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NCT04587492
Metabolomics of Children With SMA
trial testing Nusinersen in Spinal Muscular Atrophy in 35 participants. Completed in 30 September 2020.
30 September 2020
Quick facts
| Lead sponsor | University Medical Centre Ljubljana |
|---|---|
| Status | Completed |
| Study type | OBSERVATIONAL |
| Enrollment | 35 |
| Start date | 1 January 2017 |
| Primary completion | 30 September 2020 |
| Estimated completion | 30 September 2020 |
| Sites | 2 locations across Slovenia |
Drugs / interventions tested
- Nusinersen (NUSINERSEN) — full drug profile →
Conditions studied
- Spinal Muscular Atrophy — all drugs for Spinal Muscular Atrophy →
Sponsor
University Medical Centre Ljubljana
Who can join
Under 21, any sex, with Spinal Muscular Atrophy. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
The aim of the proposed project is to evaluate whether the metabolome of patients with spinal muscular atrophy (SMA) before the initiation of treatment with nusinersen differs from the metabolome of healthy individuals and whether it changes 14 months after treatment with nusinersen.
Publications & conference data
2 peer-reviewed publications reference this trial (live from Europe PMC):
-
Spinal Muscular Atrophy after Nusinersen Therapy: Improved Physiology in Pediatric Patients with No Significant Change in Urine, Serum, and Liquor 1H-NMR Metabolomes in Comparison to an Age-Matched, Healthy Cohort.
Deutsch L, Osredkar D, Plavec J, Stres B. · · 2021 · cited 26× · PMID 33808177 · DOI 10.3390/metabo11040206 -
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 NCT04587492
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
Other trials of Nusinersen
Trials testing the same drug.
- NCT06555419 — A Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFlex DRx™ System in Adult and Pe · Phase 1 · recruiting
- NCT04576494 — Study of the Functional Effects of Nusinersen in 5q-spinal Muscular Amyotrophy Adults (SMA Type 2 or 3 Forms) · NA · completed
- NCT05067790 — A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spina · Phase 3 · active not recruiting
- NCT05187260 — Antisense Oligonucleotide for Spinal Muscular Atrophy · unknown
- NCT04729907 — A Study to Learn About the Long-Term Safety of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants · Phase 3 · active not 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 University Medical Centre Ljubljana trials
Trials by the same sponsor.
- NCT07466472 — Image-Guided Central Catheter Insertion With Concurrent Assessment of Vascular Wall Integrity · not yet recruiting
- NCT07446244 — Catheter Ablation of Advanced Forms of Atrial Fibrillation · NA · enrolling by invitation
- NCT07109076 — Effect of Immediate Skin-to-Skin Contact on Neonatal Heart Rate Variability After Cesarean Secton · NA · recruiting
- NCT07354880 — Effect of Tirzepatide on Cardiovascular and Metabolic Parameters in Obese Adult Patients With Congenital Heart Disease · Phase 4 · not yet recruiting
- NCT07496736 — Post-exposure Influenza Prophylaxis in a Hospital Setting · Phase 3 · active not 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 NCT04587492 (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 University Medical Centre Ljubljana
- Last refreshed: 10 November 2022
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/NCT04587492.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing