- Primary completion
- 1 Jun 2020 (actual)
- Study completion
- 1 Jun 2020 (actual)
- First posted
- 16 Oct 2018 (actual)
- 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.
Hypothesis: There exists patients who have met ALS or PMA diagnostic criteria and subsequently experienced robust and sustained improvement, i.e. a "reversal." Thirty-eight of these patients were identified in the prior Duke University study, Documentation of Known ALS Reversals (St.A.R. Protocol 1, Duke IRB Pro00076395). The investigators hypothesize these patients have had different environmental exposures than patients with typically progressive ALS. Identification of specific environmental influences may point to exposures which are protective or exposure that lead to the development of a rare and novel reversible ALS-like disease.
Objective: This study seeks to identify environmental exposures associated with ALS reversals.
Detailed description
Amyotrophic Lateral Sclerosis (ALS) is a devastating motor neuron disease that typically causes rapidly progressive muscle weakness, disability and premature death. In spite of a large number of attempted ALS trials, there are no significant disease-modifying therapies for this condition to-date.
There exists a small group of patients who meet diagnostic criteria for ALS or progressive muscular atrophy (PMA), progress for a period of time, and then significantly improve. Some of these "ALS reversals" even make a complete recovery back to normal neurological function. The investigators have independently verified 38 of these cases so far through review of medical records and peer-reviewed literature. These patients are different in their demographics and disease characteristics as compared to patients with more typically progressive ALS.
One possible explanation for these cases is that these patients have had protective environmental exposures. Another possible explanation is that these patients have had unique environmental exposures that led to a reversible form of ALS. Study of these selected reversal patients may yield valuable clues to environmental mechanisms of ALS resistance.
This is a pilot case-control study attempting to discover environmental exposure correlates to ALS reversals. The investigators will recruit and enroll ALS reversal "cases" to fill out an online survey form about their life. Topics include demographics, employment history, military service, substance use, physical activity, family medical history, disease progression, residential history, occupational exposures, home exposures, hobby exposures, hormonal and reproductive history (female identifying subjects only), caffeine, head and neck injuries, electrical shocks, health insurance, subjective perception of the etiology of ALS, and clinical features of disease. "Control" participant data will come from a pre-existing database.
Study design
- Observational model
- Case-control
- Time perspective
- Cross-sectional
- Target follow-up
- 1 Day
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ALS and PMA Reversals | – | – |
Who may qualify
- Sex
- All
- Age groups
- Child, Adult, Older adult
- Study population
- Participants will be recruited from the cohort of subjects (38 to-date) who participated in the Documentation of Known ALS Reversals (Duke IRB Pro00076395) protocol and were subsequently thought to have an ALS Reversal.
Key inclusion criteria (4 listed)
- Prior participation in Documentation of Known ALS Reversals (Duke IRB Pro00076395)
- Confirmation of ALS or PMA (primary muscular atrophy) diagnosis through medical record review (previously documented in Documentation of Known ALS Reversals protocol)
- Sustained, robust improvement on at least one objective ALS outcomes measure (ex. ALSFRS-R, FVC, strength testing, EMG) (previously documented in Documentation of Known ALS Reversals protocol)
- Able to understand English
Key exclusion criteria (1 listed)
- History of cognitive impairment severe enough to preclude informed consent, reported by patient on direct questioning or as suspected by research personnel from Documentation of Known ALS Reversals (Duke IRB Pro00076395) study data
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
Life Time Environmental Exposures
Time frame: 2 hours to fill out survey
Case-control analysis of differences in environmental exposures over life time of subject. Environmental exposures will be measured with the National ALS Registry survey. Cases are "ALS Reversals." Controls are typically progressive patients with ALS.
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 20 Jan 2023.
- Background: PubMed 21981685ALSUntangled Group. ALSUntangled No. 12: Dean Kraft, Energy Healer. Amyotroph Lateral Scler. 2011 Sep;12(5):389-91. doi: 10.3109/17482968.2011.609309. No abstract available.
- Background: PubMed 26658909Bedlack RS, Vaughan T, Wicks P, Heywood J, Sinani E, Selsov R, Macklin EA, Schoenfeld D, Cudkowicz M, Sherman A. How common are ALS plateaus and reversals? Neurology. 2016 Mar 1;86(9):808-12. doi: 10.1212/WNL.0000000000002251. Epub 2015 Dec 9.
- Backgroundhttp://www.wsj.com/articles/the-mystery-of-als-patients-who-see-improvement-1465845332
- Background: PubMed 29607695Harrison D, Mehta P, van Es MA, Stommel E, Drory VE, Nefussy B, van den Berg LH, Crayle J, Bedlack R; Pooled Resource Open-Access ALS Clinical Trials Consortium. "ALS reversals": demographics, disease characteristics, treatments, and co-morbidities. Amyotroph Lateral Scler Frontotemporal Degener. 2018 Nov;19(7-8):495-499. doi: 10.1080/21678421.2018.1457059. Epub 2018 Apr 2.
- BackgroundCrayle J and Bedlack R. Unpublished data. 2018.
- Individual participant dataSharing planned: No
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site 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 Progressive Muscular Atrophy, run by Duke University at 1 site 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 4 inclusion and 1 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 20 Jan 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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?
Life Time Environmental Exposures (time frame: 2 hours to fill out survey). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03706391, which also lists 5 linked publications.
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 20 Jan 2023.