Improving Standards of Care and Translational Research in Spinal Muscular Atrophy (SMA)
SMA-REACH
1 other identifier
observational
600
1 country
1
Brief Summary
The SMA REACH UK Network is a national and international partnership between doctors and therapists involved in the care of children and adults with Spinal Muscular Atrophy. This network is supported by Biogen and SMA UK.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for all trials
Started Dec 2013
Longer than P75 for all trials
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
December 17, 2013
CompletedFirst Submitted
Initial submission to the registry
January 30, 2018
CompletedFirst Posted
Study publicly available on registry
May 9, 2018
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 12, 2023
CompletedStudy Completion
Last participant's last visit for all outcomes
August 12, 2023
CompletedAugust 17, 2022
August 1, 2022
9.7 years
January 30, 2018
August 16, 2022
Conditions
Outcome Measures
Primary Outcomes (2)
Physiotherapy assessment using The Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND)
This is a scale used for the assessment of movement and function of very weak infants with SMA type 1. It consists of 16 items of motor function graded 0-4 with a maximum achievable score of 64.
1 year
Physiotherapy assessment using the Test of Infant Motor Performance Screening Items (TIMPSI)
This scale assesses motor performance in infants born pre-term to 4 months of age. It consists of 29 items, with 3 item sets (screening, easy and hard sets).
1 year
Secondary Outcomes (3)
Patient´s perception about the condition, interventions performed and Standards of Care assessed by patient interviews
1 year
Patients perception about the condition, interventions performed and Standards of Care assessed using the Paediatric Outcomes Data Collection Instrument (PODCI)
1 year
Patients perception about the condition, interventions performed and Standards of Care assessed using the Egan Klassifikation Scale (EK2)
1 year
Study Arms (3)
SMA TYPE 1
genetically confirmed SMA
SMA TYPE 2
genetically confirmed SMA
SMA TYPE 3
genetically confirmed SMA, Ambulant and non-ambulant
Eligibility Criteria
Patients with genetically confirmed SMA types 0, I, II and III (ambulant and non-ambulant) followed at each open and recruiting neuromuscular centre.
You may qualify if:
- It will be genetically confirmed SMA
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
Dubowitz Neuromuscular Centre
London, WC1N 1EH, United Kingdom
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Central Study Contacts
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- CROSS SECTIONAL
- Target Duration
- 18 Months
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
January 30, 2018
First Posted
May 9, 2018
Study Start
December 17, 2013
Primary Completion
August 12, 2023
Study Completion
August 12, 2023
Last Updated
August 17, 2022
Record last verified: 2022-08
Data Sharing
- IPD Sharing
- Will not share