Registry for patients with Spinal Muscular Atrophy in the UK
Spinal Muscular Atrophy Patient Registry of the United Kingdom and Ireland
Newcastle University · NCT04292574
This study is collecting information from people with Spinal Muscular Atrophy to better understand their experiences and improve future treatments.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 800 (estimated) |
| Sex | All |
| Sponsor | Newcastle University (other) |
| Locations | 1 site (Newcastle upon Tyne) |
| Trial ID | NCT04292574 on ClinicalTrials.gov |
What this trial studies
This registry collects data from patients diagnosed with Spinal Muscular Atrophy (SMA) through online questionnaires. Participants provide information about their condition, including motor function, daily life experiences, and quality of life. The registry collaborates with clinical networks to link patient-reported outcomes with clinical data, enhancing understanding and care for SMA patients. The initiative aims to create a comprehensive database that can inform future research and treatment strategies.
Who should consider this trial
Good fit: Ideal candidates for this registry are individuals with a confirmed or pending diagnosis of Spinal Muscular Atrophy.
Not a fit: Patients without a confirmed diagnosis of Spinal Muscular Atrophy may not benefit from participation in this registry.
Why it matters
Potential benefit: If successful, this registry could lead to improved understanding and management of Spinal Muscular Atrophy, ultimately enhancing patient care.
How similar studies have performed: Other patient registries for SMA have shown success in improving patient care and informing research, indicating that this approach is promising.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * All patients with a confirmed SMA diagnosis (or pending diagnosis) are eligible for inclusion. Diagnosis will be confirmed via genetic testing results Exclusion Criteria: * There are no exclusion criteria for the registry
Where this trial is running
Newcastle upon Tyne
- John Walton Muscular Dystrophy Research Centre — Newcastle upon Tyne, United Kingdom (RECRUITING)
Study contacts
- Principal investigator: Chiara Marini-Bettolo, MD, PhD — Newcastle University
- Study coordinator: Patient Registry manager and curator
- Email: smaregistry@newcastle.ac.uk
- Phone: 0191 2418640
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: Spinal Muscular Atrophy, SMA, Neuromuscular Diseases, Motor Neuron Disease, Bulbo-Spinal Atrophy, X-Linked, Kennedy Disease, Spinal Muscular Atrophy with Respiratory Distress 1, Distal Spinal Muscular Atrophy