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| ID | Type | Description | Link |
|---|---|---|---|
| PID17165 | Other Identifier | University Oxford Hospitals NHS Foundation Trust |
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| Name | Class |
|---|---|
| Oxford Brookes University | OTHER |
| Oxford University Hospitals NHS Trust | OTHER |
| ACE SMA Charity | UNKNOWN |
| Roche Products Limited |
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The goal of this study is to investigate the acceptability, feasibility, safety and efficacy of an optimized rehabilitation program for treated patients with spinal muscular atrophy (SMA) compared to the current rehabilitation program in the United Kingdom. The aim is to provide patients with more hands on physiotherapy and access to rehabilitation devices at home to support parents currently providing rehabilitation on their own.
The main study objective is to investigate whether an optimized rehabilitation program would be accepted and is feasible within the SMA patient community.
The optimized program will comprise of goal orientated rehabilitation with more frequent hands on physiotherapy (every 2 weeks) as well as a SMA approved rehabilitation device used at home on a weekly basis, to provide the most appropriate therapy for each individual patient. All study participants will receive the optimized rehabilitation program over 12 months. An external control group will be used to determine the potential efficacy.
Research study visits will take place at baseline, month 6 and month 12. At these visits a general physical exam will be undertaken alongside collecting the participant and carer perception, satisfaction and compliancy of the rehabilitation program. Motor function assessments according to age and ambulatory status will be assessed to help evaluate potential therapeutic benefits.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Treated cohort | Other |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Optimized rehabilitation program | Other | Consisting of: goal-oriented rehabilitation, hands on physiotherapy sessions and home use of SMA approved rehabilitation device. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Acceptability of an optimized rehabilitation program. Measured as number of eligible patients who accepted to participate in the study compared to the number of eligible patients who have refused to participate. Endpoint: 70% of acceptability. | Number of eligible patients who accepted to participate in the study compared to the number of eligible patients who have refused to participate. | Baseline visit |
| Feasibility of an optimized rehabilitation program. Measured as the number of patients who complete the study compared to the number of included patients. Endpoint: 60% of feasibility. | Number of patients who complete the study compared to the number of included patients. | Month 12 (end of study) |
| Measure | Description | Time Frame |
|---|---|---|
| Safety of an optimized rehabilitation program. Measured as the number of serious adverse events (SAE) compared to the year before the baseline. | Number of Serious Adverse Events (SAEs). | From baseline to month 12 (end of study) |
| Carer perception and satification measured through the Clinical Global Impression scale - Improvement (CGI I). Comparison of CGI-S score with CGI-I score of the treated group. |
| Measure | Description | Time Frame |
|---|---|---|
| The Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND): maximum score is 64 points. Measuring a change in functional total score between the treated study cohort and a control dataset. | Evaluation potential therapeutic benefits through assessment of motor functional scales. | Baseline, Month 6, Month 12 (end of study). |
Patient participant Eligibility Criteria:
Inclusion Criteria:
Exclusion Criteria:
The participant may not enter the trial if ANY of the following apply:
Carer (considered as participant) Eligibility Criteria:
Inclusion Criteria:
Exclusion Criteria:
• No exclusion criteria.
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| Name | Affiliation | Role |
|---|---|---|
| Charlotte Lilien | University of Oxford, Department of Paediatrics | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Oxford Brookes University | Oxford | Oxfordshire | OX3 0BP | United Kingdom |
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| UNKNOWN |
| Scholar Rock, Inc. | INDUSTRY |
| Biohaven Therapeutics Ltd. | INDUSTRY |
| Rally Round Rupert Charity | UNKNOWN |
All participants will receive the optimized rehabilitation program (intervention). An external control group will be used to determine the potential efficacy.
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Potential effects of the optimized rehabilitation program on patients experience. |
| Month 6 and Month 12 (end of study). |
| Carer perception and satification measured through the Clinical Global Impression scale - Severity (CGI-S). Comparison of CGI-S score with CGI-I score of the treated group. | Potential effects of the optimized rehabilitation program on patients experience. | Baseline visit: CGI-S. |
| The Hammersmith Infant Neurological Examination section 2 (HINE-2): maximum score is 26 points. Measuring a change in functional total score between the treated study cohort and a control dataset. | Evaluation potential therapeutic benefits through assessment of motor functional scales. | Baseline, Month 6, Month 12 (end of study). |
| Revised Hammersmith Scale (RHS). The maximum score for RHS is 69 points. Measuring a change in functional total score between the treated study cohort and a control dataset. | Evaluation potential therapeutic benefits through assessment of motor functional scales. | Baseline, Month 6, Month 12 (end of study). |
| Hammersmith Functional Motor Scale Expanded (HFMSE) combined assessment. The maximum score is 66 points. Measuring a change in functional total score between the treated study cohort and a control dataset. | Evaluation potential therapeutic benefits through assessment of motor functional scales. | Baseline, Month 6, Month 12 (end of study). |
| World Health organization (WHO) developmental milestones: the final result is the highest achievable motor milestone. Measuring a change in functional total score between the treated study cohort and a control dataset. | Evaluation potential therapeutic benefits through assessment of motor functional scales. | Baseline, Month 6, Month 12 (end of study). |
| Revised upper limb module (RULM): from 30 months of age: maximum score is 37 points. Measuring a change in functional total score between the treated study cohort and a control dataset. | Evaluation potential therapeutic benefits through assessment of motor functional scales. | Baseline, Month 6, Month 12 (end of study). |
| 6 Minute Walk test (6MWT): from 4 years of age. Measuring a change in functional total score between the treated study cohort and a control dataset. | Evaluation potential therapeutic benefits through assessment of motor functional scales. | Baseline, Month 6, Month 12 (end of study). |
| ID | Term |
|---|---|
| D009134 | Muscular Atrophy, Spinal |
| ID | Term |
|---|---|
| D013118 | Spinal Cord Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D016472 | Motor Neuron Disease |
| D019636 | Neurodegenerative Diseases |
| D009468 | Neuromuscular Diseases |
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