Screening and Natural History: Primary Lateral Sclerosis and Related Disorders

NCT00015444 | Completed

• 2 other identifiers: 01014501-N-0145
• Study Type: observational
• Target: 189
• Locations: 1 country
• Sites: 1

Brief Summary

Objective: The objectives of this protocol are: to develop and maintain a repository of clinically characterized patients with primary lateral sclerosis for future research protocols, to characterize the natural history of neurodegenerative disorders with corticospinal neuron degeneration, to investigate proposed etiologies, risk factors, and biomarkers for the development of these disorders and for disease progression Study Population: 240 patients with adult-onset progressive spasticity with a diagnosis of primary lateral sclerosis or related upper motor neuron disorder Design: Patients who have been referred by physicians for primary lateral sclerosis will undergo a screening evaluation at the first visit. The screening visit will include review of outside medical records, neurological examination, and diagnostic testing to determine possible causes of spasticity. Patients fulfilling the clinical criteria for primary lateral sclerosis by history or examination will be followed to determine the natural history of this disorder. Measures of motor and cognitive function will be made at baseline and follow-up visits to follow clinical progression. Magnetic resonance imaging will be carried out to determine if imaging changes occur over time. Patients identified in this protocol who are eligible for other research protocols will be invited to participate in additional protocols. Outcome Measures: Clinical progression will be documented by measures of finger-tapping, timed gait, speech. The association between clinical progression and MRI measures will be assessed as a secondary outcome....

Conditions

Primary Lateral Sclerosis

Keywords

Apparently Sporadic Spastic Paraplegia; Spasticity; Motor Neuron Disease; Amyotrophic Lateral Sclerosis; Primary Lateral Sclerosis; Muscle Stiffness; Involuntary Muscle Contraction; Isaacs Syndrome

Outcome Measures

Primary Outcomes (1)

• The primary outcome of this protocol is to document the natural history of clinical progression in PLS, defined as the change in clinical measures of movement speed over time: finger tapping, timed gait, and time to read a standard passage.

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age 18 years of age or older
• Adult onset of progressive spasticity
• No family history of a similar disorder
• Able to provide consent or with a legally-authorized representative who can provide consent

You may not qualify if:

• History of stroke, cerebral palsy, traumatic brain injury or other known etiology of spasticity
• Non-neurological disorders producing muscle stiffness, such as fasciitis or rheumatological conditions
• Disorders in which pain limits the ability to move muscles, such as fibromyalgia or complex regional pain syndromes
• Profound weakness of voluntary movement
• Inability to travel to NIH

Sponsors & Collaborators

• National Institute of Neurological Disorders and Stroke (NINDS): lead

Study Sites (1)

National Institutes of Health Clinical Center, 9000 Rockville Pike

Bethesda, Maryland, 20892, United States

Location

Related Publications (4)

• Leclerc KM, Landry FJ. Benign nocturnal leg cramps. Current controversies over use of quinine. Postgrad Med. 1996 Feb;99(2):177-8, 181-4.

  PMID: 8632965 BACKGROUND

• Bentley S. Exercise-induced muscle cramp. Proposed mechanisms and management. Sports Med. 1996 Jun;21(6):409-20. doi: 10.2165/00007256-199621060-00003.

  PMID: 8784961 BACKGROUND

• Auger RG. AAEM minimonograph #44: diseases associated with excess motor unit activity. Muscle Nerve. 1994 Nov;17(11):1250-63. doi: 10.1002/mus.880171103.

  PMID: 7935547 BACKGROUND

• Clark MG, Smallwood Shoukry R, Huang CJ, Danielian LE, Bageac D, Floeter MK. Loss of functional connectivity is an early imaging marker in primary lateral sclerosis. Amyotroph Lateral Scler Frontotemporal Degener. 2018 Nov;19(7-8):562-569. doi: 10.1080/21678421.2018.1517180. Epub 2018 Oct 9.

  PMID: 30299161 DERIVED

MeSH Terms

Conditions

Motor Neuron Disease; Muscle Spasticity; Amyotrophic Lateral Sclerosis; Dyskinesias; Isaacs Syndrome

Condition Hierarchy (Ancestors)

Neurodegenerative Diseases; Nervous System Diseases; Neuromuscular Diseases; Muscular Diseases; Musculoskeletal Diseases; Muscle Hypertonia; Neuromuscular Manifestations; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Spinal Cord Diseases; Central Nervous System Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases; Movement Disorders; Peripheral Nervous System Diseases

Study Officials

• Mary Kay Floeter, M.D.

  National Institute of Neurological Disorders and Stroke (NINDS)

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: OTHER
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 18, 2001
• First Posted: April 19, 2001
• Study Start: May 1, 2001
• Study Completion: April 24, 2019
• Last Updated: December 16, 2019

Record last verified: 2019-04-24

Locations

US (1)