免费获得国外相关药品，最快 1 个工作日回馈药物信息

出境医 / 临床实验 / Sinemet for Spasticity and Function in Amyotrophic Lateral Sclerosis and Primary Lateral Sclerosis (ALS and PLS)

Sinemet for Spasticity and Function in Amyotrophic Lateral Sclerosis and Primary Lateral Sclerosis (ALS and PLS)

Study Description
Brief Summary:
Motivated by the success of dopaminergic drugs in treating rigidity associated with Parkinson's disease, some neurologists have used carbidopa-levodopa (Sinemet) to attempt to improve spasticity in ALS and PLS patients. However, data on the efficacy of carbidopa/levodopa is limited. Given the limited data and potential to improve the quality of life of these patients, the effectiveness of carbidopa-levodopa in ALS and PLS patients with severe spasticity should be studied. The investigators hypothesis is that administration of carbidopa-levodopa will improve spasticity in ALS and PLS patients.

Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis Motor Neuron Disease Drug: carbidopa-levodopa Drug: Placebo Oral Tablet Phase 1

Study Design
Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 15 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Intervention Model Description: Identified participants will be randomized to receive either placebo or carbidopa-levodopa for a period of three weeks before crossing over to the other arm of the study. The two periods will be separated by a one day washout period.
Masking: Double (Participant, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Sinemet in ALS and PLS
Actual Study Start Date : May 13, 2019
Estimated Primary Completion Date : May 2022
Estimated Study Completion Date : May 2022
Arms and Interventions
Arm Intervention/treatment
Active Comparator: carbidopa-levodopa
Each tablet of carbidopa-levodopa in this study will be equivalent to half of a standard carbidopa-levodopa 25/100mg tablet. Participants will take one tablet three times a day for the first week of the study period, increasing to two tablets three times a day for the remainder of the study period.
 Drug: carbidopa-levodopa
Motivated by the success of dopaminergic drugs in treating rigidity associated with Parkinson's disease, some neurologists have used carbidopa-levodopa to attempt to improve spasticity in ALS and PLS patients.
Other Name: Sinemet
Placebo Comparator: Placebo
Participants will take one placebo tablet three times a day for the first week of the study period, increasing to two tablets three times a day for the remainder of the study period.
 Drug: Placebo Oral Tablet
Placebo will be given to maintain blinding of participants and study team.
Outcome Measures
Primary Outcome Measures :
  1. Visual Analog Scale - Change of spasticity severity from baseline with treatment and placebo [ Time Frame: Weekly from screening to end of study (six weeks) ]
    Numerical rating scale from 0-10, where 0 is no spasticity and 10 is worst possible spasticity


Secondary Outcome Measures :
  1. Visual Analog Scale - Change of pain severity from baseline with treatment and placebo [ Time Frame: Weekly from screening to end of study (six weeks) ]
    Numerical rating scale from 0-10, where 0 is no pain and 10 is worst possible pain

  2. Visual Analog Scale - Change of muscle spasm severity from baseline with treatment and placebo [ Time Frame: Weekly from screening to end of study (six weeks) ]
    Numerical rating scale from 0-10, where 0 is no muscle spasm and 10 is worst possible muscle spasm

  3. Strength [ Time Frame: At screening, at 3 weeks (following first arm), and at 6 weeks (following second arm) ]
    Medical Research Council scale for muscle strength which grades power on a scale of 0 to 5 in relation to the maximum expected for that muscle, 0 being no movement observed to 5 being muscle contracts normally against full resistance.

  4. Spasticity [ Time Frame: At screening, at 3 weeks (following first arm), and at 6 weeks (following second arm) ]
    The Ashworth scale measures severity of spasticity on a scale of 1 to 5, where 1 is normal muscle tone and 5 is a rigid limb.

  5. Upper extremity function [ Time Frame: At screening, at 3 weeks (following first arm), and at 6 weeks (following second arm) ]
    9-hole peg test

  6. Lower extremity function:10-meter Walk Test [ Time Frame: At screening, at 3 weeks (following first arm), and at 6 weeks (following second arm) ]
    10-meter Walk Test is a performance measure used to assess walking speed in meters per second over a short distance.

  7. Lower extremity function: Timed Up and Go (TUG) Test [ Time Frame: At screening, at 3 weeks (following first arm), and at 6 weeks (following second arm) ]
    The TUG test measures the time that a person takes to rise from a chair, walk three meters, turn around, walk back to the chair, and sit down to asses a person's mobility and lower extremity function.


Eligibility Criteria
Layout table for eligibility information
Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Diagnosis of ALS or PLS
  • Age greater than 18 years
  • Clinically significant spasticity.

Exclusion Criteria:

  • Individuals currently taking carbidopa-levodopa or with known hypersensitivity of any component of carbidopa-levodopa
  • Narrow-angle glaucoma
  • Current use of a non-selective monoamine oxidase inhibitor (MAOI)
  • History of malignant melanoma or suspicious skin lesions
  • History of depression, suicidal ideation, or psychosis
  • History of myocardial infarction, ventricular arrhythmia, or severe cardiopulmonary disease
  • Uncontrolled hypertension
  • Asthma
  • Renal disease
  • Hepatic disease
  • Endocrine disease
  • History of peptic ulcer
  • Pregnant and/or breastfeeding
  • Current participation in another interventional study
Contacts and Locations

Locations
Layout table for location information
United States, Missouri
Washington University School of Medicine
Saint Louis, Missouri, United States, 63110
Sponsors and Collaborators
Washington University School of Medicine
Investigators
Layout table for investigator information
Principal Investigator: Timothy M Miller, MD, PhD Washington University School of Medicine
Tracking Information
First Submitted Date  ICMJE April 10, 2019
First Posted Date  ICMJE April 26, 2019
Last Update Posted Date June 7, 2021
Actual Study Start Date  ICMJE May 13, 2019
Estimated Primary Completion Date May 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 24, 2019) 		Visual Analog Scale - Change of spasticity severity from baseline with treatment and placebo [ Time Frame: Weekly from screening to end of study (six weeks) ]
Numerical rating scale from 0-10, where 0 is no spasticity and 10 is worst possible spasticity
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: April 24, 2019)
  • Visual Analog Scale - Change of pain severity from baseline with treatment and placebo [ Time Frame: Weekly from screening to end of study (six weeks) ]
    Numerical rating scale from 0-10, where 0 is no pain and 10 is worst possible pain
  • Visual Analog Scale - Change of muscle spasm severity from baseline with treatment and placebo [ Time Frame: Weekly from screening to end of study (six weeks) ]
    Numerical rating scale from 0-10, where 0 is no muscle spasm and 10 is worst possible muscle spasm
  • Strength [ Time Frame: At screening, at 3 weeks (following first arm), and at 6 weeks (following second arm) ]
    Medical Research Council scale for muscle strength which grades power on a scale of 0 to 5 in relation to the maximum expected for that muscle, 0 being no movement observed to 5 being muscle contracts normally against full resistance.
  • Spasticity [ Time Frame: At screening, at 3 weeks (following first arm), and at 6 weeks (following second arm) ]
    The Ashworth scale measures severity of spasticity on a scale of 1 to 5, where 1 is normal muscle tone and 5 is a rigid limb.
  • Upper extremity function [ Time Frame: At screening, at 3 weeks (following first arm), and at 6 weeks (following second arm) ]
    9-hole peg test
  • Lower extremity function:10-meter Walk Test [ Time Frame: At screening, at 3 weeks (following first arm), and at 6 weeks (following second arm) ]
    10-meter Walk Test is a performance measure used to assess walking speed in meters per second over a short distance.
  • Lower extremity function: Timed Up and Go (TUG) Test [ Time Frame: At screening, at 3 weeks (following first arm), and at 6 weeks (following second arm) ]
    The TUG test measures the time that a person takes to rise from a chair, walk three meters, turn around, walk back to the chair, and sit down to asses a person's mobility and lower extremity function.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Sinemet for Spasticity and Function in Amyotrophic Lateral Sclerosis and Primary Lateral Sclerosis
Official Title  ICMJE Sinemet in ALS and PLS
Brief Summary Motivated by the success of dopaminergic drugs in treating rigidity associated with Parkinson's disease, some neurologists have used carbidopa-levodopa (Sinemet) to attempt to improve spasticity in ALS and PLS patients. However, data on the efficacy of carbidopa/levodopa is limited. Given the limited data and potential to improve the quality of life of these patients, the effectiveness of carbidopa-levodopa in ALS and PLS patients with severe spasticity should be studied. The investigators hypothesis is that administration of carbidopa-levodopa will improve spasticity in ALS and PLS patients.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: Randomized
Intervention Model: Crossover Assignment
Intervention Model Description:
Identified participants will be randomized to receive either placebo or carbidopa-levodopa for a period of three weeks before crossing over to the other arm of the study. The two periods will be separated by a one day washout period.
Masking: Double (Participant, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE
  • Amyotrophic Lateral Sclerosis
  • Motor Neuron Disease
Intervention  ICMJE
  • Drug: carbidopa-levodopa
    Motivated by the success of dopaminergic drugs in treating rigidity associated with Parkinson's disease, some neurologists have used carbidopa-levodopa to attempt to improve spasticity in ALS and PLS patients.
    Other Name: Sinemet
  • Drug: Placebo Oral Tablet
    Placebo will be given to maintain blinding of participants and study team.
Study Arms  ICMJE
  • Active Comparator: carbidopa-levodopa
    Each tablet of carbidopa-levodopa in this study will be equivalent to half of a standard carbidopa-levodopa 25/100mg tablet. Participants will take one tablet three times a day for the first week of the study period, increasing to two tablets three times a day for the remainder of the study period.
    Intervention: Drug: carbidopa-levodopa
  • Placebo Comparator: Placebo
    Participants will take one placebo tablet three times a day for the first week of the study period, increasing to two tablets three times a day for the remainder of the study period.
    Intervention: Drug: Placebo Oral Tablet
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Enrolling by invitation
Estimated Enrollment  ICMJE
 (submitted: April 24, 2019) 		15
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE May 2022
Estimated Primary Completion Date May 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Diagnosis of ALS or PLS
  • Age greater than 18 years
  • Clinically significant spasticity.

Exclusion Criteria:

  • Individuals currently taking carbidopa-levodopa or with known hypersensitivity of any component of carbidopa-levodopa
  • Narrow-angle glaucoma
  • Current use of a non-selective monoamine oxidase inhibitor (MAOI)
  • History of malignant melanoma or suspicious skin lesions
  • History of depression, suicidal ideation, or psychosis
  • History of myocardial infarction, ventricular arrhythmia, or severe cardiopulmonary disease
  • Uncontrolled hypertension
  • Asthma
  • Renal disease
  • Hepatic disease
  • Endocrine disease
  • History of peptic ulcer
  • Pregnant and/or breastfeeding
  • Current participation in another interventional study
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03929068
Other Study ID Numbers  ICMJE Sinemet-001
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Washington University School of Medicine
Study Sponsor  ICMJE Washington University School of Medicine
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Timothy M Miller, MD, PhD Washington University School of Medicine
PRS Account Washington University School of Medicine
Verification Date June 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

治疗医院

新药特效药

治疗案例

前沿医讯