Amyotrophic Lateral SclerosisPhase 1ALS and PLSNCT03929068
Sinemet for Spasticity and Function in Amyotrophic Lateral Sclerosis and Primary Lateral Sclerosis
A Phase 1 study testing carbidopa-levodopa in people with Amyotrophic Lateral Sclerosis and Motor Neuron Disease, run by Washington University School of Medicine at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyotrophic Lateral Sclerosis, Motor Neuron Disease
- What is being tested?
- carbidopa-levodopa
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 15 participants
- Where
- 1 siteUnited States
- Study dates
- 13 May 2019to 8 Jul 2022
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of ALS or PLS
- Age greater than 18 years
- Clinically significant spasticity.
Who cannot join
- 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
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- Uncontrolled hypertension
- Asthma
- Renal disease
- Hepatic disease
- Endocrine disease
- History of peptic ulcer
- Pregnant and/or breastfeeding
- Current participation in another interventional study
Trial locations and contacts
1 site in 1 country.
United States1 site
Washington University School of Medicine
St Louis, Missouri, 63110
About this study
Official title: Sinemet in ALS and PLS
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.
- Being tested
- carbidopa-levodopa, Placebo Oral Tablet
- Keywords
- ALS, PLS, Primary Lateral Sclerosis
- Medical subject headings
- Amyotrophic Lateral Sclerosis, Motor Neuron Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
carbidopa-levodopaDrug
Also called Sinemet
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.
Placebo Oral TabletDrug
Placebo will be given to maintain blinding of participants and study team.
| Study group | Role | What they receive |
|---|---|---|
| carbidopa-levodopa | Active comparator | 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. Receives: Drug: carbidopa-levodopa |
| Placebo | Placebo comparator | 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. Receives: Drug: Placebo Oral Tablet |
What is being measured
The primary outcome is the main question the study is designed to answer.
Visual Analog Scale - Change of spasticity severity from baseline with treatment and placebo
Numerical rating scale from 0-10, where 0 is no spasticity and 10 is worst possible spasticity
Measured: Weekly from screening to end of study (six weeks)
Secondary outcomes (7)
Visual Analog Scale - Change of pain severity from baseline with treatment and placebo
Numerical rating scale from 0-10, where 0 is no pain and 10 is worst possible pain
Measured: Weekly from screening to end of study (six weeks)
Visual Analog Scale - Change of muscle spasm severity from baseline with treatment and placebo
Numerical rating scale from 0-10, where 0 is no muscle spasm and 10 is worst possible muscle spasm
Measured: Weekly from screening to end of study (six weeks)
Strength
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.
Measured: At screening, at 3 weeks (following first arm), and at 6 weeks (following second arm)
Spasticity
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.
Measured: At screening, at 3 weeks (following first arm), and at 6 weeks (following second arm)
Upper extremity function
9-hole peg test
Measured: At screening, at 3 weeks (following first arm), and at 6 weeks (following second arm)
Lower extremity function:10-meter Walk Test
10-meter Walk Test is a performance measure used to assess walking speed in meters per second over a short distance.
Measured: At screening, at 3 weeks (following first arm), and at 6 weeks (following second arm)
Lower extremity function: Timed Up and Go (TUG) Test
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.
Measured: At screening, at 3 weeks (following first arm), and at 6 weeks (following second arm)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Apr 2019
- Study start13 May 2019
- Primary completion8 Jul 2022
- Study completion8 Jul 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- More trials run by Washington University School of MedicineThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- 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 blind
- Who is masked
- Participant, Outcomes assessor
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Washington University School of Medicine
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Timothy M Miller, MD, PhD, Principal investigator, Washington University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03929068
- Sponsor's protocol ID
- Sinemet-001
- Acronym
- ALS and PLS
- Status last verified
- Jul 2022
- First submitted
- 10 Apr 2019
- Last updated
- 11 Jul 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Jul 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Sinemet in ALS and PLS
- ClinicalTrials.gov ID
- NCT03929068
- Acronym / sponsor ID
- ALS and PLS / Sinemet-001
- Keywords
- ALS, PLS, Primary Lateral Sclerosis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Washington University School of MedicineUniversity, hospital or other
- Study start
- 13 May 2019 (actual)
- Primary completion
- 8 Jul 2022 (actual)
- Study completion
- 8 Jul 2022 (actual)
- First posted
- 26 Apr 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind (participant, outcomes assessor)
- Number of arms
- 2
- 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.
| Arm | Type | Receives |
|---|---|---|
| carbidopa-levodopa | Active comparator | Drug: carbidopa-levodopaEach 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. |
| Placebo | Placebo comparator | Drug: Placebo Oral TabletParticipants 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Diagnosis of ALS or PLS
- Age greater than 18 years
- Clinically significant spasticity.
Key exclusion criteria (14 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| carbidopa-levodopaAlso: SinemetMotivated 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. | Drug | carbidopa-levodopa |
| Placebo Oral TabletPlacebo will be given to maintain blinding of participants and study team. | Drug | Placebo |
Primary outcome
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
7 secondary outcomes
Visual Analog Scale - Change of pain severity from baseline with treatment and placebo
Time frame: Weekly from screening to end of study (six weeks)
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)
Strength
Time frame: At screening, at 3 weeks (following first arm), and at 6 weeks (following second arm)
Spasticity
Time frame: At screening, at 3 weeks (following first arm), and at 6 weeks (following second arm)
Upper extremity function
Time frame: At screening, at 3 weeks (following first arm), and at 6 weeks (following second arm)
Lower extremity function:10-meter Walk Test
Time frame: At screening, at 3 weeks (following first arm), and at 6 weeks (following second arm)
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)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Jul 2022.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Timothy M Miller, MD, PhD principal investigator
Washington University School of Medicine
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing carbidopa-levodopa in people with Amyotrophic Lateral Sclerosis and Motor Neuron Disease, run by Washington University School of Medicine at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 11 Jul 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Visual Analog Scale - Change of spasticity severity from baseline with treatment and placebo (time frame: Weekly from screening to end of study (six weeks)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Jul 2022.