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Found 60 clinical trials
Registry and Natural History Study for Early Onset Hereditary Spastic Paraplegia (HSP)

The Registry and Natural History Study for Early Onset Hereditary Spastic Paraplegia (HSP) is focused on gathering longitudinal clinical data as well as biological samples (skin and/or blood and/or saliva) from male or female patients who exhibited onset of HSP symptoms at 18 years old or younger with (1) a …

Accepts healthy volunteers
  • 21 Jul, 2022
  • 1 location
The Impact of Charcot-Marie-Tooth Disease in the Real World

An observational, non-interventional registry study to collect real-world data from people living with Charcot-Marie-Tooth disease (CMT) and its treatment, which will be available to researchers to further the knowledge of Charcot-Marie-Tooth disease and improve patient care.

  • 16 May, 2022
  • 1 location
A Study of Imlifidase in Patients With Guillain-Barré Syndrome

The study participants are patients which have been diagnosed with Guillain-Barré Syndrome (GBS) and are planned to receive treatment with intravenous immunoglobulin (IVIg). IVIg is a standard of care treatment for GBS patients. The patients in this study will be treated with the study medicine imlifidase on day 1, and …

  • 26 Apr, 2022
  • 9 locations
Immunoadsorption Versus Plasma Exchange for Treatment of Guillain-Barr Syndrome (GBS)

This is an observations study evaluating safety and efficacy of immunoadsorption compared to plasma exchange in Guillain-Barr Syndrome.

  • 10 Oct, 2021
  • 1 location
Tools for Therapeutic Evaluation in Charcot-Marie-Tooth Disease Type 1A: Outcome Measures and Biomarkers (CMT-TOOLS)

This is a 2-year follow-up study of a cohort of 60 CMT1A patients. The objective is to identify markers allowing to better understand the phenotypic variability observed on patients with CMT1A, to identify predictive markers of the disease's progression and to provide validated measurement tools that can be used as …

Accepts healthy volunteers
  • 10 Aug, 2022
  • 2 locations
Intrathecal Administration of scAAV9/JeT-GAN for the Treatment of Giant Axonal Neuropathy

Title: Intrathecal Administration of scAAV9/JeT-GAN for the Treatment of Giant Axonal Neuropathy Background The Gigaxonin gene lets the body make a protein chemical called Gigaxonin. Nerves need Gigaxonin to work properly. Giant Axonal Neuropathy (GAN) causes a shortage of functional Gigaxonin. Nerves stop working normally in people with GAN. This …

skin biopsy
MRI Scan
  • 28 Jul, 2022
  • 1 location
Efficacy and Safety of ANX005 in Subjects With Guillain-Barr Syndrome

This study is intended to evaluate the efficacy and safety of ANX005 administered by intravenous (IV) infusion to participants recently diagnosed with Guillain-Barr Syndrome (GBS). The total duration of study participation is approximately 6 months.

  • 28 Jan, 2021
  • 1 location
Firm Observational Clinical Unicenter Study on Guillain Barré Syndrome (FOCUS on GBS)

The aim of this retrospective observational single-center cohort study is to gain a deeper understanding regarding the frequency, the clinical and electrophysiologic characteristics (e.g., the diagnostic work up), complications, treatment regimes, and their associations with specific courses of disease and outcomes in adult patients with the suspected or proven diagnosis …

  • 10 May, 2022
  • 1 location
Validation Study of Posturology Platforms for Evaluating Postural Control of Hemiparetic and Neuro-muscular Patients (KPLATES)

The instrumental evaluation of standing postural control by posturographic analysis supplements the clinical evaluation and, as such, is recommended by the French National Authority for Health (HAS 2007). The quantitative data obtained after a standardized postural examination appear relevant for the longitudinal follow-up of neuromuscular patients and hemiparetic patients. Neuro …

Accepts healthy volunteers
  • 29 Jun, 2022
  • 1 location
Genetic and Physical Study of Childhood Nerve and Muscle Disorders

Background Some nerve and muscle disorders that start early in life (before age 25), like some forms of muscular dystrophy, can run in families. However, the genetic causes of these disorders are not known. Also, doctors do not fully understand how symptoms of these disorders change over time. Researchers want …

Accepts healthy volunteers
  • 28 Jul, 2022
  • 1 location