Study of ALS Reversals 2: Genetic Analyses
StAR2
ALS Reversals: Genetic Analyses (St.A.R. Protocol 2) RDCRN CReATe Protocol #8007
2 other identifiers
observational
26
1 country
1
Brief Summary
The purpose of this study is to try to understand why reversals of amyotrophic lateral sclerosis (ALS) and primary muscular atrophy (PMA) take place. The study will enroll patients with ALS or PMA reversals to give saliva samples in order to determine if the ALS or PMA reversal is because of certain changes in the genetic code.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for all trials
Started Jun 2018
Longer than P75 for all trials
1 active site
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
First Submitted
Initial submission to the registry
March 8, 2018
CompletedFirst Posted
Study publicly available on registry
March 14, 2018
CompletedStudy Start
First participant enrolled
June 22, 2018
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 1, 2023
CompletedStudy Completion
Last participant's last visit for all outcomes
December 1, 2023
CompletedJanuary 11, 2024
January 1, 2024
5.4 years
March 8, 2018
January 9, 2024
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
genetic comparison
comparison of genes of participants with ALS reversals to genes of more typically progressive patients with ALS
1 day
Secondary Outcomes (1)
factors associated with genes
1 day
Eligibility Criteria
Participants will be enrolled by invitation only. Potential participants will have had a previously verified diagnosis of ALS or PMA and a verified sustained and robust reversal of disease.
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
- Prior participation in the Phenotype Genotype and Biomarkers in ALS and Related Disorders (RDCRN #8001) protocol
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Duke Universitylead
- CReATe Consortium (funded by NIH/NCATS/NINDS)collaborator
Study Sites (1)
Duke ALS Clinic / DUSOM Dept of Neurology / DUHS
Durham, North Carolina, 27705, United States
Related Publications (7)
Ozdinler PH, Silverman RB. Treatment of amyotrophic lateral sclerosis: lessons learned from many failures. ACS Med Chem Lett. 2014 Oct 8;5(11):1179-81. doi: 10.1021/ml500404b. eCollection 2014 Nov 13.
PMID: 25408825BACKGROUNDhttp://www.wsj.com/articles/the-mystery-of-als-patients-who-see-improvement-1465845332
BACKGROUNDALSUntangled 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: 21981685BACKGROUNDHarrison D and Bedlack R (unpublished data).
BACKGROUNDBedlack 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: 26658909BACKGROUNDSamson M, Libert F, Doranz BJ, Rucker J, Liesnard C, Farber CM, Saragosti S, Lapoumeroulie C, Cognaux J, Forceille C, Muyldermans G, Verhofstede C, Burtonboy G, Georges M, Imai T, Rana S, Yi Y, Smyth RJ, Collman RG, Doms RW, Vassart G, Parmentier M. Resistance to HIV-1 infection in caucasian individuals bearing mutant alleles of the CCR-5 chemokine receptor gene. Nature. 1996 Aug 22;382(6593):722-5. doi: 10.1038/382722a0.
PMID: 8751444BACKGROUNDCrayle JI, Rampersaud E, Myers JR, Wuu J, Taylor JP, Wu G, Benatar M, Bedlack RS. Genetic Associations With an Amyotrophic Lateral Sclerosis Reversal Phenotype. Neurology. 2024 Aug 27;103(4):e209696. doi: 10.1212/WNL.0000000000209696. Epub 2024 Jul 30.
PMID: 39079071DERIVED
Related Links
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY CHAIR
Richard S Bedlack, MD, PhD
Duke Health
Study Design
- Study Type
- observational
- Observational Model
- CASE CONTROL
- Time Perspective
- OTHER
- Target Duration
- 1 Day
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
March 8, 2018
First Posted
March 14, 2018
Study Start
June 22, 2018
Primary Completion
December 1, 2023
Study Completion
December 1, 2023
Last Updated
January 11, 2024
Record last verified: 2024-01
Data Sharing
- IPD Sharing
- Will share
Information obtained from this analysis (genotypic data), as well as information about disease signs and symptoms (phenotypic data), will be entered into one or more scientific repositories maintained by organizations such as the New York Genome Center (NYGC) and the Federal Government. One such repository is the Database of Genotypes and Phenotypes (dbGaP), a data repository at the NIH. All data and information will be submitted via a secure transmission process to a high security network within NIH. While the information and data resulting from this study may be presented at scientific meetings or published in a scientific journal, your name or other personal information will not be revealed.