Amyotrophic Lateral SclerosisNCT05725759
Rehabilitation in SOD1 ALS Treated With Tofersen
An observational study of Amyotrophic Lateral Sclerosis and Lou Gehrig Disease, run by Washington University School of Medicine at 1 site in United States.
Trial summary
The primary objective of this study is to document and describe the effects of a personalized rehabilitation program for patients with SOD1 ALS participating in the tofersen expanded access program. Participants currently receiving tofersen treatment will be referred to outpatient physical and/or occupational therapy. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyotrophic Lateral Sclerosis, Lou Gehrig Disease, Familial Amyotrophic Lateral Sclerosis
- What is being tested?
- Rehabilitation
- Type of treatment
- Lifestyle or therapy programme
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- 8 Nov 2022to Dec 2024
- Phase
- Observational
- 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
Study population: Patients with SOD1 ALS
Who may be able to join
- Confirmed diagnosis of ALS with an SOD1 mutation
- Current enrollment in the tofersen expanded access program
- Age greater than 18 years
- Medically able to participate in outpatient physical therapy and/or home-based rehabilitation, as determined by the treating health care provider
Who cannot join
- Any comorbidities or conditions that, in the opinion of the treating healthcare provider, would unacceptably increase the risk of participation in outpatient physical therapy and/or home-based rehabilitation
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: The Effects of Rehabilitation on Functional Outcomes in Patients With SOD1 ALS Treated With Tofersen
The primary objective of this study is to document and describe the effects of a personalized rehabilitation program for patients with SOD1 ALS participating in the tofersen expanded access program. Participants currently receiving tofersen treatment will be referred to outpatient physical and/or occupational therapy. Participants will have an initial assessment performed and an individualized rehabilitation program will be prescribed. Each participant is encouraged to follow the prescribed recommendations that will include scheduled outpatient therapy sessions, functional assessments, and/or a home-based rehabilitation program. Functional assessments will be done at a minimum of every three months.
- Conditions
- Amyotrophic Lateral Sclerosis, Lou Gehrig Disease, Familial Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Motor Neuron Disease, Familial
- Being tested
- Rehabilitation
- 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.
RehabilitationBehavioural
Also called physical therapy, occupational therapy
Particapants currently receiving tofersen treatment will be referred to outpatient physical and/or occupational therapy.
| Study group | Role | What they receive |
|---|---|---|
| Rehabilitation Arm | Participants will participate in outpatient physical and/or occupational therapies while participating in the tofersen early access program (EAP) Receives: Behavioral: Rehabilitation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS)
ALSFRS-R measures functional disease severity in four functional domains, bulbar function, gross motor skills, fine motor skills, and respiratory. The assessment contains 12 questions scored from 0 (no function) to 4 (full function), with a total possible score of 48, which will indicate the highest level of function.
Measured: 12 months
Secondary outcomes (8)
1. Change from baseline in Handheld Dynamometry (HHD)
Quantitative muscle strength will be evaluated using HHD of multiple muscles using standard participant positioning.
Measured: 12 months
2. Change from baseline in Rasch-Built Overall Amyotrophic Lateral Sclerosis Functional Rating (ROADS)
ROADS measures disability across multiple daily activities. For each question, response options are as follows: 0=unable to perform; 1=abnormal, able to perform but with difficulty; and 2=normal, able to perform without difficulty.
Measured: 12 months
3. Change from baseline in ALS Assessment Questionnaire, 5-item (ALSAQ-5)
Quality of life will be measured using the 5-item Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ-5) patient-reported outcome (PRO)
Measured: 12 months
Change from baseline in motor Functional Independence Measure (motor FIM)
Measures level of disability and indicates level of assistance required to perform activities of daily living.
