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NCT04587492

Metabolomics of Children With SMA

Completed Last updated 10 November 2022
What this trial tests

trial testing Nusinersen in Spinal Muscular Atrophy in 35 participants. Completed in 30 September 2020.

Timeline
1 January 2017
Primary endpoint
30 September 2020
30 September 2020

Quick facts

Lead sponsorUniversity Medical Centre Ljubljana
StatusCompleted
Study typeOBSERVATIONAL
Enrollment35
Start date1 January 2017
Primary completion30 September 2020
Estimated completion30 September 2020
Sites2 locations across Slovenia

Drugs / interventions tested

Conditions studied

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):

  1. 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
  2. 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:

Other trials of Nusinersen

Trials testing the same drug.

Other recruiting trials for Spinal Muscular Atrophy

Currently open trials in the same condition.

Other University Medical Centre Ljubljana trials

Trials by the same sponsor.

Verify against primary sources

Data sources for this page

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