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NCT04602195: NusiMFM
Responsiveness and Validation Study of MFM-32 in SMA Patients Treated With Nusinersen
trial in Spinal Muscular Atrophy in 33 participants. Completed in 10 November 2023.
10 November 2023
Quick facts
| Lead sponsor | Hospices Civils de Lyon |
|---|---|
| Status | Completed |
| Study type | OBSERVATIONAL |
| Enrollment | 33 |
| Start date | 19 January 2021 |
| Primary completion | 10 November 2023 |
| Estimated completion | 10 November 2023 |
| Sites | 10 locations across France |
Conditions studied
- Spinal Muscular Atrophy — all drugs for Spinal Muscular Atrophy →
Sponsor
Hospices Civils de Lyon — full company profile →
Who can join
Adults 2 to 6, any sex, with Spinal Muscular Atrophy. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
The Motor Function Measure (MFM), a reliable tool assessing motor function and its progression in most neuromuscular diseases, is widely used in France in many teams. It can be used regardless of the severity of the motor impairment or the ambulatory status of the patient, allowing its use throughout the whole follow-up period of the patient, even in case of the loss of walking. Two versions of the MFM exist, one composed of 32 items originally validated for patients from 6 years old (MFM-32) and a shorter version composed of 20 items originally validated for patients between 2 and 6 years old (MFM-20). In order to prove the possible use of MFM-32 as early as the age of 2 years to validly and reliably monitor the evolution of the motor function of children treated with Nusinersen, we propose in this project to study the sensitivity to treatment-induced change of MFM-32 and the validity of the scale in this population.
Publications & conference data
1 peer-reviewed publication reference this trial (live from Europe PMC):
-
Pharmacological Therapies of Spinal Muscular Atrophy: A Narrative Review of Preclinical, Clinical-Experimental, and Real-World Evidence.
Crisafulli S, Boccanegra B, Vitturi G, Trifirò G, et al · · 2023 · cited 11× · PMID 37891814 · DOI 10.3390/brainsci13101446
Verify or expand the search:
- PubMed search for NCT04602195
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
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 Hospices Civils de Lyon trials
Trials by the same sponsor.
- NCT07569536 — Efficacy of the Alpha 2 Agonist Dexmedetomidine for Sympathetic Deactivation in REfractory Septic Shock · Phase 3 · not yet recruiting
- NCT07529314 — Evaluating Interventional Radiology for Cancer Pain Management · NA · not yet recruiting
- NCT07273929 — Comparative Study of the Effectiveness of Three Access Routes for Implanting an Electronic Intracardiac Device · Phase 3 · not yet recruiting
- NCT07474532 — Attitudes and Beliefs Related to Benzodiazepine Deprescribing · not yet recruiting
- NCT07313007 — Assessment of Gut Microbiota-Derived Amino Acid Metabolite Production in Patients With MASLD · NA · not yet 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 NCT04602195 (US National Library of Medicine, public domain)
- Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by Hospices Civils de Lyon
- Last refreshed: 21 December 2023
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/NCT04602195.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing