StARLiTE: Study of ALS Reversals 4: LifeTime Exposures

Sponsor

Duke University (Other)

Overall Status

Completed

CT.gov ID

NCT03706391

Collaborator

National ALS Registry (Other), Centers for Disease Control and Prevention (U.S. Fed), Agency for Toxic Substances and Disease Registry (Other)

Enrollment

Location

20.2

Actual Duration (Months)

1.2

Patients Per Site Per Month

Study Details

Study Description

Brief 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. 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	Intervention/Treatment	Phase
Amyotrophic Lateral Sclerosis Progressive Muscular Atrophy Motor Neuron Disease

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

Study Type:

Observational [Patient Registry]

Actual Enrollment :

25 participants

Observational Model:

Case-Control

Time Perspective:

Cross-Sectional

Official Title:

Study of ALS Reversals 4: LifeTime Exposures

Actual Study Start Date :

Sep 26, 2018

Actual Primary Completion Date :

Jun 1, 2020

Actual Study Completion Date :

Jun 1, 2020

Arms and Interventions

Arm	Intervention/Treatment
ALS and PMA Reversals

Outcome Measures

Primary Outcome Measures

Life Time Environmental Exposures [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.

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A and Older

Sexes Eligible for Study:

All

Inclusion Criteria:

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

Exclusion Criteria:

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