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Currently Enrolling Studies

neuromuscular ultrasound. Dr. Glenn

  • A Phase II Randomized, Placebo Controlled Trial of Tocilizumab in ALS Subjects

    This research study is being done to find out if tocilizumab can help with ALS.

  • A Phase II, Double-Blind , Randomized, Placebo-Controlled, Multiple Dose Study of CK-2127107 in two Ascending Dose Cohorts of Patients with Spinal Muscular Atrophy (SMA)

    The primary objective of this study is to demonstrate a pharmacodynamics effect of CK-2127107 on measures of skeletal muscle function or fatigability in patients with SMA Types II-IV.

  • Phenotype, Genotype & Biomarkers in ALS and Related Disorders

    • The goals of this study are: (1) to better understand the relationship between the phenotype and genotype of amyotrophic lateral sclerosis (ALS) and related diseases, including primary lateral sclerosis (PLS), hereditary spastic paraplegia (HSP), progressive muscular atrophy (PMA), and frontotemporal dementia (FTD), and (2) to develop biomarkers that might be useful in aiding therapy development for the group of disorders.

      • Study Type: Natural History

      • Who: Individuals with ALS, HSP, PMA, PLS, FTD, & MSP

      • Procedures: Questionnaires, respiratory function, blood collection, neurological exam

      • Duration: 5 office visits (duration depends on disease)


  • Safety and Efficacy Study of NP001 in Patients with Amyotrophic Lateral Sclerosis (ALS) and Systemic Inflammation

    This study evaluates NP001 in patients with ALS and evidence of systemic inflammation.

  • Open Label Study of Subcutaneous Immunoglobulin (SCIg) in Myasthenia Gravis

    The purpose of this study is to determine whether Hizentra is a safe and effective treatment for people with myasthenia gravis (MG).

  • A Study to Evaluate the Efficacy and Safety of IVIG-C in Symptomatic subjects with Generalized Myasthenia Gravis

    The primary objective is to evaluate whether IVIG-C improves MG symptoms as compared to placebo in subjects with MG.

  • Agrin/LRP4

    The purpose of this study is to collect blood from MG patients and determine whether they are LRP4/Argin positive.

  • An Open-Label, Expanded Access Protocol for Amifampridine Phosphate Treatment in Patients with Lambert-Eaton Myasthenic Syndrome (LEMS), Congenital Myasthenic Syndrome (CMS) and downbeat Nystagmus

    The primary objective of the study is to provide patients with LEMS/CMS/downbeat nystagmus access to amifampridine phosphate therapy until the product becomes commercially available.

  • Peripheral Neuropathy Research Registry (PNRR) Consortium Partners

    The Foundation for Peripheral Neuropathy (FPN) has established a network of leading neurological consortium partner clinics who work with each other and FPN to build the PNRR.

  • A Phase III, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Assess the Safety and Efficacy of VM202 in subjects with Painful Diabetic Peripheral Neuropathy

    The purpose of this study is to determine the safety and efficacy of bilateral intramuscular injections of VM202 versus placebo in the treatment of painful diabetic neuropathy.

  • Patient Assisted Intervention for Neuropathy: Comparison of Treatment in Real Life Situations (PAIN-CONTRoLs)

    The purpose of this large comparative effectiveness study is to learn about the safety and effectiveness of nortriptyline, duloxetine, pregabaline, and mexiletine in treating cryptogenic sensory polyneuropathy (CSPN).

  • A Phase 2, Randomized, Double-Blind, Placebo-Controlled Trail of IMO-8400 in Patients with Dermatomyositis

    The purpose of this study is to determine how safe and effective IMO-8400 is in adults with dermatomyositis.

  • Tocilizumab in the Treatment of Refractory Polymyositis and Dermatomyositis

    The purpose of this multi-center pilot study is to determine if the drug tocilizumab (Actemra) is effective in the treatment of patients with refractory adult polymyositis (PM) and dermatomyositis (DM).

