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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 : February 8, 2019
Sponsor:
Collaborators:
National ALS Registry
Centers for Disease Control and Prevention
Agency for Toxic Substances and Disease Registry
Information provided by (Responsible Party):
Duke University

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
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.


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Study Type : Observational [Patient Registry]
Estimated Enrollment : 30 participants
Observational Model: Case-Control
Time Perspective: Cross-Sectional
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 : March 2019
Estimated Study Completion Date : March 2019


Group/Cohort
ALS and PMA Reversals



Primary Outcome Measures :
  1. 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.



Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Sampling Method:   Non-Probability Sample
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.
Criteria

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

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): NCT03706391


Locations
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United States, North Carolina
Duke ALS Clinic / DUSOM Dept of Neurology / DUHS
Durham, North Carolina, United States, 27705
Sponsors and Collaborators
Duke University
National ALS Registry
Centers for Disease Control and Prevention
Agency for Toxic Substances and Disease Registry

Additional Information:

Publications:
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Responsible Party: Duke University
ClinicalTrials.gov Identifier: NCT03706391     History of Changes
Other Study ID Numbers: Pro00100219
First Posted: October 16, 2018    Key Record Dates
Last Update Posted: February 8, 2019
Last Verified: October 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Duke University:
amyotrophic lateral sclerosis
progressive muscular atrophy
motor neuron disease
ALS Reversal
PMA Reversal

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