An observational study in Spinal Muscular Atrophy, sponsored by Institut de Myologie, France. Completed at 6 sites in France. Per ClinicalTrials.gov, last updated 2021-09-13.
Sponsored by Institut de Myologie, France · Observational
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Among 39 studies in Muscular Atrophy, Spinal with a site in France — 7 open to participants now, 12 under way, 2 of them phase 3. Muscular Atrophy, Spinal · France.
Among the 39 studies in Muscular Atrophy, Spinal with a site in France, this one is one of 3 that started in 2017, is one of 6 observational ones, and was the 14th registered.
282 studies on the registry are indexed under Muscular Atrophy, Spinal; 73 are open to participants now.
Browse Muscular Atrophy, Spinal studies →7 of the 39 studies in Muscular Atrophy, Spinal with a site in France are open now. Recruiting first, the newest first:
Institut de Myologie, France · Sites in France
Ages 18–65 · Healthy volunteers
Scholar Rock, Inc. · Sites in France, United States, Belgium and 4 more countries
Ages up to 2
University Hospital, Rouen · Sites in France
Ages 18+
Biogen · Sites in France, United States, Germany and 4 more countries
Ages 3+
Centre Hospitalier Universitaire de Saint Etienne · Sites in France
Ages 6+
A study can be open while its sites in France have not opened yet or have stopped. Only those recruiting at a site in France →
Of the 58 Muscular Atrophy, Spinal studies recruiting now, 28 (48%) set no upper age limit and 27 (47%) stop at 75 or younger; 46 take children under 18; 11 take healthy volunteers.
This study: all sexes · no healthy volunteers.
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39 studies; the count open now beside each. This study's row is marked.
First in 2012; this study’s year is marked. 3 started in 2017.
This study's enrollment of 100 is below the median of 139 across 114 observational studies indexed under Muscular Atrophy, Spinal.
Median enrollment 52 across the 33 interventional studies, 125 across the 6 observational. Of the 24 that completed or were terminated, 9 (38%) have results posted.
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17 of 39 have an industry lead sponsor. The 10 most frequent:
Institut de Myologie, France is the lead sponsor of 30 studies on the registry; 5 are open to participants now.
Institut de Myologie, France has 5 studies filed under Muscular Atrophy, Spinal — fewer than ten, so there is no comparison to draw.
3 of the studies in Muscular Atrophy, Spinal with a site in France that are under way reach primary completion within a year, 0 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT07321977Institut de Myologie, FranceAssessment of a Portable Digital Device for Quantified Analysis of Markerless Walking in Volunteers With Neuromuscular Diseases or Asymptomatic Volunteers | 2026-12-31 |
| NCT07332702University Hospital, RouenLong Read Analysis in Spinal Muscular Atrophy - LOREASI | 2027-01-15 |
| NCT06555419BiogenA Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFlex DRx™ System in Adult and Pediatric Participants With Spinal Muscular Atrophy (PIERRE-PK) | 2027-09-30 |
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Among the 39 studies in Muscular Atrophy, Spinal with a site in France, from their registry updates:
This tab as a report to print or save as PDF → With an account.
IO-SMA-Registry is a prospective, longitudinal and observational study which objective is to collect prospectively information on longevity, psychomotor development and respiratory function of patients with infantile-onset spinal muscular atrophy.
Neuromuscular reference centers
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Change from Baseline in survival
Time frame: Baseline and then every 6 months until the end of the study, up to 5 years
Change from Baseline in psychomotor development
Motor milestones acquired and/or lost
Time frame: Baseline and then every 6 months until the end of the study, up to 5 years
Change from Baseline in the number of lower track infections
Time frame: Baseline and then every 6 months until the end of the study, up to 5 years
Change from Baseline in ventilation use
Time frame: Baseline and then every 6 months until the end of the study, up to 5 years
Change from Baseline in cough assist use
Time frame: Baseline and then every 6 months until the end of the study, up to 5 years
Change from Baseline in Forced Vital Capacity
Time frame: Baseline and then every 6 months until the end of the study, up to 5 years
Change from Baseline in diurnal saturation
Time frame: Baseline and then every 6 months until the end of the study, up to 5 years
Change from Baseline in nocturnal hypercapnia
Time frame: Baseline and then every 6 months until the end of the study, up to 5 years
Change from the beginning of the treatment of psychomotor development
Retrospective and prospective collection of data from patients/parents interview and medical files Motor milestones acquired and/or lost
Time frame: Since the beginning of the treatment until the end of the study, up to 5 years
Change from the beginning of the treatment of the number of hospitalizations
Retrospective and prospective collection of data from patients/parents interview and medical files
Time frame: Since the beginning of the treatment until the end of the study, up to 5 years
Change from the beginning of the treatment of the duration of hospitalizations
Retrospective and prospective collection of data from patients/parents interview and medical files
Time frame: Since the beginning of the treatment until the end of the study, up to 5 years
Change from Baseline of Clinical Global Impressions - Improvement (CGI-I)
Quantification of patient progress and treatment response over time
Time frame: Baseline and then every 6 months until the end of the study, up to 5 years
Change from Baseline of the scoliosis occurence
Time frame: Baseline and then every 6 months until the end of the study, up to 5 years
Change from Baseline of the arthrodesis occurence
Time frame: Baseline and then every 6 months until the end of the study, up to 5 years
Change from Baseline of contractures occurrence
Time frame: Baseline and then every 6 months until the end of the study, up to 5 years
Change from Baseline of wheelchair use
Time frame: Baseline and then every 6 months until the end of the study, up to 5 years
Change from Baseline of feeding status
Feeding difficulties (swallowing, chewing, sucking), excessive drooling, need of a feeding tube, occurrence of gastrostomy
Time frame: Baseline and then every 6 months until the end of the study, up to 5 years
Change from Baseline of speech impairment
Speech incapacity, voice tone disorders
Time frame: Baseline and then every 6 months until the end of the study, up to 5 years
Change from Baseline of Hammersmith Infant Neurological Examination (HINE) score
Time frame: Baseline and then every 6 months until the end of the study, up to 5 years
Change from Baseline of Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) score
Time frame: Baseline and then every 6 months until the end of the study, up to 5 years
Change from Baseline of Motor Function Measure (MFM) score
Time frame: Baseline and then every 6 months until the end of the study, up to 5 years
Change from Baseline of Expanded Hammersmith Functional Motor Scale (HFMSE) score
Time frame: Baseline and then every 6 months until the end of the study, up to 5 years
Change from Baseline of the number of physiotherapy sessions per week
Time frame: Baseline and then every 6 months until the end of the study, up to 5 years
Change from Baseline of the number of balneotherapy sessions per week
Time frame: Baseline and then every 6 months until the end of the study, up to 5 years
Change from Baseline of the number of occupational therapy sessions per week
Time frame: Baseline and then every 6 months until the end of the study, up to 5 years
Plan to share: Undecided
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Ran from Oct 2017 to primary completion in Nov 2020, 37 months.
Of 58 observational studies under Muscular Atrophy, Spinal, half reached primary completion within 88 months of starting (the median), counting those still running; a quarter within 36. This study's 37 months is below that.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Sep 2021. You cannot join it, but the record below documents what was studied.
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Institut de Myologie, France