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New treatment option for Spinal Muscular Atrophy (SMA)
Official title VRehab-SMA Phase 1.2
ClinicalTrials.gov ID: NCT07578454
What this study is testing
- What it's testing
- Spinal muscular atrophy is a genetic disorder characterized by progressive muscle weakness, severely impacting patients' motor abilities. Several disease modifying therapies have been developed to treat Spinal muscular atrophy which have led to new disease trajectories .
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 6 to 18
You may be able to join if
- Cohort 1
- Person with SMA
- A person living with genetically confirmed SMA aged from 12-18 years
- A good understanding of English or someone who can provide a good understanding of English for me to complete the survey
- A young person living with SMA from 12-15 years of age and who's caregiver/legal guardian gives consent
You likely can't join if
- No exclusion criteria for Cohort 1, other than meeting the inclusion criteria Cohort 2 Inclusion Criteria: Participant with SMA aged 6-10 years
- Genetically confirmed SMA
- A comprehensive understanding of English
- Treated with any disease-modifying therapy post-symptomatically
- Number of SMN2 copies available
- Functional status available
See the full eligibility criteria
- Cohort 1
- Person with SMA
- A person living with genetically confirmed SMA aged from 12-18 years
- A good understanding of English or someone who can provide a good understanding of English for me to complete the survey
- A young person living with SMA from 12-15 years of age and who's caregiver/legal guardian gives consent
- A young person living with SMA from 16-18 years of age who provide their own consent Caregiver/ Legal Guardian
- A caregiver (legal guardian) of child/young person living with SMA aged from 6-18 years old
- A good understanding of English or someone who can provide a good understanding of English for me to complete the survey
- No exclusion criteria for Cohort 1, other than meeting the inclusion criteria Cohort 2 Inclusion Criteria: Participant with SMA aged 6-10 years
- Genetically confirmed SMA
- A comprehensive understanding of English
- Treated with any disease-modifying therapy post-symptomatically
- Number of SMN2 copies available
- Functional status available
- Age: Participants between 6-10 years at Visit 1 (inclusion)
- Parent(s)/legal guardian(s)/caregiver(s) able to provide written informed consent and child able to provide assent prior to participation in the study Participant with SMA aged 11-15 years
- Genetically confirmed SMA
- A comprehensive understanding of English
- Treated with any disease-modifying therapy post-symptomatically
- Number of SMN2 copies available
- Functional status available
- Age: Participants between 11-15 years at Visit 1 (inclusion)
- Parent(s)/legal guardian(s)/caregiver(s) able to provide written informed consent and child able to provide assent prior to participation in the study Participant with SMA aged 16-18 years
- Genetically confirmed SMA
- A comprehensive understanding of English
- Treated with any disease-modifying therapy post-symptomatically
- Number of SMN2 copies available
- Functional status available
- Age: Participants between 16-18 years at Visit 1 (inclusion)
- Able to provide written informed consent Exclusion Criteria: Any acute or chronic condition which, according to the investigator, significantly interferes with the use of the device (example: Upper limbs injuries...
The study team makes the final eligibility decision.
Where it's taking place
- Oxford, United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 6 years to 18 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Oxford, United Kingdom. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.