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Universitätsklinikum Essen
Forschung & Lehre

Klinische Studien

Duchenne Muskeldystrophie

    Long-Term Observational Study of Translarna Safety and Effectiveness in Usual Care (Anwendungsbeobachtungsstudie PTC124-GD-025o-DMD)
  • Study of SRP-4045 and SRP-4053 in DMD Patients (ESSENCE)
    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 (EudraCT: 2015-002069-52)
  • SRP 4045-302
    Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen (EudraCT:2017004625-32)
    • Tamoxifen in Duchenne Muscular Dystrophy: A multicenter, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy 48-week trial
    • Tamoxifen in Duchenne Muscular Dystrophy: A 48-week open label extension of a multicentre, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy trial (EudraCT: 2017-004554-42)
    A Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids (EudraCT: 2016-000602-10)
  • DSC/142357/48
    Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy (EudraCT: 2016-000401-36)
  • C3391003
    A phase 3, multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of PF-06939926 for the treatment of Duchenne Muscular Dystrophy
  • SRP-9001-301
    A Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9001 in Subjects With Duchenne Muscular Dystrophy Who Are Aged 4-7 Years
  • SRP 5051-201
    A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (EudraCT: 2019-000601-77)
  • SRP 4658-407
    A Natural History Study in Patients with Duchenne Muscular Dystrophy Managed in Routine Clinical Practice
  • SRP 4658-402
    A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping (EudraCT 2018-001762-42) 

Centronukleäre Myopathien

  • NatHis CNM
    Prospective, Longitudinal Study of the Natural History and Functional Status of Patients With Myotubular Myopathy (MTM)
  • LIBERI AT132-03
    An Open-Label Clinical Study to Evaluate the Safety and Efficacy of AT132, an AAV8-Delivered Gene Therapy, in Patients with X Linked Myotubular Myopathy (XLMTM) (EudraCT: 2019-004603-13)
  • DYN101-C101
    A Phase 1/2 trial on the safety, tolerability, pharmacokinetics, pharmacodynamics and exploratory efficacy of DYN101 in patients ≥ 16 years of age with centronuclear myopathies caused by mutations in DNM2 or MTM1. (EudraCT: 2018-004089-33)

Spinale Muskelatrophie

  • ISIS 396443-CS11
    An Open-label Extension Study for Patients with Spinal Muscular Atrophy who Previously Participated in Investigational Studies of ISIS 396443 (EudraCT 2015-001870-16)
  • Jewelfish BP 39054
    An open label study to investiagte the safety, tolerability, and pharmacokinetics/pharmacoynamics of Risdiplam RO7034067 in adult and pediatric patients with spinal muscular atrophy (EudraCT 2016-004184) 

Myastenia gravis

  • ECU-MG-303
    An Open-Label, Multicenter Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Eculizumab in Pediatric Patients With Refractory Generalized Myasthenia Gravis
Univ.-Prof. Ulrike Schara-Schmidt

Univ.-Prof. Ulrike Schara-Schmidt

Ltd. Ärztin Kinderneurologie, Stellvertretende Direktorin Klinik für Kinderheilkunde I

0201-723-2508 E-Mail schreiben

Dr. med. Frederik Braun


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 Corinna Seifert

Corinna Seifert

Studienkoordinatorin und Kinderkrankenschwester

0201-723-85170 E-Mail schreiben
 Karin Smuda

Karin Smuda

Studienkoordinatorin und Kinderkrankenschwester

0201-723-84601 E-Mail schreiben
 Barbara Andres

Barbara Andres

Physiotherapeutin, Klinische Studien

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