Measured: 12 months
Change from baseline in Modified Ashworth Scale (MAS)
The MAS is 6-point scale that that grades spasticity, scored from 0 to 4, where 0 is normal muscle tone and 4 is severe increase in muscle tone
Measured: 12 months
Change from baseline in Slow Vital Capacity (SVC)
Measured: 12 months
Change from baseline in Maximal Inspiratory Pressure (MIP)
Measured: 12 months
Change from baseline in Fatigue Severity Scale (FSS)
FSS measures how fatigue interferes with activities. It is a 9-item questionnaire scored on a 7-point scale, 1 = strongly disagree to 7 = strongly agree, and the higher the score equal to the greater the severity of fatigue
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Feb 2023
- Study start8 Nov 2022
- Primary completionDec 2024 (estimated)
- Study completionDec 2024 (estimated)
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
- Observational
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Washington University School of Medicine
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Sean E Smith, Washington University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05725759
- Sponsor's protocol ID
- 202208170
- Status last verified
- Jan 2024
- First submitted
- 1 Feb 2023
- Last updated
- 30 Jan 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Jan 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Effects of Rehabilitation on Functional Outcomes in Patients With SOD1 ALS Treated With Tofersen
- ClinicalTrials.gov ID
- NCT05725759
- Acronym / sponsor ID
- 202208170
- Conditions
- Amyotrophic Lateral Sclerosis, Lou Gehrig Disease, Familial Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Motor Neuron Disease, Familial
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Washington University School of MedicineUniversity, hospital or other
- Study start
- 8 Nov 2022 (actual)
- Primary completion
- Dec 2024 (estimated)
- Study completion
- Dec 2024 (estimated)
- First posted
- 13 Feb 2023 (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.
The primary objective of this study is to document and describe the effects of a personalized rehabilitation program for patients with SOD1 ALS participating in the tofersen expanded access program. Participants currently receiving tofersen treatment will be referred to outpatient physical and/or occupational therapy. Participants will have an initial assessment performed and an individualized rehabilitation program will be prescribed. Each participant is encouraged to follow the prescribed recommendations that will include scheduled outpatient therapy sessions, functional assessments, and/or a home-based rehabilitation program. Functional assessments will be done at a minimum of every three months.
Study design
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Rehabilitation Arm | – | Behavioral: RehabilitationParticipants will participate in outpatient physical and/or occupational therapies while participating in the tofersen early access program (EAP) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients with SOD1 ALS
Key inclusion criteria (4 listed)
- Confirmed diagnosis of ALS with an SOD1 mutation
- Current enrollment in the tofersen expanded access program
- Age greater than 18 years
- Medically able to participate in outpatient physical therapy and/or home-based rehabilitation, as determined by the treating health care provider
Key exclusion criteria (1 listed)
- Any comorbidities or conditions that, in the opinion of the treating healthcare provider, would unacceptably increase the risk of participation in outpatient physical therapy and/or home-based rehabilitation
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 |
|---|---|---|
| RehabilitationAlso: physical therapy, occupational therapyParticapants currently receiving tofersen treatment will be referred to outpatient physical and/or occupational therapy. | Behavioural | Rehabilitation Arm |
Primary outcome
Change from baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS)
Time frame: 12 months
ALSFRS-R measures functional disease severity in four functional domains, bulbar function, gross motor skills, fine motor skills, and respiratory. The assessment contains 12 questions scored from 0 (no function) to 4 (full function), with a total possible score of 48, which will indicate the highest level of function.
8 secondary outcomes
1. Change from baseline in Handheld Dynamometry (HHD)
Time frame: 12 months
2. Change from baseline in Rasch-Built Overall Amyotrophic Lateral Sclerosis Functional Rating (ROADS)
Time frame: 12 months
3. Change from baseline in ALS Assessment Questionnaire, 5-item (ALSAQ-5)
Time frame: 12 months
Change from baseline in motor Functional Independence Measure (motor FIM)
Time frame: 12 months
Change from baseline in Modified Ashworth Scale (MAS)
Time frame: 12 months
Change from baseline in Slow Vital Capacity (SVC)
Time frame: 12 months
Change from baseline in Maximal Inspiratory Pressure (MIP)
Time frame: 12 months
Change from baseline in Fatigue Severity Scale (FSS)
Time frame: 12 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Jan 2024.
- Washington University ALS Center websiteLink provided by the sponsor.
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Amyotrophic Lateral Sclerosis and Lou Gehrig 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 4 inclusion and 1 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: status not confirmed, as of the sponsor's last update on 30 Jan 2024. 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
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?
Change from baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS) (time frame: 12 months). 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 30 Jan 2024.