Last reviewed · How we verify
NCT04292574
UK SMA Patient Registry
trial testing Patient Registry in Spinal Muscular Atrophy in 800 participants. Currently enrolling.
31 May 2025
Quick facts
| Lead sponsor | Newcastle University |
|---|---|
| Status | Recruiting now |
| Study type | OBSERVATIONAL |
| Enrollment | 800 |
| Start date | 13 July 2008 |
| Primary completion | 31 May 2025 |
| Estimated completion | 31 May 2025 |
| Sites | 1 location across United Kingdom |
Drugs / interventions tested
- Patient Registry
Conditions studied
- Spinal Muscular Atrophy — all drugs for Spinal Muscular Atrophy →
- SMA — all drugs for SMA →
Sponsor
Newcastle University
Who can join
Eligibility, any sex, with Spinal Muscular Atrophy or SMA. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
Spinal muscular atrophy (SMA) is a form of motor neuron disease, most commonly caused by a mutation in the survival motor neuron 1 gene (SMN1) which results in a wide disease spectrum affecting children and adults. It is an autosomal recessive disorder and is therefore caused by inheritance of a mutated gene from each parent. All forms of SMA have an estimated combined incidence of 1 in 6,000 to 1 in 10,000 live births, with a carrier frequency of 1/40 to 1/60. The patient registry aims to facilitate a questionnaire-based research study in order to better characterise and understand the disease in the UK and in Ireland. Entry is via self-registration over a secure internet connection (https://www.sma-registry.org.uk/). Online, patients are asked to read an information sheet about the research project and then indicate their consent to demonstrate willingness to participate. Following online consent, subjects will be entered into the registry. This is an on-going database and all participants are invited to update their information on a biannual basis.
Publications & conference data
No peer-reviewed publications indexed yet for this trial.
Verify or expand the search:
- PubMed search for NCT04292574
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
Other trials of Patient Registry
Trials testing the same drug.
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- NCT05848271 — Natural History Study of Patients with HPDL Mutations · recruiting
Other recruiting trials for Spinal Muscular Atrophy
Currently open trials in the same condition.
- NCT07478172 — Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease · NA · recruiting
- NCT06955897 — Characterizing Perceived Physical Fatigability in Nusinersen-treated SMA · recruiting
- NCT06396325 — A Registry Based Randomized-Controlled Trial of an Upper Limb Exergaming Intervention for Children and Adolescents With · NA · recruiting
- NCT06288230 — An Open Label Study of Gene Therapy Product (Vesemnogene Lantuparvovec) in Spinal Muscular Atrophy · Phase 1, PHASE2 · recruiting
- NCT06321965 — Characterization of New Phenotypes of Patients With Spinal Muscular Atrophy Treated With SMN Restoring Therapy · NA · recruiting
Other Newcastle University trials
Trials by the same sponsor.
- NCT07206433 — Transient Ischemic Attack (TIA) and Spoken Language · not yet recruiting
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- NCT06551012 — Neuromuscular Fatigability in Individuals With Heart Failure · NA · not yet recruiting
- NCT07049874 — The Effect of Huel on Markers of Inflammation · NA · active not recruiting
- NCT05713474 — Micro-EMG: A Novel Multi-electrode System for Intramuscular Imaging · recruiting
Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT04292574 (US National Library of Medicine, public domain)
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by Newcastle University
- Last refreshed: 23 July 2024
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04292574.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing