Clinical Research Directory
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3 clinical studies listed.
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Tundra lists 3 SMA - Spinal Muscular Atrophy clinical trials. Each listing includes eligibility criteria, study locations, and direct links to research sites in the Tundra directory.
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NCT07467187
Invasive Home Ventilation in Denmark
The aim of this study is to describe national trends over the past 10 years in patients receiving invasive home mechanical ventilation (HMV) in Denmark. This includes indications for invasive HMV, diagnostic groups, and one-year mortality.
Gender: All
Updated: 2026-03-27
NCT07223320
HABIT-ILE + FST in Children With SMA
This single-arm pilot study will assess the feasibility and preliminary effectiveness of an intensive motor skill intervention (HABIT-ILE) combined with functional strength training (FST) in children with SMA who are receiving disease-modifying therapies. Participants will attend one 6-hour HABIT-ILE + FST session each weekend (Saturday or Sunday) for 15 weeks, for a total of 90 hours of training. Feasibility will be evaluated through questionnaires, and effectiveness through standardized motor function assessments.
Gender: All
Ages: 5 Years - 17 Years
Updated: 2026-03-16
1 state
NCT06971094
Safety and Efficacy Evaluation of GC101 Gene Therapy Via Intrathecal (IT) Injectionin the Treatment of Patients With Type 2 Spinal Muscular Atrophy (SMA) - Phase III
This trial employs a multicenter, randomized, open-label, standard-of-care-controlled design and plans to enroll 50 patients with Type 2 SMA aged 2 to 12 years who have previously received nusinersen. The primary objective of the trial is to evaluate the efficacy of GC101 in treating Type 2 SMA. The secondary objectives are to assess the efficacy, safety, and pharmacokinetic (PK) profile of GC101 in treating Type 2 SMA.
Gender: All
Ages: 2 Years - 12 Years
Updated: 2025-06-05