NCT03706391

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
25

participants targeted

Target at below P25 for all trials

Timeline
Completed

Started Sep 2018

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

September 26, 2018

Completed
15 days until next milestone

First Submitted

Initial submission to the registry

October 11, 2018

Completed
5 days until next milestone

First Posted

Study publicly available on registry

October 16, 2018

Completed
1.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 1, 2020

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

June 1, 2020

Completed
Last Updated

January 20, 2023

Status Verified

July 1, 2020

Enrollment Period

1.7 years

First QC Date

October 11, 2018

Last Update Submit

January 18, 2023

Conditions

Amyotrophic Lateral SclerosisProgressive Muscular AtrophyMotor Neuron Disease

Keywords

amyotrophic lateral sclerosisprogressive muscular atrophymotor neuron diseaseALS ReversalPMA Reversal

Outcome Measures

Primary Outcomes (1)

  • 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.

    2 hours to fill out survey

Study Arms (1)

ALS and PMA Reversals

Eligibility Criteria

Sexall
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-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.

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Duke ALS Clinic / DUSOM Dept of Neurology / DUHS

Durham, North Carolina, 27705, United States

Location

Related Publications (5)

  • 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.

    PMID: 21981685BACKGROUND

  • Bedlack 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.

    PMID: 26658909BACKGROUND

  • http://www.wsj.com/articles/the-mystery-of-als-patients-who-see-improvement-1465845332

    BACKGROUND

  • 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.

    PMID: 29607695BACKGROUND

  • Crayle J and Bedlack R. Unpublished data. 2018.

    BACKGROUND

MeSH Terms

Conditions

Amyotrophic Lateral SclerosisMuscular Atrophy, SpinalMotor Neuron Disease

Condition Hierarchy (Ancestors)

Spinal Cord DiseasesCentral Nervous System DiseasesNervous System DiseasesNeurodegenerative DiseasesTDP-43 ProteinopathiesNeuromuscular DiseasesProteostasis DeficienciesMetabolic DiseasesNutritional and Metabolic Diseases

Study Design

Study Type
observational
Observational Model
CASE CONTROL
Time Perspective
CROSS SECTIONAL
Target Duration
1 Day
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 11, 2018

First Posted

October 16, 2018

Study Start

September 26, 2018

Primary Completion

June 1, 2020

Study Completion

June 1, 2020

Last Updated

January 20, 2023

Record last verified: 2020-07

Data Sharing

IPD Sharing
Will not share

Locations

US(1)