Spinal Muscular Atrophy Clinical Trials

A listing of Spinal Muscular Atrophy medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.

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Found 38 clinical trials
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

nusinersen
gene deletion
  • 2 views
  • 07 Oct, 2022
  • 20 locations
A Randomized, Sham-controlled, Double-blind Study to Evaluate the Efficacy and Safety of Intrathecal OAV101 in Type 2 Spinal Muscular Atrophy (SMA) Patients Who Are ≥ 2 to < 18 Years of Age, Treatment Naive, Sitting, and Never Ambulatory (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
  • 24 Oct, 2022
  • 11 locations
A Randomised Study of Consolidation CTRT Versus Observation After First Line Chemotherapy in Advanced Gallbladder Cancer (RACE-GB)

This will be a phase III randomized trial of advanced gall bladder cancers. 140 patients will be randomized. Randomisation will be on a 1:1 ratio between the experimental arm and the control arm. Observation Arm : 6 cycles of Chemotherapy with Gemcitabine and Cisplatin will be followed by observation Chemotherapy …

  • 0 views
  • 07 Oct, 2022
  • 1 location
Development of Non-Invasive Brain Stimulation Techniques

Background Noninvasive brain stimulation (NIBS) may help diagnose and treat psychiatric and neurological illness. But there is not enough research on how to apply NIBS. This includes how strong to make it, where on the brain to apply it, and for how long. Researchers also want to see what the …

Accepts healthy volunteers
  • 128 views
  • 04 Oct, 2022
  • 1 location
A Phase 3b Study to Evaluate Higher Dose Nusinersen (BIIB058) in Patients With Spinal Muscular Atrophy Previously Treated With Risdiplam (ASCEND)

The primary objective of this study is to evaluate motor function following treatment with HD nusinersen in participants with spinal muscular atrophy (SMA) previously treated with risdiplam.

nusinersen
risdiplam
gene deletion
  • 0 views
  • 21 Oct, 2022
  • 12 locations
Phase 3, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Apitegromab (SRK-015) in Patients With Later-Onset Spinal Muscular Atrophy Receiving Background Nusinersen or Risdiplam Therapy (SAPPHIRE)

This Phase 3 trial (Study SRK-015-003) is being conducted in patients ≥2 years old at Screening, who were previously diagnosed with later-onset spinal muscular atrophy (SMA) (i.e., Type 2 and

nusinersen
risdiplam
  • 0 views
  • 23 Oct, 2022
  • 24 locations
  • 0 views
  • 23 Oct, 2022
  • 8 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
  • 04 Oct, 2022
  • 1 location
Long-Term Follow-Up Study of Patients With Spinal Muscular Atrophy Receiving Risdiplam Treatment (WeSMA)

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
  • 15 Oct, 2022
  • 9 locations
Pharmacokinetic Study of Skeletal Muscle Area-based Paclitaxel Infusion in Patients With Breast Cancer

The primary objective of this pharmacokinetics study is to compare the maximum concentration level of paclitaxel in patients with low/sarcopenic skeletal muscle area (SMA), at the end of a 2-3

doxorubicin
pertuzumab
cyclophosphamide
trastuzumab
carboplatin
  • 0 views
  • 04 Oct, 2022
  • 1 location