  • An Open-Label, Multi-Center Study to evaluate the Safety, Efficacy and Tolerability of Etiplirsen in Early Stage Duchenne Muscular Dystrophy (Sarepta 4658-301)

    This is an open-label study to assess the safety, tolerability, efficacy and pharmacokinetics of etiplirsen in patients with early stage Duchenne muscular dystrophy (DMD) who are amendable to exon 51 skipping.

  • A Double-Blind, Placebo-Controlled, Multi-Center Study with an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients with Duchenne Muscular Dystrophy (Sarepta 4045-301)

    The main objective of this study is to evaluate the efficacy of SRP-4045 and SRP-4053 compared to placebo in DMD patients with out-of-frame deletion mutations amenable to skipping exon 45 and exon 53 respectively. Additional objectives include evaluation of safety, pharmacokinetics and biomarkers.

  • A Phase 2 Randomized, Double-blind, Placebo-Controlled, Multiple-Ascending Dose Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Pf-06252616 in Ambulatory Boys with DMD (Pfizer B5161002)

    The purpose of this study is to administer PF-06252616 to ambulatory boys diagnosed with DMD.

  • A Phase 3 Double-Blind, Placebo-Controlled Study Assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids (Sideros)

    The purpose of the study is to assess the efficacy of Idebenone in delaying the respiratory function in patients with DMD receiving concomitant glucocorticoid steroids.

  • An Open-Label, Expanded Access Protocol Intended to Provide Treatment with MP-104 (Deflazacort) to U.S. Children, Adolescents, and/or adults with Duchenne Muscular Dystrophy

    The expanded access program will provide access to treatment with Deflazacort in children, adolescent and adults with DMD in the US who are ineligible, unable, or otherwise unwilling to enroll in a clinical study examining efficacy of Deflazacort while a new drug application in under preparation and review.

  • Rasch-Analysis of Clinical Severity in FSHD ( ROC-FSH)

The purpose of this study is to make a standardized and scalable Rasch-built clinical severity scale to help in finding genetic and environmental modifiers of disease in Facioscapulohumeral muscular dystrophy (FSHD).

      • Study Type: Natural History
      • Who: Individuals 18 or over with genetically verified FSHD1 or FSHD2
      • Procedures: Questionnaires, Physical and Neurological Exam, Facial Function Testing, Strength Testing, Functional Testing, Respiratory function testing, Blood draw
      • Duration: 1 visit approximately 4 hours
  • A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of ACE-083 in Patients with Facioscapulohumeral Muscular Dystrophy (A083-02)

Study A083-02 is a multicenter, Phase 2 study to evaluate the safety, tolerability, pharmacodynamics (PD), efficacy, and pharmacokinetics (PK) of ACE 083 in patients with FSHD to be conducted in two parts. Part 1 is open-label, dose-escalation and Part 2 is randomized, double-blind, and placebo-controlled.

  • Pompe Disease Registry

    The Pompe Registry is an ongoing, international multicenter, strictly observational program that tracks the routine clinical outcomes for patients with Pompe disease, irrespective of treatment status.

      • Study Type: Observational, registry

      • Who: Individuals with genetically diagnosed Pompe disease, must be followed by Neurology at KU Medical Center

      • Procedures: Routine office visits with physician based on standard of care

      • Duration: Duration of office visits or until consent is withdrawn


  • Investigating Pompe Prevalence in Neuromuscular Medicine Academic Practices

    This study will measure the incidence of Pompe disease based on manifest laboratory abnormality, namely low GAA enzyme activity. Analysis of GAA enzyme activity will be determined through blood sample of 4mL.

      • Study Type:

      • Who: Individuals with muscle weakness of unknown cause and/or elevated Creatinine Kinase blood values, must be scheduled to be seen by Neurology at KU Medical Center

      • Procedures: Standard of care office visit, medical history, and blood draw

      • Duration: Up to 2 office visits


Last modified: Sep 28, 2018
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