Spinal Muscular AtrophyNCT05931211
Motor Function and Activity of Daily Life in Spinal Muscular Atrophy
An observational study of Spinal Muscular Atrophy, run by National Taiwan University Hospital at 1 site in Taiwan.
Trial summary
This study will incorporate patients/ caregivers' perspectives to investigate the performance in daily activities of individuals with SMA and how it relates to their motor function abilities. Read more about this study
- Who is it for?
- Ages 3 years and older
- Conditions the study is for
- Spinal Muscular Atrophy
- How many people
- 100 participants
- Where
- 1 siteTaiwan
- Study dates
- 30 Jun 2023to 31 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 3 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disease that causes progressive muscle weakness and atrophy and eventually leads to loss of function.
Who may be able to join
- Having a diagnosis of SMA with genetics confirmed as 5q SMA.
- Above 3 years of age (capable of performing motor assessments).
Who cannot join
- Having severe respiratory and other medical conditions precluding safe participation in standardized motor function tests.
- Having any orthopedic surgery during the past 6 months.
- Women who are pregnant.
Trial locations and contacts
1 site in 1 country.
Taiwan1 site
National Taiwan University Hospital
Taipei, 100
Hsi-Wen Huang, MSc, 886-2-23123456 ext. 270409, lovelyeva39@gmail.com
About this study
Official title: Translation of Motor Function to Daily Activity Performance in Individuals With SMA
This study will incorporate patients/ caregivers' perspectives to investigate the performance in daily activities of individuals with SMA and how it relates to their motor function abilities.
Read the detailed description
Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disease that causes progressive muscle weakness and atrophy and eventually leads to loss of function. Despite recent advances in disease-modifying treatments, SMA can still significantly impact an individual's functioning in daily living and quality of life. This study will incorporate patients/ caregivers' perspectives to investigate the performance in daily activities of individuals with SMA and how it relates to their motor function abilities.
- Conditions
- Spinal Muscular Atrophy
- Medical subject headings
- Muscular Atrophy, Spinal
What is being measured
The primary outcome is the main question the study is designed to answer.
Revised Upper Limb Module
The physiotherapist will perform a serious of motor test to evaluate the patient's upper limb motor function. This test is to observe the manual ability on different tasks in sitting position, including drawing along the path, opening Ziploc container, or lifting weight. And the therapist will score for each item. The Revised Upper Limb Module (RULM) are consist of 19 items. It takes about 15-20 minutes for testing.
Measured: 15-20 minutes
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jul 2023
- Study start30 Jun 2023 (estimated)
- Primary completion30 Jun 2024 (estimated)
- Study completion31 Dec 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Spinal Muscular AtrophyStudies of the same condition, open and under way.
- More trials run by National Taiwan University HospitalThe 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
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- National Taiwan University Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Hsi-Wen Huang, MSc, Principal investigator, National Taiwan University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05931211
- Sponsor's protocol ID
- 202305039RINB
- Status last verified
- May 2023
- First submitted
- 26 Jun 2023
- Last updated
- 7 Jul 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Jul 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Translation of Motor Function to Daily Activity Performance in Individuals With SMA
- ClinicalTrials.gov ID
- NCT05931211
- Acronym / sponsor ID
- 202305039RINB
- Conditions
- Spinal Muscular Atrophy
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- National Taiwan University HospitalUniversity, hospital or other
- Study start
- 30 Jun 2023 (estimated)
- Primary completion
- 30 Jun 2024 (estimated)
- Study completion
- 31 Dec 2024 (estimated)
- First posted
- 5 Jul 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.
This study will incorporate patients/ caregivers' perspectives to investigate the performance in daily activities of individuals with SMA and how it relates to their motor function abilities.
Detailed description
Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disease that causes progressive muscle weakness and atrophy and eventually leads to loss of function. Despite recent advances in disease-modifying treatments, SMA can still significantly impact an individual's functioning in daily living and quality of life. This study will incorporate patients/ caregivers' perspectives to investigate the performance in daily activities of individuals with SMA and how it relates to their motor function abilities.
Study design
- Observational model
- Cohort
- Time perspective
- Cross-sectional
Who may qualify
- Ages
- 3 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disease that causes progressive muscle weakness and atrophy and eventually leads to loss of function.
Key inclusion criteria (2 listed)
- Having a diagnosis of SMA with genetics confirmed as 5q SMA.
- Above 3 years of age (capable of performing motor assessments).
Key exclusion criteria (3 listed)
- Having severe respiratory and other medical conditions precluding safe participation in standardized motor function tests.
- Having any orthopedic surgery during the past 6 months.
- Women who are pregnant.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Revised Upper Limb Module
Time frame: 15-20 minutes
The physiotherapist will perform a serious of motor test to evaluate the patient's upper limb motor function. This test is to observe the manual ability on different tasks in sitting position, including drawing along the path, opening Ziploc container, or lifting weight. And the therapist will score for each item. The Revised Upper Limb Module (RULM) are consist of 19 items. It takes about 15-20 minutes for testing.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Jul 2023.
- Individual participant dataSharing planned: No
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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.
Hsi-Wen Huang, MSc principal investigator
National Taiwan University Hospital
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 Spinal Muscular Atrophy, run by National Taiwan University Hospital at 1 site in Taiwan.
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 2 inclusion and 3 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 7 Jul 2023. 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 Taiwan. 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?
Contact Hsi-Wen Huang, MSc (886-2-23123456 ext. 270409, lovelyeva39@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Revised Upper Limb Module (time frame: 15-20 minutes). 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 7 Jul 2023.