Screening and Natural History: Primary Lateral Sclerosis and Related Disorders

• ClinicalTrials.gov Identifier: NCT00015444
• Recruitment Status: Completed
• First Posted: April 19, 2001
• Last Update Posted: December 16, 2019
• Sponsor: National Institute of Neurological Disorders and Stroke (NINDS)
• Information provided by (Responsible Party): National Institutes of Health Clinical Center (CC) ( National Institute of Neurological Disorders and Stroke (NINDS) )

Study Description

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....

Condition or disease
Primary Lateral Sclerosis

Detailed Description:

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. Blood samples may be collected for measurement of potential etiologies of PLS, including risk factor genes. 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.

Study Design

• Study Type: Observational
• Actual Enrollment: 189 participants
• Observational Model: Cohort
• Time Perspective: Other
• Official Title: Screening and Natural History: Primary Lateral Sclerosis and Related Disorders
• Study Start Date: May 1, 2001
• Study Completion Date: April 24, 2019

Groups and Cohorts

Outcome Measures

Primary Outcome Measures :

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

• Ages Eligible for Study: 18 Years and older (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

• INCLUSION CRITERIA:
• 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

EXCLUSION CRITERIA:

• 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
• Anticoagulation will be an exclusion for needle EMG studies
• Implanted devices or metal fragments in the brain or spinal cord will be an exclusion for MRI scanning

Contacts and Locations

Locations

United States, Maryland

National Institutes of Health Clinical Center, 9000 Rockville Pike

Bethesda, Maryland, United States, 20892

Sponsors and Collaborators

National Institute of Neurological Disorders and Stroke (NINDS)

Investigators

• Principal Investigator: Mary Kay Floeter, M.D.; National Institute of Neurological Disorders and Stroke (NINDS)

More Information

Publications:

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

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

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.

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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.

• Responsible Party: National Institute of Neurological Disorders and Stroke (NINDS)
• ClinicalTrials.gov Identifier: NCT00015444
• Other Study ID Numbers: 010145; 01-N-0145
• First Posted: April 19, 2001
• Last Update Posted: December 16, 2019
• Last Verified: April 24, 2019

Keywords provided by National Institutes of Health Clinical Center (CC) ( National Institute of Neurological Disorders and Stroke (NINDS) ):

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

Additional relevant MeSH terms:

Motor Neuron Disease; Sclerosis; Pathologic Processes; Neurodegenerative Diseases; Nervous System Diseases; Neuromuscular Diseases