Screening and Natural History: Primary Lateral Sclerosis and Related Disorders

Primary Lateral Sclerosis Clinical Trial

Official title:

Screening and Natural History: Primary Lateral Sclerosis and Related Disorders

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00015444
• Other study ID #: 010145
• Secondary ID: 01-N-0145
• Status: Completed
• Start date: May 1, 2001
• Est. completion date: April 24, 2019

Study information

• Verified date: April 24, 2019
• Source: National Institutes of Health Clinical Center (CC)
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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....

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 189
• Est. completion date: April 24, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

• 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

Related Conditions & MeSH terms

• Motor Neuron Disease
• Primary Lateral Sclerosis
• Sclerosis

Locations

Country	Name	City	State
United States	National Institutes of Health Clinical Center, 9000 Rockville Pike	Bethesda	Maryland

Sponsors (1)

Lead Sponsor	Collaborator
National Institute of Neurological Disorders and Stroke (NINDS)

Country where clinical trial is conducted

United States, 

References & Publications (3)

Auger RG. AAEM minimonograph #44: diseases associated with excess motor unit activity. Muscle Nerve. 1994 Nov;17(11):1250-63. Review. — View Citation

Bentley S. Exercise-induced muscle cramp. Proposed mechanisms and management. Sports Med. 1996 Jun;21(6):409-20. Review. — View Citation

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

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.