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Found 48 clinical trials
Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy (DEVOTE)

The primary objectives of this study are to examine the clinical efficacy of nusinersen administered intrathecally at higher doses to participants with spinal muscular atrophy (SMA), as measured

muscle atrophy
gene deletion
nusinersen
  • 168 views
  • 11 Jul, 2022
  • 53 locations
A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec (RESPOND)

The primary objective of this study is to evaluate the clinical outcomes following treatment with nusinersen in participants with spinal muscular atrophy (SMA) who previously received

gene deletion
nusinersen
  • 2 views
  • 09 Jul, 2022
  • 19 locations
Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA) (STEER)

To evaluate the efficacy, safety and tolerability of intrathecal (IT) OAV101 in treatment naive patients with Type 2 spinal muscular atrophy (SMA) who are ≥ 2 to < 18 years of age over a 15

  • 4 views
  • 25 Jul, 2022
  • 4 locations
Bioavailability and Bioequivalence of Two Risdiplam Tablets in Healthy Participants

The study is a randomized, single oral dose, crossover study in up to three parts to investigate the relative bioavailability and bioequivalence of two different formulations of risdiplam 5 mg (dispersible tablets) versus the current risdiplam oral solution formulation in healthy male and female participants. The effect of food on …

Accepts healthy volunteers
risdiplam
  • 2 views
  • 29 Jul, 2022
  • 1 location
Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (SMART)

To evaluate the safety, tolerability and efficacy of intravenous administration of OAV101 (AVXS-101) in patients with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival

a 12
  • 3 views
  • 04 Jun, 2022
  • 11 locations
Spinal Cord Stimulation in Spinal Muscular Atrophy (SCSinSMA)

neural circuits to residual cortical inputs. This pilot will test if SCS can show evidence to improve motor deficits in people with type 3 or 4 spinal muscular atrophy (SMA). The investigators will

  • 0 views
  • 26 Jul, 2022
  • 1 location
Virtual Reality for the Mitigation of Anxiety During Intrathecal Administration in Participants With Spinal Muscular Atrophy (REALITY)

The primary objective of the study is to evaluate anxiety level during intrathecal administration (IT) under standard of care (SOC) and virtual reality (VR) conditions using a reliable self-rating scale.

nusinersen
  • 0 views
  • 24 Jul, 2022
  • 10 locations
Long-Term Follow-Up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)

® U.S. Package Insert (USPI) in adult and pediatric participants with spinal muscular atrophy (SMA). In this study, participants will be followed for up to 5 years from enrollment or until withdrawal of

risdiplam
  • 0 views
  • 25 Jul, 2022
  • 4 locations
Outcome Measures and Biomarkers in a Cohort of Spinal Muscular Atrophy Type III/ IV Patients (SMOB)

The "SMOB" project intends to contribute to fill the gap with reliable and operational outcome measures for type III and IV SMA. In analysing the reliability in imaging (spinal and muscular), electrophysiology analysis (MUNIX), and evaluate the evolution of respiratory function for 50 patients' cohort. The investigators would also take …

atrophy
  • 0 views
  • 22 Mar, 2022
  • 4 locations
Study of the Functional Effects of Nusinersen in 5q-spinal Muscular Amyotrophy Adults (SMA Type 2 or 3 Forms) (NUSI-AD-5qSM)

Spinal Muscular Atrophy (SMA) is an autosomal recessive disease caused by a mutation of exon 7, in 95% of cases, encoding the gene for the motor neuron survival protein called SMN1 (Survival

spinal cord
nusinersen
deficit
neuromuscular diseases
  • 0 views
  • 27 Apr, 2022
  • 1 location