Study of ALS Reversals 4: LifeTime Exposures (StARLiTE)
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|ClinicalTrials.gov Identifier: NCT03706391|
Recruitment Status : Enrolling by invitation
First Posted : October 16, 2018
Last Update Posted : August 16, 2019
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.
|Condition or disease|
|Amyotrophic Lateral Sclerosis Progressive Muscular Atrophy Motor Neuron Disease|
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 Type :||Observational [Patient Registry]|
|Estimated Enrollment :||30 participants|
|Target Follow-Up Duration:||1 Day|
|Official Title:||Study of ALS Reversals 4: LifeTime Exposures|
|Actual Study Start Date :||September 26, 2018|
|Estimated Primary Completion Date :||August 1, 2020|
|Estimated Study Completion Date :||August 1, 2020|
|ALS and PMA Reversals|
- 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.
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): NCT03706391
|United States, North Carolina|
|Duke ALS Clinic / DUSOM Dept of Neurology / DUHS|
|Durham, North Carolina, United States, 27705|