Screening and Natural History: Primary Lateral Sclerosis... | NCT00015444

Studies
Screening and Natural History: Primary Lateral Sclerosis and Related Disorders

NCT00015444

Sponsor

National Institute of Neurological Disorders and Stroke (NINDS)

Status

Completed

Last Update Posted

Dec 16, 2019Actual

Enrollment

189Actual

Phase

Not provided

Conditions

Primary Lateral Sclerosis

Interventions

Not provided

Countries

United States

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

ID	Type	Description	Link
01-N-0145

Status Module

Record Verification Date

Apr 24, 2019

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

May 1, 2001

Primary Completion Date

Not provided

Completion Date

Apr 24, 2019

First Submitted Date

Apr 18, 2001

First Submission Date that Met QC Criteria

Apr 18, 2001

First Posted Date

Apr 19, 2001Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Dec 13, 2019

Last Update Posted Date

Dec 16, 2019Actual

Description Module

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....

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.

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

No data available

No data is available for this block.

Enrollment

189Actual

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
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.

Secondary Outcomes

Not provided

Other Outcomes

Not provided

Eligibility Module

Eligibility 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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

Not provided

Standard Ages

AdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Mary Kay Floeter, M.D.	National Institute of Neurological Disorders and Stroke (NINDS)	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
National Institutes of Health Clinical Center, 9000 Rockville Pike		Bethesda	Maryland	20892	United States

References Module

Citations

PubMed Identifier	Type	Citation	Retractions
8632965	Background	Leclerc KM, Landry FJ. Benign nocturnal leg cramps. Current controversies over use of quinine. Postgrad Med. 1996 Feb;99(2):177-8, 181-4.
8784961	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.

ID	Term
D016472	Motor Neuron Disease
D009128	Muscle Spasticity
D000690	Amyotrophic Lateral Sclerosis
D020820	Dyskinesias
D020386	Isaacs Syndrome

ID	Term
D019636	Neurodegenerative Diseases
D009422	Nervous System Diseases
D009468	Neuromuscular Diseases
D009135	Muscular Diseases